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6. Torelli GF, Guarini A, Maggio R, Alfieri C, Vitale A, Foà R: Expansion of natural killer cells with lytic activity against autologous blasts from adult and pediatric acute lymphoid leukemia patients in complete hematologic remission. Haematologica; 2005 Jun;90(6):785-92
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Expansion of natural killer cells with lytic activity against autologous blasts from adult and pediatric acute lymphoid leukemia patients in complete hematologic remission.
  • The anti-leukemic activity of NK cells against acute myeloid leukemia (AML) blasts has been described, but very few data are available for acute lymphoid leukemia (ALL).
  • The present study was designed to investigate whether: (i) NK effectors could be expanded from adult and pediatric ALL patients in complete remission;.
  • (v) any differences in cytotoxic activity could be found between expanded effectors from adult and pediatric patients.
  • No differences in expansion and cytotoxic activity were found between pediatric and adult patients.
  • INTERPRETATION AND CONCLUSIONS: These findings document for the first time the possibility of expanding ex vivo cytotoxic effectors with autologous killing capacity from ALL patients in remission, and suggest a new potential immunotherapeutic strategy for the management of early disease recurrence or of residual disease.
  • [MeSH-major] Immunotherapy / methods. Killer Cells, Natural / cytology. Precursor Cell Lymphoblastic Leukemia-Lymphoma / metabolism. Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy
  • [MeSH-minor] Adult. Child. Coculture Techniques. Cytokines / metabolism. Flow Cytometry. Humans. Interleukin-15 / metabolism. Interleukin-2 / metabolism. Receptors, IgG / metabolism. Remission Induction. Signal Transduction

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  • (PMID = 15951291.001).
  • [ISSN] 1592-8721
  • [Journal-full-title] Haematologica
  • [ISO-abbreviation] Haematologica
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] Italy
  • [Chemical-registry-number] 0 / Cytokines; 0 / Interleukin-15; 0 / Interleukin-2; 0 / Receptors, IgG
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7. Pollard JA, Alonzo TA, Gerbing RB, Ho PA, Zeng R, Ravindranath Y, Dahl G, Lacayo NJ, Becton D, Chang M, Weinstein HJ, Hirsch B, Raimondi SC, Heerema NA, Woods WG, Lange BJ, Hurwitz C, Arceci RJ, Radich JP, Bernstein ID, Heinrich MC, Meshinchi S: Prevalence and prognostic significance of KIT mutations in pediatric patients with core binding factor AML enrolled on serial pediatric cooperative trials for de novo AML. Blood; 2010 Mar 25;115(12):2372-9
ClinicalTrials.gov. clinical trials - ClinicalTrials.gov .

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  • [Title] Prevalence and prognostic significance of KIT mutations in pediatric patients with core binding factor AML enrolled on serial pediatric cooperative trials for de novo AML.
  • KIT receptor tyrosine kinase mutations are implicated as a prognostic factor in adults with core binding factor (CBF) acute myeloid leukemia (AML).
  • However, their prevalence and prognostic significance in pediatric CBF AML is not well established.
  • We performed KIT mutational analysis (exon 8 and exon 17) on diagnostic specimens from 203 pediatric patients with CBF AML enrolled on 4 pediatric AML protocols.
  • Rates of complete remission, overall survival, disease-free survival, or relapse were not significantly different for patients with or without KIT mutations.
  • Our study shows that KIT mutations lack prognostic significance in a large series of pediatric patients with CBF AML.
  • This finding, which differs from adult series and a previously published pediatric study, may reflect variations in therapeutic approaches and/or biologic heterogeneity within CBF AML.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Core Binding Factors / genetics. Leukemia, Myeloid, Acute. Proto-Oncogene Proteins c-kit / genetics

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  • (PMID = 20056794.001).
  • [ISSN] 1528-0020
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Databank-accession-numbers] ClinicalTrials.gov/ NCT00002798/ NCT00070174
  • [Grant] United States / NCI NIH HHS / CA / K12 CA076930; United States / NCI NIH HHS / CA / R21 CA10262-01; United States / NCI NIH HHS / CA / R01 CA114563-01; United States / NCI NIH HHS / CA / R01 CA114563; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / 5K12CA076930-08; United States / NCI NIH HHS / CA / U10 CA98543
  • [Publication-type] Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Core Binding Factors; EC 2.7.10.1 / Proto-Oncogene Proteins c-kit
  • [Other-IDs] NLM/ PMC2845895
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8. Ho PA, Alonzo TA, Gerbing RB, Pollard J, Stirewalt DL, Hurwitz C, Heerema NA, Hirsch B, Raimondi SC, Lange B, Franklin JL, Radich JP, Meshinchi S: Prevalence and prognostic implications of CEBPA mutations in pediatric acute myeloid leukemia (AML): a report from the Children's Oncology Group. Blood; 2009 Jun 25;113(26):6558-66
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  • [Title] Prevalence and prognostic implications of CEBPA mutations in pediatric acute myeloid leukemia (AML): a report from the Children's Oncology Group.
  • CEBPA mutations have been associated with improved outcome in adult acute myeloid leukemia (AML).
  • We evaluated the prevalence and prognostic significance of CEBPA mutations in 847 children with AML treated on 3 consecutive pediatric trials.
  • Actuarial event-free survival at 5 years was 70% versus 38% (P = .015) with a cumulative incidence of relapse from complete remission of 13% versus 44% (P = .007) for those with and without CEBPA mutations.
  • [MeSH-major] CCAAT-Enhancer-Binding Proteins / genetics. Leukemia, Myeloid / genetics. Neoplasm Proteins / genetics
  • [MeSH-minor] Acute Disease. Adolescent. Child. Child, Preschool. Clinical Trials as Topic / statistics & numerical data. DNA Mutational Analysis. DNA, Neoplasm / genetics. Disease-Free Survival. Female. Humans. Infant. Kaplan-Meier Estimate. Male. Polymorphism, Genetic. Prevalence. Prognosis. Protein Structure, Tertiary. Retrospective Studies. Treatment Outcome. Young Adult

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  • (PMID = 19304957.001).
  • [ISSN] 1528-0020
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Databank-accession-numbers] ClinicalTrials.gov/ NCT00002798/ NCT00070174
  • [Grant] United States / NCI NIH HHS / CA / T32 CA009351; United States / NCI NIH HHS / CA / R21 CA10262-01; United States / NCI NIH HHS / CA / R01 CA114563-01; United States / NCI NIH HHS / CA / R01 CA114563; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / U10 CA98543
  • [Publication-type] Journal Article; Multicenter Study; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / CCAAT-Enhancer-Binding Proteins; 0 / CEBPA protein, human; 0 / DNA, Neoplasm; 0 / Neoplasm Proteins
  • [Other-IDs] NLM/ PMC2943755
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9. Neudorf S, Sanders J, Kobrinsky N, Alonzo TA, Buxton A, Buckley JD, Howells W, Gold S, Barnard DR, DeSwarte J, Kalousek D, Lange BJ, Woods WG: Autologous bone marrow transplantation for children with AML in first remission. Bone Marrow Transplant; 2007 Aug;40(4):313-8
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Autologous bone marrow transplantation for children with AML in first remission.
  • In Children's cancer group (CCG) 2891, newly diagnosed patients with AML were randomized between standard and intensive timing induction therapies.
  • Patients in first remission who lacked an HLA matched family donor were randomized between an autologous bone marrow transplantation (ABMT) where marrow was purged with 4 hydroperoxycyclophosphamide and consolidation chemotherapy.
  • Myeloid and platelet engraftment occurred at a median of 44 and 42 days, respectively.
  • The results confirm previous studies that ABMT is effective post remission therapy for pediatric patients with AML in first remission.
  • [MeSH-major] Bone Marrow Transplantation / methods. Leukemia, Myeloid, Acute / therapy. Remission Induction / methods


10. Honda Y, Manabe A, Tsuchida M, Zaike Y, Masunaga A, Inoue M, Kobayashi R, Ohtsuka Y, Kikuchi A, Nakahata T, MDS Committee, the Japanese Society of Pediatric Hematology: Clinicopathological characteristics of erythroblast-rich RAEB and AML M6a in children. Int J Hematol; 2008 Dec;88(5):524-9
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  • [Title] Clinicopathological characteristics of erythroblast-rich RAEB and AML M6a in children.
  • The distinction between RAEB, RAEB-T and AML M6a is difficult when erythroblasts in the bone marrow (BM) exceed 50%.
  • We analyzed 19 children (2 RAEB, 13 RAEB-T and 4 AML M6a) enrolled in a prospective pathological central review in Japan and divided them into two groups according to the myeloblasts percentage among non-erythroid cells in BM: group A (n = 8), 5-19% myeloblasts; group B (n = 11), 20% or more myeloblasts.
  • Six with group A and seven with group B treated with AML type chemotherapy achieved complete remission.
  • Erythroblast-rich RAEB and AML M6a in children have similar characteristics and may belong to a single disease entity.
  • [MeSH-major] Anemia, Refractory, with Excess of Blasts / pathology. Erythroblasts / pathology. Granulocyte Precursor Cells / pathology. Leukemia, Myeloid, Acute / pathology
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Female. Humans. Infant. Japan. Leukocyte Count. Male. Prospective Studies. Remission Induction. Stem Cell Transplantation. Time Factors. Transplantation, Homologous

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  • (PMID = 18951200.001).
  • [ISSN] 1865-3774
  • [Journal-full-title] International journal of hematology
  • [ISO-abbreviation] Int. J. Hematol.
  • [Language] eng
  • [Publication-type] Journal Article; Multicenter Study
  • [Publication-country] United States
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16. Woods WG: Curing childhood acute myeloid leukemia (AML) at the half-way point: promises to keep and miles to go before we sleep. Pediatr Blood Cancer; 2006 May 1;46(5):565-9
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Curing childhood acute myeloid leukemia (AML) at the half-way point: promises to keep and miles to go before we sleep.
  • Childhood and adolescent acute myeloid leukemia (AML) is traditionally one of the hardest childhood cancers to successfully treat and had an overall survival well under 10% in the 1960s.
  • (1) the role of aggressive induction therapy in not only improving CR rates but in post-remission outcomes; and (2) the role of aggressive post-remission therapy in further improving survival, with an emphasis on high-dose Ara C-based chemotherapy, BMT, and supportive care.
  • Some of the challenges that will lead to ongoing reduction of population-based mortality for AML through young adulthood include:.
  • (1) improving access of adolescents to pediatric AML therapy;.
  • (3) individualized therapy based on individual genetics and leukemia cell biology;.
  • [MeSH-major] Leukemia, Myeloid / therapy
  • [MeSH-minor] Acute Disease. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Child. Disease-Free Survival. Enzyme Inhibitors / therapeutic use. Hematopoietic Stem Cell Transplantation. Humans. Remission Induction. Transplantation, Autologous. Transplantation, Homologous. Treatment Outcome


17. Berger M, Ferrero I, Vassallo E, Gastaldo L, Carraro F, Biasin E, Madon E, Fagioli F: Stem cell transplantation as consolidation therapy for children in first-remission AML: a single-center report. Pediatr Hematol Oncol; 2005 Oct-Nov;22(7):597-608
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  • [Title] Stem cell transplantation as consolidation therapy for children in first-remission AML: a single-center report.
  • A large number of patients affected by acute myeloid leukemia (AML) achieve complete remission following induction chemotherapy based on high-dose aracytin and anthracyclines.
  • However, a postremission consolidation treatment appears to be essential to maintain the remission status.
  • Sixteen patients with newly diagnosed AML received induction chemotherapy according to the AIEOP LAM 92P/Mod protocol.
  • Patients with very high-risk AML (cytogenetics with t(9;22), hyperleukocytosis (540x10(9)/L), and AML-M7 with trilineage myelodysplasia) underwent unrelated donor transplantation.
  • All patients achieved complete remission following one course.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Leukemia, Myeloid, Acute / therapy. Stem Cell Transplantation
  • [MeSH-minor] Adolescent. Antineoplastic Agents / administration & dosage. Bone Marrow Purging / methods. Child. Child, Preschool. Cytarabine / administration & dosage. Disease-Free Survival. Etoposide / administration & dosage. Female. Humans. Idarubicin / administration & dosage. Infant. Male. Recurrence. Remission Induction. Retrospective Studies. Transplantation, Autologous. Transplantation, Homologous

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  • (PMID = 16166053.001).
  • [ISSN] 0888-0018
  • [Journal-full-title] Pediatric hematology and oncology
  • [ISO-abbreviation] Pediatr Hematol Oncol
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 04079A1RDZ / Cytarabine; 6PLQ3CP4P3 / Etoposide; ZRP63D75JW / Idarubicin
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18. Shah M, Agarwal B: Recent advances in management of acute myeloid leukemia (AML). Indian J Pediatr; 2008 Aug;75(8):831-7
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Recent advances in management of acute myeloid leukemia (AML).
  • Acute myeloid leukemia (AML) is the most common childhood malignancy.
  • AML has therapeutically been difficult to treat.
  • In 2001, the World Health Organization (WHO), in conjunction with the Society for Hematopathology and the European Association of Hematopathology, published a new classification for myeloid neoplasms.
  • A number of chromosomal abnormalities are used to predict outcome and stratify therapeutic risk groups in children with AML.
  • Recently, alterations in receptor tyrosine kinases, tyrosine phosphatases and in oncogenes such as RAS have been implicated in the pathogenesis of AML.
  • This article aims to review the recent development in diagnosis, treatment and monitoring of AML.
  • Better understanding of the molecular pathogenesis of AML has led to the development of target-specific therapies.
  • The role of allogenic SCT, particularly whether it should be done during first CR or reserved for second remission, remains the most controversial issue in pediatric AML.
  • There is a need of collaboration with international pediatric cooperative oncology groups and definitive clinical trials in order to establish use of these newer molecules in pediatric populations.
  • [MeSH-major] Antibodies, Monoclonal / therapeutic use. Antineoplastic Agents / therapeutic use. Immunologic Factors / therapeutic use. Leukemia, Myeloid, Acute / therapy. Neoplasm, Residual / drug therapy
  • [MeSH-minor] Antigens, CD / blood. Antigens, Differentiation, Myelomonocytic / blood. Child. Child, Preschool. Humans. Prognosis. Remission Induction. Sialic Acid Binding Ig-like Lectin 3. fms-Like Tyrosine Kinase 3 / antagonists & inhibitors. fms-Like Tyrosine Kinase 3 / genetics

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  • (PMID = 18769895.001).
  • [ISSN] 0973-7693
  • [Journal-full-title] Indian journal of pediatrics
  • [ISO-abbreviation] Indian J Pediatr
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] India
  • [Chemical-registry-number] 0 / Antibodies, Monoclonal; 0 / Antigens, CD; 0 / Antigens, Differentiation, Myelomonocytic; 0 / Antineoplastic Agents; 0 / CD33 protein, human; 0 / Immunologic Factors; 0 / Sialic Acid Binding Ig-like Lectin 3; EC 2.7.10.1 / fms-Like Tyrosine Kinase 3
  • [Number-of-references] 43
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19. Ravindranath Y, Chang M, Steuber CP, Becton D, Dahl G, Civin C, Camitta B, Carroll A, Raimondi SC, Weinstein HJ, Pediatric Oncology Group: Pediatric Oncology Group (POG) studies of acute myeloid leukemia (AML): a review of four consecutive childhood AML trials conducted between 1981 and 2000. Leukemia; 2005 Dec;19(12):2101-16
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  • [Title] Pediatric Oncology Group (POG) studies of acute myeloid leukemia (AML): a review of four consecutive childhood AML trials conducted between 1981 and 2000.
  • From 1981 to 2000, a total of 1823 children with acute myeloid leukemia (AML) enrolled on four consecutive Pediatric Oncology Group (POG) clinical trials.
  • POG 8101 demonstrated that the induction rate associated with the 3+7+7 combination of daunorubicin, Ara-C, and 6-thioguanine (DAT) was greater than that associated with an induction regimen used to treat acute lymphoblastic leukemia (82 vs 61%; P=0.02).
  • The POG 9421 AML study evaluated high-dose Ara-C as part of the first induction course and the use of the multidrug resistance modulator cyclosporine.
  • Preliminary results showed that patients receiving both high-dose Ara-C for remission induction and the MDR modulator for consolidation had a superior outcome (5-year EFS estimate, 42+/-8.2%) than did patients receiving other treatment; however, the difference was not statistically significant.
  • These four studies demonstrate the importance of dose intensification of Ara-C in the treatment of childhood AML; cytogenetics as the single most prognostic factor and the unique curability of AML in children with Down's syndrome.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Antineoplastic Protocols / standards. Leukemia, Myeloid / therapy
  • [MeSH-minor] Acute Disease. Adolescent. Bone Marrow Transplantation. Child. Child, Preschool. Cytarabine / therapeutic use. Dose-Response Relationship, Drug. Down Syndrome / complications. Down Syndrome / drug therapy. Follow-Up Studies. Humans. Infant. Infant, Newborn. Prognosis. Remission Induction / methods. Survival Analysis. Treatment Outcome

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  • (PMID = 16136167.001).
  • [ISSN] 0887-6924
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA-03161; United States / NCI NIH HHS / CA / CA-05587; United States / NCI NIH HHS / CA / CA-07431; United States / NCI NIH HHS / CA / CA-11233; United States / NCI NIH HHS / CA / CA-15525; United States / NCI NIH HHS / CA / CA-15898; United States / NCI NIH HHS / CA / CA-20549; United States / NCI NIH HHS / CA / CA-25408; United States / NCI NIH HHS / CA / CA-28383; United States / NCI NIH HHS / CA / CA-28439; United States / NCI NIH HHS / CA / CA-28476; United States / NCI NIH HHS / CA / CA-29139; United States / NCI NIH HHS / CA / CA-29293; United States / NCI NIH HHS / CA / CA-29691; United States / NCI NIH HHS / CA / CA-32053; United States / NCI NIH HHS / CA / CA-33587; United States / NCI NIH HHS / CA / CA-33603; United States / NCI NIH HHS / CA / CA-33625; United States / NCI NIH HHS / CA / CA-41573; United States / NCI NIH HHS / CA / CA-69177; United States / NCI NIH HHS / CA / CA-69428
  • [Publication-type] Clinical Trial; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, N.I.H., Extramural
  • [Publication-country] England
  • [Chemical-registry-number] 04079A1RDZ / Cytarabine
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20. Trobaugh-Lotrario AD, Kletzel M, Quinones RR, McGavran L, Proytcheva MA, Hunger SP, Malcolm J, Schissel D, Hild E, Giller RH: Monosomy 7 associated with pediatric acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS): successful management by allogeneic hematopoietic stem cell transplant (HSCT). Bone Marrow Transplant; 2005 Jan;35(2):143-9
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  • [Title] Monosomy 7 associated with pediatric acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS): successful management by allogeneic hematopoietic stem cell transplant (HSCT).
  • Pediatric acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) with monosomy 7 is associated with poor disease-free survival when treated by conventional chemotherapy, immunosuppression or supportive measures.
  • To better understand the curative potential of HSCT in these patients, all cases of AML and MDS with monosomy 7 treated by two transplant programs (1992 to present) were reviewed.
  • Primary diagnoses were MDS (N = 5), therapy-related MDS (N = 3), AML (N = 5) and therapy-related AML (N = 3).
  • Toxicity caused deaths of the five nonsurviving patients, four of whom were transplanted with active leukemia.
  • Allogeneic HSCT is effective therapy for childhood AML and MDS associated with monosomy 7, particularly for patients with AML in complete remission and MDS.
  • [MeSH-major] Chromosomes, Human, Pair 7. Hematopoietic Stem Cell Transplantation. Leukemia, Myeloid / therapy. Monosomy. Myelodysplastic Syndromes / therapy
  • [MeSH-minor] Acute Disease. Adolescent. Cause of Death. Child. Child, Preschool. Disease Management. Female. Humans. Male. Neoplasms, Second Primary / mortality. Neoplasms, Second Primary / therapy. Remission Induction. Retrospective Studies. Survival Rate. Transplantation, Homologous. Treatment Outcome


21. Slats AM, Egeler RM, van der Does-van den Berg A, Korbijn C, Hählen K, Kamps WA, Veerman AJ, Zwaan CM: Causes of death--other than progressive leukemia--in childhood acute lymphoblastic (ALL) and myeloid leukemia (AML): the Dutch Childhood Oncology Group experience. Leukemia; 2005 Apr;19(4):537-44
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  • [Title] Causes of death--other than progressive leukemia--in childhood acute lymphoblastic (ALL) and myeloid leukemia (AML): the Dutch Childhood Oncology Group experience.
  • We analyzed causes of death, other than resistant disease or relapse, in 875 children with acute lymphoblastic leukemia (ALL) and 229 with acute myeloid leukemia (AML), treated on three different Dutch Childhood Oncology Group (DCOG) ALL and three AML protocols.
  • Overall, 23 (2.6%) ALL and 44 (19.2%) AML patients died.
  • Early death (ED, before remission was reached) occurred in nine ALL (1%) and thirty AML (13.1%) patients, including three and ten deaths before treatment was initiated.
  • Chemotherapy-related mortality in remission (CRM) occurred in nine ALL (1.1%) and eight AML (4.4%) patients.
  • For AML a decrease in ED was observed (from 26% to approximately 10%), but counter-balanced by an increase in CRM (from 3 to 8%), maybe related to the scheduling of intensification blocks in AML-92/94.
  • Including transplant-related mortality, death in CR rates in AML increased from 3 to 15% in the last study.
  • We conclude that mortality dropped favorably in ALL, but not in AML.
  • Especially for AML, effective but less toxic therapy and better supportive care guidelines need to be developed.
  • [MeSH-major] Leukemia, Myeloid / mortality. Precursor Cell Lymphoblastic Leukemia-Lymphoma / mortality
  • [MeSH-minor] Acute Disease. Adolescent. Antineoplastic Agents / adverse effects. Cause of Death. Child. Child, Preschool. Female. Humans. Infant. Male. Netherlands / epidemiology. Remission Induction


22. MRD-AML-BFM Study Group, Langebrake C, Creutzig U, Dworzak M, Hrusak O, Mejstrikova E, Griesinger F, Zimmermann M, Reinhardt D: Residual disease monitoring in childhood acute myeloid leukemia by multiparameter flow cytometry: the MRD-AML-BFM Study Group. J Clin Oncol; 2006 Aug 1;24(22):3686-92
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  • [Title] Residual disease monitoring in childhood acute myeloid leukemia by multiparameter flow cytometry: the MRD-AML-BFM Study Group.
  • PURPOSE: Monitoring of residual disease (RD) by flow cytometry in childhood acute myeloid leukemia (AML) may predict outcome.
  • PATIENTS AND METHODS: Five hundred forty-two specimens of 150 children enrolled in the AML-Berlin-Frankfurt-Muenster (BFM) 98 study were analyzed by four-color immunophenotyping at up to four predefined time points during treatment.
  • For each of the 12 leukemia-associated immunophenotypes and time points, a threshold level based on a previous retrospective analysis of another cohort of children with AML and on control bone marrows was determined.
  • Compared with commonly defined risk factors in the AML-BFM studies, flow cytometry does not provide additional information for outcome prediction, but may be helpful to evaluate the remission status at day 28.
  • [MeSH-major] Flow Cytometry. Leukemia, Myeloid, Acute / diagnosis. Neoplasm, Residual / diagnosis


23. Horikoshi Y, Kobayashi R, Endo M, Watanabe A, Kikuta A, Koike K, Hanada R, Hosoya R, Ohara A, Ikuta K, Goto H, Asami K, Sugita K, Horibe K, Tsurusawa M, Hori T, Hara J, Nishimura S, Nagatoshi Y, Mugishima H, Ohta S, Adachi S, Tsukimoto I: [Effectiveness of remission induction with high-dose cytarabine for relapsed or refractory pediatric acute leukemia]. Rinsho Ketsueki; 2010 Feb;51(2):104-13
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  • [Title] [Effectiveness of remission induction with high-dose cytarabine for relapsed or refractory pediatric acute leukemia].
  • We conducted a multicenter postmarketing study to investigate the efficacy and safety of reinduction therapy with a high-dose cytarabine-containing regimen for pediatric patients with relapsed or refractory acute leukemia.
  • Seven of 13 patients (53.8%) with ALL achieved complete or partial remission, and only 1 of 6 patients (16.7%) with AML achieved partial remission.
  • These results indicated that a high-dose cytarabine regimen is effective as reinduction therapy in pediatric patients with relapsed ALL, and supportive care is essential to prevent or control treatment-related adverse events, such as infection.
  • [MeSH-major] Antimetabolites, Antineoplastic / administration & dosage. Cytarabine / administration & dosage. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Female. Humans. Infant. Invasive Pulmonary Aspergillosis / etiology. Invasive Pulmonary Aspergillosis / prevention & control. Leukemia, Myeloid, Acute / drug therapy. Male. Pulse Therapy, Drug. Recurrence. Remission Induction. Treatment Outcome

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  • (PMID = 20379101.001).
  • [ISSN] 0485-1439
  • [Journal-full-title] [Rinshō ketsueki] The Japanese journal of clinical hematology
  • [ISO-abbreviation] Rinsho Ketsueki
  • [Language] jpn
  • [Publication-type] Clinical Trial; English Abstract; Journal Article; Multicenter Study
  • [Publication-country] Japan
  • [Chemical-registry-number] 0 / Antimetabolites, Antineoplastic; 04079A1RDZ / Cytarabine
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4. Bachas C, Schuurhuis GJ, Hollink IH, Kwidama ZJ, Goemans BF, Zwaan CM, van den Heuvel-Eibrink MM, de Bont ES, Reinhardt D, Creutzig U, de Haas V, Assaraf YG, Kaspers GJ, Cloos J: High-frequency type I/II mutational shifts between diagnosis and relapse are associated with outcome in pediatric AML: implications for personalized medicine. Blood; 2010 Oct 14;116(15):2752-8
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] High-frequency type I/II mutational shifts between diagnosis and relapse are associated with outcome in pediatric AML: implications for personalized medicine.
  • Although virtually all pediatric patients with acute myeloid leukemia (AML) achieve a complete remission after initial induction therapy, 30%-40% of patients will encounter a relapse and have a dismal prognosis.
  • To determine relevance of established AML type I/II mutations that may serve as therapeutic targets, we assessed frequencies of these mutations and their persistence during disease progression in a large group (n = 69) of paired diagnosis and relapse pediatric AML specimens.
  • [MeSH-major] Leukemia, Myeloid, Acute / genetics. Mutation. Precision Medicine

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  • [CommentIn] Blood. 2010 Oct 14;116(15):2622-3 [20947687.001]
  • (PMID = 20592250.001).
  • [ISSN] 1528-0020
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Biomarkers, Tumor; 0 / DNA Primers; 0 / DNA, Neoplasm; EC 2.7.10.1 / FLT3 protein, human; EC 2.7.10.1 / fms-Like Tyrosine Kinase 3
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25. Sayar D, Burstein Y, Bielorai B, Toren A, Dvir R: Upfront use of gemtuzumab ozogamicin in young children with CD33-positive AML. Pediatr Blood Cancer; 2010 Jul 15;55(1):183-5
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  • [Title] Upfront use of gemtuzumab ozogamicin in young children with CD33-positive AML.
  • Gemtuzumab ozogamicin (GO) is a humanized anti-CD33 antibody used for treating patients with CD33+ acute myeloid leukemia (AML).
  • We report three young children (two infants and one toddler) with AML treated with GO 9 mg/m(2).
  • GO was well tolerated and all three achieved remission.
  • All were transplanted: one relapsed after 5 months and died of disease, one died a toxic death in remission due to pulmonary fibrosis, and one survived (41 months from diagnosis).
  • [MeSH-major] Aminoglycosides / immunology. Aminoglycosides / therapeutic use. Antibodies, Monoclonal / immunology. Antibodies, Monoclonal / therapeutic use. Antigens, CD / immunology. Antigens, Differentiation, Myelomonocytic / immunology. Leukemia, Myeloid, Acute / therapy

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  • (PMID = 20310000.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Aminoglycosides; 0 / Antibodies, Monoclonal; 0 / Antibodies, Monoclonal, Humanized; 0 / Antigens, CD; 0 / Antigens, Differentiation, Myelomonocytic; 0 / CD33 protein, human; 0 / Sialic Acid Binding Ig-like Lectin 3; 0 / gemtuzumab
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26. Ho PA, Zeng R, Alonzo TA, Gerbing RB, Miller KL, Pollard JA, Stirewalt DL, Heerema NA, Raimondi SC, Hirsch B, Franklin JL, Lange B, Meshinchi S: Prevalence and prognostic implications of WT1 mutations in pediatric acute myeloid leukemia (AML): a report from the Children's Oncology Group. Blood; 2010 Aug 05;116(5):702-10
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  • [Title] Prevalence and prognostic implications of WT1 mutations in pediatric acute myeloid leukemia (AML): a report from the Children's Oncology Group.
  • Recent studies of WT1 mutations in acute myeloid leukemia (AML) mostly report an association with unfavorable clinical outcome.
  • We screened 842 patients treated on 3 consecutive pediatric AML trials for WT1 zinc-finger mutations.
  • Patients with or without mutations had similar rates of complete remission after one course of induction chemotherapy.
  • In current risk stratification schemes incorporating cytogenetics and FLT3/ITD status, the presence of WT1 mutations has no independent prognostic significance in predicting outcome in pediatric AML.
  • [MeSH-major] Genes, Wilms Tumor. Leukemia, Myeloid / genetics. Mutation
  • [MeSH-minor] Acute Disease. Adolescent. Child. Child, Preschool. Cohort Studies. DNA Mutational Analysis. DNA, Neoplasm / genetics. Exons / genetics. Female. Humans. Infant. Infant, Newborn. Kaplan-Meier Estimate. Karyotyping. Male. Prevalence. Prognosis. Proportional Hazards Models. Retrospective Studies. Tandem Repeat Sequences / genetics. Treatment Outcome. Young Adult. fms-Like Tyrosine Kinase 3 / genetics

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  • (PMID = 20413658.001).
  • [ISSN] 1528-0020
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Databank-accession-numbers] ClinicalTrials.gov/ NCT00002798/ NCT00070174
  • [Grant] United States / NCI NIH HHS / CA / R01 CA114563; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / R21CA10262-01; United States / NCI NIH HHS / CA / U10 CA98543
  • [Publication-type] Journal Article; Multicenter Study; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / DNA, Neoplasm; EC 2.7.10.1 / FLT3 protein, human; EC 2.7.10.1 / fms-Like Tyrosine Kinase 3
  • [Other-IDs] NLM/ PMC2918327
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27. Mizushima Y, Taki T, Shimada A, Yui Y, Hiraumi Y, Matsubara H, Watanabe M, Watanabe K, Kamitsuji Y, Hayashi Y, Tsukimoto I, Kobayashi R, Horibe K, Tawa A, Nakahata T, Adachi S: Prognostic significance of the BAALC isoform pattern and CEBPA mutations in pediatric acute myeloid leukemia with normal karyotype: a study by the Japanese Childhood AML Cooperative Study Group. Int J Hematol; 2010 Jun;91(5):831-7
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  • [Title] Prognostic significance of the BAALC isoform pattern and CEBPA mutations in pediatric acute myeloid leukemia with normal karyotype: a study by the Japanese Childhood AML Cooperative Study Group.
  • High BAALC (brain and acute leukemia, cytoplasmic) gene expression may indicate an adverse prognosis for adults who have acute myeloid leukemia (AML) and a normal karyotype, but its prognostic significance for pediatric AML cases is unclear.
  • Newly diagnosed AML patients with normal karyotype who were treated by the Japanese Childhood AML Cooperative Treatment Protocol AML 99 were analyzed in terms of their BAALC expression levels (n = 29), BAALC isoforms (n = 29), and CEBPA mutations (n = 49).
  • All four patients have maintained complete remission for at least 5 years.
  • Thus, 1-5-6-8 isoform expression may be associated with an adverse prognosis in pediatric AML with normal karyotype.
  • [MeSH-major] Asian Continental Ancestry Group / genetics. CCAAT-Enhancer-Binding Proteins. Gene Expression Regulation, Leukemic. Leukemia, Myeloid, Acute / diagnosis. Mutation. Neoplasm Proteins

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  • (PMID = 20495894.001).
  • [ISSN] 1865-3774
  • [Journal-full-title] International journal of hematology
  • [ISO-abbreviation] Int. J. Hematol.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / BAALC protein, human; 0 / CCAAT-Enhancer-Binding Proteins; 0 / CEBPA protein, human; 0 / Neoplasm Proteins; 0 / Protein Isoforms
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28. Tomizawa D, Tabuchi K, Kinoshita A, Hanada R, Kigasawa H, Tsukimoto I, Tsuchida M, Tokyo Children's Cancer Study Group: Repetitive cycles of high-dose cytarabine are effective for childhood acute myeloid leukemia: long-term outcome of the children with AML treated on two consecutive trials of Tokyo Children's Cancer Study Group. Pediatr Blood Cancer; 2007 Aug;49(2):127-32
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  • [Title] Repetitive cycles of high-dose cytarabine are effective for childhood acute myeloid leukemia: long-term outcome of the children with AML treated on two consecutive trials of Tokyo Children's Cancer Study Group.
  • BACKGROUND: Various methods of intensive chemotherapy have contributed to an improved survival in pediatric acute myeloid leukemia (AML).
  • We here report the long-term results of the two consecutive trials of Tokyo Children's Cancer Study Group (TCCSG), incorporating repetitive use of high-dose cytarabine (HD-Ara-C) based combination chemotherapy in post-remission phase.
  • PROCEDURE: A total of 216 eligible children with newly diagnosed AML were treated in the two consecutive multi-center trials of TCCSG, M91-13 and M96-14, from August 1991 to September 1998.
  • In M91-13 trial, patients received eight courses of intensive post-remission chemotherapy, including six HD-Ara-C containing courses, after remission-induction therapy.
  • RESULTS: The remission-induction rate was 88.8% and probability of 5-year Overall survival (OS) and event-free survival (EFS) were 62% (56-69% with 95% Confidence intervals (CIs)) and 56% (49-62%), respectively.
  • Among patients without Down syndrome (DS) or acute promyelocytic leukemia (APL), the presence of t(8;21) or inv(16) was a significant good prognostic factor both in the univariate and multivariate analyses.
  • CONCLUSIONS: These results suggest that repetitive use of HD-Ara-C was effective and safe for childhood AML.
  • However, further optimization of AML therapy is required.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid / drug therapy
  • [MeSH-minor] Acute Disease. Adolescent. Antimetabolites, Antineoplastic / administration & dosage. Antimetabolites, Antineoplastic / therapeutic use. Child. Child, Preschool. Combined Modality Therapy. Cytarabine / administration & dosage. Cytarabine / therapeutic use. Disease-Free Survival. Down Syndrome / complications. Doxorubicin / administration & dosage. Doxorubicin / analogs & derivatives. Drug Administration Schedule. Etoposide / administration & dosage. Female. Hematopoietic Stem Cell Transplantation. Humans. Hydrocortisone / administration & dosage. Infant. Infection / etiology. Infection / mortality. Japan / epidemiology. Kaplan-Meier Estimate. Male. Methotrexate / administration & dosage. Mitoxantrone / administration & dosage. Remission Induction. Survival Analysis. Transplantation, Autologous. Transplantation, Homologous. Treatment Outcome. Tretinoin / administration & dosage. Vincristine / administration & dosage


29. Garderet L, Labopin M, Gorin NC, Polge E, Baruchel A, Meloni G, Ortega J, Vossen J, Bunjes D, Leverger G, Blaise D, Ferrant A, Brune M, Dore E, Gadner H, Zintl F, Yaniv I, Dini G, Frassoni F, Acute Leukemia Working Party and Pediatric Working Party of the European Group for Blood and Marrow Transplantation: Hematopoietic stem cell transplantation for de novo acute megakaryocytic leukemia in first complete remission: a retrospective study of the European Group for Blood and Marrow Transplantation (EBMT). Blood; 2005 Jan 1;105(1):405-9
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  • [Title] Hematopoietic stem cell transplantation for de novo acute megakaryocytic leukemia in first complete remission: a retrospective study of the European Group for Blood and Marrow Transplantation (EBMT).
  • Acute megakaryoblastic leukemia (M7 AML) is a highly aggressive disease.
  • We evaluated outcomes in 57 children (11 with Down syndrome) and 69 adults with M7 AML after first complete remission (CR1) following autologous or HLA-identical allogeneic transplantation.
  • Results for autologous transplantation were (children and adults, respectively): engraftment, 90% and 100%; 3-year treatment-related mortality (TRM) rate, 3% and 8%; relapse rate, 45% and 64%; leukemia-free survival (LFS) rate, 52% and 27%; overall survival (OS) rate, 61% and 30%.
  • We conclude that M7 AML patients in CR1 (except children with Down syndrome, who already have better outcomes) can benefit from transplantation.
  • [MeSH-major] Hematopoietic Stem Cell Transplantation. Leukemia, Megakaryoblastic, Acute / pathology. Leukemia, Megakaryoblastic, Acute / surgery

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  • (PMID = 15191953.001).
  • [ISSN] 0006-4971
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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30. de Jonge HJ, Valk PJ, Veeger NJ, ter Elst A, den Boer ML, Cloos J, de Haas V, van den Heuvel-Eibrink MM, Kaspers GJ, Zwaan CM, Kamps WA, Löwenberg B, de Bont ES: High VEGFC expression is associated with unique gene expression profiles and predicts adverse prognosis in pediatric and adult acute myeloid leukemia. Blood; 2010 Sep 9;116(10):1747-54
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  • [Title] High VEGFC expression is associated with unique gene expression profiles and predicts adverse prognosis in pediatric and adult acute myeloid leukemia.
  • High VEGFC mRNA expression of acute myeloid leukemia (AML) blasts is related to increased in vitro and in vivo drug resistance.
  • We studied effect of VEGFC on treatment outcome and investigated gene expression profiles associated with VEGFC using microarray data of 525 adult and 100 pediatric patients with AML.
  • High VEGFC expression appeared strongly associated with reduced complete remission rate (P = .004), reduced overall and event-free survival (OS and EFS) in adult AML (P = .002 and P < .001, respectively).
  • Also, in pediatric AML high VEGFC was related to reduced OS (P = .041).
  • A unique series of differentially expressed genes was identified that distinguished AML with high VEGFC from AML with low VEGFC, that is, 331 up-regulated genes (representative of proliferation, vascular endothelial growth factor receptor activity, signal transduction) and 44 down-regulated genes (eg, related to apoptosis) consistent with a role in enhanced chemoresistance.
  • [MeSH-major] Gene Expression Profiling. Gene Expression Regulation, Leukemic. Leukemia, Myeloid, Acute / genetics. Vascular Endothelial Growth Factor C / genetics


31. Barnard DR, Alonzo TA, Gerbing RB, Lange B, Woods WG, Children's Oncology Group: Comparison of childhood myelodysplastic syndrome, AML FAB M6 or M7, CCG 2891: report from the Children's Oncology Group. Pediatr Blood Cancer; 2007 Jul;49(1):17-22
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  • [Title] Comparison of childhood myelodysplastic syndrome, AML FAB M6 or M7, CCG 2891: report from the Children's Oncology Group.
  • BACKGROUND: Myelodysplastic syndromes (MDS), acute erythroleukemia (FAB M6), and acute megakaryocytic leukemia (FAB M7) have overlapping features.
  • PROCEDURE: Children without Down syndrome or acute promyelocytic leukemia who were newly diagnosed with primary myelodysplastic syndrome or acute myeloid leukemia (AML) M6 or M7 were compared to children with de novo AML M0-M5.
  • RESULTS: The presentation and outcomes of the 132 children diagnosed with MDS (60 children), AML FAB M6 (19 children), or AML FAB M7 (53 children) were similar.
  • Children with AML FAB M7 were diagnosed at a significantly younger age (P = 0.001).
  • All three groups had significantly inferior overall survival (OS) (P < 0.001) and event free survival (P < 0.001) compared with the 748 children diagnosed with AML FAB M0-M5 when assessed from entry on study.
  • However, when assessed from successful completion of induction therapy, the 5-year OS (P = 0.090)(49.1 vs. 56.9%) and disease-free survival (DFS) (P = 0.113)(38.0 vs. 46.3%) therapy were not significantly different from other children with AML.
  • CONCLUSIONS: Childhood AML FAB M6 and AML M7 resemble MDS in presentation, poor induction success rates, and outcomes.
  • [MeSH-major] Leukemia, Erythroblastic, Acute / diagnosis. Leukemia, Megakaryoblastic, Acute / diagnosis. Myelodysplastic Syndromes / diagnosis
  • [MeSH-minor] Acute Disease. Child. Child, Preschool. Diagnosis, Differential. Disease-Free Survival. Female. Humans. Male. Prognosis. Remission Induction. Survival Rate. Treatment Outcome


32. Dłuzniewska A, Balwierz W, Balcerska A, Chybicka A, Kamieńska E, Karolczyk G, Karpińska-Derda I, Krawczuk-Rybak M, Kowalczyk JR, Lewandowska D, Maciejka-Kapuścińska L, Kołtano S, Malinowska I, Matysiak M, Mikołajczyk M, Mizia-Malarz A, Muszyńska-Rosłan K, Niedźwiedzki M, Pohorecka J, Sikorska-Fic B, Sobol G, Sońta-Jakimczyk D, Tomaszewska R, Urasiński T, Wachowiak J, Wieczorek M, Wójcik B, Wysocki M: [Treatment failure in children with acute myelocytic leukemia: over 25-year experience of Polish Pediatric Leukemia/Lymphoma Study Group with four consecutive unified treatment protocols for childhood acute myelocytic leukemia]. Przegl Lek; 2010;67(6):366-70
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  • [Title] [Treatment failure in children with acute myelocytic leukemia: over 25-year experience of Polish Pediatric Leukemia/Lymphoma Study Group with four consecutive unified treatment protocols for childhood acute myelocytic leukemia].
  • Four consecutive intensive unified regimens (BFM-AML-83, PGP-AML 94, PGP-AML 98 AML-BFM 2004 Interim) for acute myelocytic leukemia (AML) have been conducted by the Polish Pediatric Leukemia/Lymphoma Study Group (PPLLSG) since 1983.
  • There were 726 children with AML diagnosed (226, 102, 247 and 151 in the I, II , III and IV periods, respectively), and 603 of them were eligible for evaluation (208, 83, 195 and 117, respectively).
  • Complete remission rates were: 71.4%, 67.5%, 81.4% and 87% in consecutive periods, respectively.
  • Five-year overall survival (OS) and event-free survival (EFS) rates were: 33% and 32% for PGP-AML 83 regimen, 38% and 36% for PGP-AML 94 regimen, and 53% and 46% for PGP-AML 98 regimen, respectively.
  • For AML-BFM Interim 2004 the 3-year OS and EFS were 57% and 57%, respectively.
  • Deaths in remission decreased from 10% in first and second period to 3.5% at present.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Disease-Free Survival. Female. Humans. Infant. Male. Poland / epidemiology. Remission Induction. Survival Rate. Treatment Failure

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  • (PMID = 21344763.001).
  • [ISSN] 0033-2240
  • [Journal-full-title] Przegla̧d lekarski
  • [ISO-abbreviation] Prz. Lek.
  • [Language] pol
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] Poland
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33. Majeed F, Jadko S, Freedman MH, Dror Y: Mutation analysis of SBDS in pediatric acute myeloblastic leukemia. Pediatr Blood Cancer; 2005 Dec;45(7):920-4
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  • [Title] Mutation analysis of SBDS in pediatric acute myeloblastic leukemia.
  • BACKGROUND: Shwachman-Diamond syndrome (SDS) is associated with a high risk of myelodysplasia, acute myeloid leukemia (AML), and chromosome 7 abnormalities.
  • Herein, we studied the role of genetic alterations in SBDS in AML.
  • PROCEDURE: DNA was extracted from marrows of SDS patients with AML, as well as from children with de novo AML.
  • To study whether SBDS heterozygosity confers a risk for MDS/AML, data on family members of SDS patients on the Canadian Inherited Marrow Failure Registry (CIMFR) was analyzed.
  • RESULTS: Of two SDS patients with SDS/AML one was homozygous 258 + 2T > C, and one was compound heterozygous 183-184TA > CT/258 + 2T > C.
  • To determine whether a subset of patients with SDS can present with AML, we analyzed 48 AML samples at remission, but no mutations were identified.
  • To address whether acquired mutated SBDS gene is associated with leukemic transformation in de novo AML, we analyzed 77 AML samples at diagnosis or relapse (4 with -7 and 7q-) for SBDS mutations; no alterations were detected.
  • Also, among the relatives of an SDS patient cohort on the registry no cases of MDS/AML were reported.
  • CONCLUSIONS: Common mutations occurred in our SDS patients who develop AML, and thus, AML is not confined to a rare genetic subgroup of SDS.
  • Newly diagnosed patients with AML are unlikely to have an underlying undiagnosed SDS.
  • Acquired SBDS gene mutations also would appear unlikely to play a mechanistic role in de novo AML, and might not be involved in the pathogenesis of chromosome 7 abnormalities as well.
  • [MeSH-major] Chromosome Aberrations. Chromosomes, Human, Pair 7 / genetics. Exons. Leukemia, Myeloid, Acute / genetics. Point Mutation. Proteins / genetics

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  • [Copyright] 2005 Wiley-Liss, Inc.
  • (PMID = 16007594.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Proteins; 0 / SBDS protein, human
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34. Aplenc R, Alonzo TA, Gerbing RB, Lange BJ, Hurwitz CA, Wells RJ, Bernstein I, Buckley P, Krimmel K, Smith FO, Sievers EL, Arceci RJ, Children's Oncology Group: Safety and efficacy of gemtuzumab ozogamicin in combination with chemotherapy for pediatric acute myeloid leukemia: a report from the Children's Oncology Group. J Clin Oncol; 2008 May 10;26(14):2390-3295
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  • [Title] Safety and efficacy of gemtuzumab ozogamicin in combination with chemotherapy for pediatric acute myeloid leukemia: a report from the Children's Oncology Group.
  • PURPOSE: While gemtuzumab ozogamicin (GTMZ) is commonly used in the treatment of acute myeloid leukemia (AML) in combination with standard chemotherapy agents, the pediatric maximum-tolerated dose (MTD) of GMTZ in combination with chemotherapy has not been determined.
  • Toxicities observed in both treatment regimens were typical of those seen in the relapsed AML setting and consisted primarily of infectious complications.
  • The overall remission response rate (mean +/- SE) was 45% +/- 15% and the 1 year event-free survival and overall survival estimates were 38% +/- 14% and 53% +/- 15%, respectively.
  • CONCLUSION: This trial determined the pediatric MTD for GMTZ with two commonly used AML chemotherapy combinations.
  • Based on these results, an ongoing phase III trial conducted within the Children's Oncology Group is evaluating the effect of GMTZ when added to standard AML therapy.

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  • (PMID = 18467731.001).
  • [ISSN] 1527-7755
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / R01 CA108862; United States / NCI NIH HHS / CA / U10 CA098413; United States / NCI NIH HHS / CA / 1 R01 CA108862; United States / NCI NIH HHS / CA / 1 U10 CA98413-01
  • [Publication-type] Clinical Trial; Comparative Study; Journal Article; Multicenter Study; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Aminoglycosides; 0 / Antibodies, Monoclonal; 0 / Antibodies, Monoclonal, Humanized; 0 / gemtuzumab; 04079A1RDZ / Cytarabine; BZ114NVM5P / Mitoxantrone; EC 3.5.1.1 / Asparaginase
  • [Other-IDs] NLM/ PMC4558626
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35. Kang HJ, Lee JW, Kho SH, Kim MJ, Seo YJ, Kim H, Shin HY, Ahn HS: High transcript level of FLT3 associated with high risk of relapse in pediatric acute myeloid leukemia. J Korean Med Sci; 2010 Jun;25(6):841-5
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  • [Title] High transcript level of FLT3 associated with high risk of relapse in pediatric acute myeloid leukemia.
  • Identification of prognostic factors and risk-based post-remission therapy was proposed to improve the outcomes of acute myeloid leukemia (AML) and a mutation of FLT3 has been reported to be a risk factor, especially for pediatric patients.
  • Recently, FLT3 expression level was implicated to have prognostic significance in adults, but little is known for childhood AML.
  • To define the prognostic significance, transcript level of FLT3 was analyzed in 52 pediatric AML patients.
  • [MeSH-major] Leukemia, Myeloid, Acute / genetics. fms-Like Tyrosine Kinase 3 / genetics

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  • (PMID = 20514303.001).
  • [ISSN] 1598-6357
  • [Journal-full-title] Journal of Korean medical science
  • [ISO-abbreviation] J. Korean Med. Sci.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] Korea (South)
  • [Chemical-registry-number] EC 2.7.10.1 / fms-Like Tyrosine Kinase 3
  • [Other-IDs] NLM/ PMC2877222
  • [Keywords] NOTNLM ; FLT3 / Leukemia, Myeloid, Acute / Pediatric Age / Transcript Level
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36. Hijiya N, Gaynon P, Barry E, Silverman L, Thomson B, Chu R, Cooper T, Kadota R, Rytting M, Steinherz P, Shen V, Jeha S, Abichandani R, Carroll WL: A multi-center phase I study of clofarabine, etoposide and cyclophosphamide in combination in pediatric patients with refractory or relapsed acute leukemia. Leukemia; 2009 Dec;23(12):2259-64
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  • [Title] A multi-center phase I study of clofarabine, etoposide and cyclophosphamide in combination in pediatric patients with refractory or relapsed acute leukemia.
  • This Phase I study of clofarabine with etoposide and cyclophosphamide for children with relapsed/refractory acute lymphoblastic leukemia (ALL) or acute myelogenous leukemia (AML) was conducted to determine the maximum tolerated dose (MTD), dose-limiting toxicities and the recommended phase 2 doses (RP2Ds).
  • A total of 25 patients (20 ALL and 5 AML) were enrolled in five cohorts.
  • Complete remission (CR) was achieved in 10 patients (ALL: nine; AML: one), and CR without platelet recovery in six patients (ALL: two; AML: four) for an overall response rate of 64% (ALL: 55%; AML: 100%).
  • In conclusion, the combination of clofarabine, etoposide and cyclophosphamide was well tolerated and effective in pediatric patients with relapsed/refractory leukemia.
  • [MeSH-major] Adenine Nucleotides / administration & dosage. Arabinonucleosides / administration & dosage. Cyclophosphamide / administration & dosage. Etoposide / administration & dosage. Leukemia / drug therapy. Salvage Therapy / methods
  • [MeSH-minor] Acute Disease. Adolescent. Antineoplastic Combined Chemotherapy Protocols / adverse effects. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Antineoplastic Combined Chemotherapy Protocols / toxicity. Child. Child, Preschool. Drug-Induced Liver Injury. Hematopoietic Stem Cell Transplantation. Humans. Infant. Leukemia, Myeloid, Acute / complications. Leukemia, Myeloid, Acute / drug therapy. Maximum Tolerated Dose. Precursor Cell Lymphoblastic Leukemia-Lymphoma / complications. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy. Remission Induction. Treatment Outcome. Young Adult

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  • (PMID = 19741725.001).
  • [ISSN] 1476-5551
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] eng
  • [Publication-type] Clinical Trial, Phase I; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Adenine Nucleotides; 0 / Arabinonucleosides; 6PLQ3CP4P3 / Etoposide; 762RDY0Y2H / clofarabine; 8N3DW7272P / Cyclophosphamide
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37. Dincaslan HU, Yavuz G, Unal E, Tacyildiz N, Ikinciogullari A, Dogu F, Guloglu D, Yuksek N, Ertem U: Does serum soluble vascular endothelial growth factor levels have different importance in pediatric acute leukemia and malignant lymphoma patients? Pediatr Hematol Oncol; 2010 Oct;27(7):503-16
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  • [Title] Does serum soluble vascular endothelial growth factor levels have different importance in pediatric acute leukemia and malignant lymphoma patients?
  • There are limited data related to childhood hematologic malignancies.
  • The aim of the study was to evaluate soluble VEGF (sVEGF) levels in children with acute leukemia and malignant lymphoma (ML) at diagnosis and in remission.
  • The levels of serum sVEGF were measured by enzyme-linked immunosorbent assay (ELISA) in 20 children with acute leukemia, 33 children with different histopathological subtypes of ML, and 20 healthy controls.
  • The levels of sVEGF at diagnosis (range 2 -1040 pg/mL; median 52 pg/mL) was significantly lower than in remission (range 136 -1960 pg/mL; median 630 pg/mL) in acute myeloid leukemia (AML) group (P = .018).
  • The sVEGF levels at diagnosis (range: 2 -640 pg/mL; median 89 pg/mL) was significantly lower compared to remission values (range: 116 -1960 pg/mL; median 136 pg/mL) in patients with acute lymphoblastic leukemia (ALL) (P = .002).
  • In ML group, including Burkitt's lymphoma (BL), T-cell non-Hodgkin's lymphoma (NHL), and Hodgkin's lymphoma (HL), sVEGF levels at diagnosis were higher than remission levels, but there was no statistically significant difference (P >.05).
  • The authors noticed that sVEGF levels showed distinct behavioral pattern in different childhood malignancies at diagnosis and in remission.
  • In acute leukemia and ML patients, VEGF acts through different pathophysiological mechanisms, in both bone marrow (BM) angiogenesis and lymphoid tissue lymphangiogenesis.
  • [MeSH-major] Hodgkin Disease / blood. Leukemia, Myeloid, Acute / blood. Lymphoma, Non-Hodgkin / blood. Precursor Cell Lymphoblastic Leukemia-Lymphoma / blood. Vascular Endothelial Growth Factors / blood
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Female. Humans. Infant. Male. Remission Induction. Sensitivity and Specificity. Solubility

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  • (PMID = 20677920.001).
  • [ISSN] 1521-0669
  • [Journal-full-title] Pediatric hematology and oncology
  • [ISO-abbreviation] Pediatr Hematol Oncol
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Vascular Endothelial Growth Factors
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38. Sisler IY, Koehler E, Koyama T, Domm JA, Ryan R, Levine JE, Pulsipher MA, Haut PR, Schultz KR, Taylor DS, Frangoul HA: Impact of conditioning regimen in allogeneic hematopoetic stem cell transplantation for children with acute myelogenous leukemia beyond first complete remission: a pediatric blood and marrow transplant consortium (PBMTC) study. Biol Blood Marrow Transplant; 2009 Dec;15(12):1620-7
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  • [Title] Impact of conditioning regimen in allogeneic hematopoetic stem cell transplantation for children with acute myelogenous leukemia beyond first complete remission: a pediatric blood and marrow transplant consortium (PBMTC) study.
  • Total body irradiation (TBI)-based conditioning regimens for pediatric patients with acute myelogenous leukemia (AML) beyond first complete remission (CR1) are controversial.
  • We retrospectively evaluated 151 pediatric patients with AML beyond CR1, comparing outcomes in 90 patients who received a TBI-based conditioning regimen and 61 patients who received a Bu-based conditioning regimen.
  • There were no differences between the 2 groups with respect to age, sex, duration of CR1, time from most recent remission to transplantation, or donor source.
  • Our study provides no evidence of an advantage to using TBI in children with AML beyond CR1.
  • [MeSH-major] Antineoplastic Agents, Alkylating / therapeutic use. Busulfan / therapeutic use. Hematopoietic Stem Cell Transplantation / methods. Leukemia, Myeloid, Acute / therapy. Transplantation Conditioning / methods
  • [MeSH-minor] Adolescent. Adult. Bone Marrow Transplantation / methods. Bone Marrow Transplantation / mortality. Child. Child, Preschool. Cohort Studies. Cord Blood Stem Cell Transplantation / adverse effects. Cord Blood Stem Cell Transplantation / methods. Disease-Free Survival. Female. Humans. Infant. Male. Multivariate Analysis. Remission Induction. Retrospective Studies. Survival Rate. Treatment Outcome. Whole-Body Irradiation. Young Adult

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  • (PMID = 19896086.001).
  • [ISSN] 1523-6536
  • [Journal-full-title] Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
  • [ISO-abbreviation] Biol. Blood Marrow Transplant.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents, Alkylating; G1LN9045DK / Busulfan
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39. Lehrnbecher T, Zimmermann M, Reinhardt D, Dworzak M, Stary J, Creutzig U: Prophylactic human granulocyte colony-stimulating factor after induction therapy in pediatric acute myeloid leukemia. Blood; 2007 Feb 1;109(3):936-43
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  • [Title] Prophylactic human granulocyte colony-stimulating factor after induction therapy in pediatric acute myeloid leukemia.
  • Children with acute myelogenous leukemia (AML) have a high risk of infectious complications that might be reduced by prophylactic granulocyte colony-stimulating factor (G-CSF).
  • However, G-CSF could induce AML blast proliferation.
  • The prospective randomized trial AML-BFM 98 investigated the impact of G-CSF on hematopoetic recovery and infectious complications (primary endpoints) and on outcome (secondary endpoint) in children (aged 0-18 years) with de novo AML.
  • Between 1998 and 2003, 161 children with AML were randomized to receive G-CSF after inductions 1 and 2, whereas 156 patients were assigned to the control group.
  • Since G-CSF does not influence the risk of infectious complications or outcome in children undergoing therapy for AML, one cannot advocate the routine use of G-CSF in this patient group.
  • [MeSH-major] Granulocyte Colony-Stimulating Factor / administration & dosage. Leukemia, Myeloid, Acute / drug therapy
  • [MeSH-minor] Adolescent. Cell Proliferation / drug effects. Child. Child, Preschool. Disease-Free Survival. Hematopoiesis / drug effects. Humans. Infant. Infection / drug therapy. Infection / mortality. Infection Control. Neutropenia. Premedication. Remission Induction. Treatment Outcome

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  • (PMID = 17008536.001).
  • [ISSN] 0006-4971
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Publication-type] Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 143011-72-7 / Granulocyte Colony-Stimulating Factor
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40. Hartwig M, Weigel S, Bernig T, Bader P, Dölken R, Beck J: Maintenance immunotherapy by repetitive low-dose donor lymphocytes infusions in a child with relapse state aml after allogeneic stem cell transplantation. Pediatr Hematol Oncol; 2007 Mar;24(2):137-40
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  • [Title] Maintenance immunotherapy by repetitive low-dose donor lymphocytes infusions in a child with relapse state aml after allogeneic stem cell transplantation.
  • The treatment of a child with a relapsed state acute leukemia after allogeneic stem cell transplantation (allo-SCT) is a challenge.
  • The authors report about a child with an acute myelogenous leukemia (AML), which relapsed after allo-SCT despite immunological intervention.
  • Presently, the child is in continuous complete remission and has a good quality of life.
  • [MeSH-major] Graft vs Host Disease / therapy. Leukemia, Myeloid, Acute / therapy. Lymphocyte Transfusion. Stem Cell Transplantation

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  • (PMID = 17454780.001).
  • [ISSN] 1521-0669
  • [Journal-full-title] Pediatric hematology and oncology
  • [ISO-abbreviation] Pediatr Hematol Oncol
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] England
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41. Johnston DL, Alonzo TA, Gerbing RB, Lange BJ, Woods WG: Risk factors and therapy for isolated central nervous system relapse of pediatric acute myeloid leukemia. J Clin Oncol; 2005 Dec 20;23(36):9172-8
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  • [Title] Risk factors and therapy for isolated central nervous system relapse of pediatric acute myeloid leukemia.
  • PURPOSE: CNS relapse of pediatric acute myeloid leukemia (AML) is an infrequent occurrence.
  • PATIENTS AND METHODS: Records of 886 patients with de novo AML were reviewed, and patients who entered remission at the end of one course of therapy and developed an isolated CNS relapse as their first event were analyzed (n = 690).
  • [MeSH-major] Central Nervous System Neoplasms / drug therapy. Central Nervous System Neoplasms / radiotherapy. Leukemia, Myeloid / pathology
  • [MeSH-minor] Acute Disease. Adolescent. Adult. Child. Child, Preschool. Female. Humans. Infant. Infant, Newborn. Male. Prognosis. Recurrence. Retrospective Studies. Risk Factors. Survival Analysis

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  • (PMID = 16361619.001).
  • [ISSN] 0732-183X
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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42. Arceci RJ, Sande J, Lange B, Shannon K, Franklin J, Hutchinson R, Vik TA, Flowers D, Aplenc R, Berger MS, Sherman ML, Smith FO, Bernstein I, Sievers EL: Safety and efficacy of gemtuzumab ozogamicin in pediatric patients with advanced CD33+ acute myeloid leukemia. Blood; 2005 Aug 15;106(4):1183-8
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  • [Title] Safety and efficacy of gemtuzumab ozogamicin in pediatric patients with advanced CD33+ acute myeloid leukemia.
  • This open-label, dose-escalation study evaluated the safety and efficacy of single-agent gemtuzumab ozogamicin, a humanized anti-CD33 antibody-targeted chemotherapeutic agent, for pediatric patients with multiple relapsed or primary refractory acute myeloid leukemia (AML).
  • Eight of 29 (28%) patients achieved overall remission.
  • Mean multidrug resistance-protein-mediated drug efflux was significantly lower in the leukemic blasts of patients achieving remission (P < .005).
  • Further studies in combination with standard induction therapy for childhood AML are warranted.
  • [MeSH-major] Aminoglycosides / administration & dosage. Aminoglycosides / toxicity. Antibodies, Monoclonal / administration & dosage. Antibodies, Monoclonal / toxicity. Antigens, CD. Antigens, Differentiation, Myelomonocytic. Leukemia, Myeloid / drug therapy. Salvage Therapy / methods
  • [MeSH-minor] Acute Disease. Adolescent. Antibodies, Monoclonal, Humanized. Blast Crisis. Child. Child, Preschool. Drug Resistance. Female. Hematopoietic Stem Cell Transplantation. Hepatic Veno-Occlusive Disease / chemically induced. Humans. Hyperbilirubinemia / chemically induced. Infant. Male. Maximum Tolerated Dose. Remission Induction / methods. Sialic Acid Binding Ig-like Lectin 3

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  • (PMID = 15886328.001).
  • [ISSN] 0006-4971
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Aminoglycosides; 0 / Antibodies, Monoclonal; 0 / Antibodies, Monoclonal, Humanized; 0 / Antigens, CD; 0 / Antigens, Differentiation, Myelomonocytic; 0 / CD33 protein, human; 0 / Sialic Acid Binding Ig-like Lectin 3; 0 / gemtuzumab
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43. Petropoulos D, Worth LL, Mullen CA, Madden R, Mahajan A, Choroszy M, Ha CS, Champlin RC, Chan KW: Total body irradiation, fludarabine, melphalan, and allogeneic hematopoietic stem cell transplantation for advanced pediatric hematologic malignancies. Bone Marrow Transplant; 2006 Mar;37(5):463-7
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  • [Title] Total body irradiation, fludarabine, melphalan, and allogeneic hematopoietic stem cell transplantation for advanced pediatric hematologic malignancies.
  • We evaluated the efficacy and toxicity of adding 9 Gy of total body irradiation (TBI), in three single daily fractions of 3 Gy, to the reduced intensity regimen of fludarabine 30 mg/m2 i.v. x 4 days and melphalan 140 mg/m2 i.v. x 1 day in advanced pediatric hematologic malignancies.
  • Twenty-two acute lymphoblastic leukemia (ALL), six acute myeloid leukemia (AML), and one non-Hodgkin lymphoma patients were transplanted.
  • Of these, 13 were beyond second remission, and five had prior hematopoietic stem cell transplant (HSCT).
  • With a median follow-up of 52 months, seven of 22 ALL, five of six AML, and one of one lymphoma patients are alive and in remission.
  • It was fairly well tolerated in pediatric patients, even for second transplants.

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  • (PMID = 16435013.001).
  • [ISSN] 0268-3369
  • [Journal-full-title] Bone marrow transplantation
  • [ISO-abbreviation] Bone Marrow Transplant.
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] FA2DM6879K / Vidarabine; P2K93U8740 / fludarabine; Q41OR9510P / Melphalan
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44. Wang J, Ouyang J, Zhou R, Chen B, Yang Y: Autologous hematopoietic stem cell transplantation for acute myeloid leukemia in first complete remission: a meta-analysis of randomized trials. Acta Haematol; 2010;124(2):61-71
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  • [Title] Autologous hematopoietic stem cell transplantation for acute myeloid leukemia in first complete remission: a meta-analysis of randomized trials.
  • BACKGROUND: For those patients who are not candidates for allogeneic stem cell transplantation (SCT) or who do not have an HLA-matched donor, it is unclear whether consolidation therapy with autologous SCT results in a survival benefit compared with further intensive post-remission non-myeloablative chemotherapy or no further therapy.
  • METHODS: A meta-analysis evaluating autologous SCT versus further chemotherapy or no treatment for acute myeloid leukemia (AML) in first complete remission (CR1) was completed.
  • Four studies were in pediatric patients and 9 were in adults.
  • For adults, AML in CR1 compared with non-SCT, lower relapse and higher transplantation-related mortality were associated with autologous SCT, a significant disease-free survival benefit of autologous SCT was documented, and there was no difference in overall survival when studies were pooled.
  • For pediatric AML in CR1, there were no differences in relapse, transplantation-related mortality, disease-free survival and overall survival.
  • CONCLUSION: Our results support the conclusion that autologous SCT should not be considered as the first-line post-remission therapy for AML patients in CR1.
  • [MeSH-major] Hematopoietic Stem Cell Transplantation / mortality. Hematopoietic Stem Cell Transplantation / statistics & numerical data. Leukemia, Myeloid, Acute / mortality. Leukemia, Myeloid, Acute / therapy
  • [MeSH-minor] Humans. Randomized Controlled Trials as Topic. Remission Induction. Risk Factors. Secondary Prevention. Transplantation, Autologous


45. Ishaqi MK, Afzal S, Dupuis A, Doyle J, Gassas A: Outcome of allogeneic hematopoietic stem cell transplantation for children with acute myelogenous leukemia in second complete remission: single center experience. Pediatr Transplant; 2009 Dec;13(8):999-1003
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  • [Title] Outcome of allogeneic hematopoietic stem cell transplantation for children with acute myelogenous leukemia in second complete remission: single center experience.
  • We reviewed 26 consecutive patients with AML who were transplanted in second CR2 between 1994 and 2005.
  • Acute grade III-IV and chronic extensive GVHD occurred in eight (30%) and nine (35%) patients, respectively.
  • When entering remission, children with relapsed AML have a reasonable survival with HSCT, but relapse and TRM remain a concern.
  • [MeSH-major] Hematopoietic Stem Cell Transplantation. Leukemia, Myeloid, Acute / therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Female. Humans. Immunosuppressive Agents / administration & dosage. Infant. Male. Remission Induction. Retrospective Studies. Survival Rate. Transplantation Conditioning / methods. Transplantation, Homologous. Treatment Outcome

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  • (PMID = 19032410.001).
  • [ISSN] 1399-3046
  • [Journal-full-title] Pediatric transplantation
  • [ISO-abbreviation] Pediatr Transplant
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Denmark
  • [Chemical-registry-number] 0 / Immunosuppressive Agents
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46. Vettenranta K, EBMT Paediatric Working Party: Current European practice in pediatric myeloablative conditioning. Bone Marrow Transplant; 2008 Jun;41 Suppl 2:S14-7

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  • [Title] Current European practice in pediatric myeloablative conditioning.
  • Myeloablative conditioning continues to be employed in hematopoietic stem cell transplantation among patients with pediatric transplant indications.
  • Fractionated TBI (fTBI) remains, with its considerable anti-leukemic potential, the cornerstone of conditioning in the most common of pediatric indications, ALL in its first, second or subsequent remission despite its well-established long-term sequelae.
  • The feasibility of chemotherapy-only regimens has been established and these regimens widely employed in other pediatric indications, for example, in ALL below the age of 2 years, AML, myelodysplasias or severe aplastic anemia.
  • Conditioning regimens are being modified with data accumulating on the role of, for example, pre-transplant residual disease, advanced HLA-typing or haploidentical transplantations in the pediatric setting.
  • [MeSH-minor] Child. Child, Preschool. Humans. Infant. Leukemia / therapy. Myelodysplastic Syndromes / therapy. Practice Guidelines as Topic

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  • (PMID = 18545236.001).
  • [ISSN] 0268-3369
  • [Journal-full-title] Bone marrow transplantation
  • [ISO-abbreviation] Bone Marrow Transplant.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Myeloablative Agonists
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47. Zhao JN, Qiao ZH, Xu LR, Lu QY, Niu XQ, Zhang P, Wang Z: [Expression of FLT3 internal tandem duplication in pediatric patients with acute myeloid leukemia and its correlation with multidrug resistance]. Zhongguo Shi Yan Xue Ye Xue Za Zhi; 2009 Feb;17(1):23-6
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  • [Title] [Expression of FLT3 internal tandem duplication in pediatric patients with acute myeloid leukemia and its correlation with multidrug resistance].
  • This study was aimed to investigate the expression of FLT3 internal tandem duplication (FLT3-ITD) in pediatric patients with acute myeloid leukemia (AML) and to analyse the clinical features of patients with mutations and the relation of FLT3-ITD with multidrug resistance gene 1 (mdr1).
  • RT-PCR was used to determine the expressions of FIT3-ITD and mdr1 gene in bone marrow samples from 81 new diagnosed pediatric patients with AML, the cytogenetics and immunophenotypes of bone marrow cells were routinely examined.
  • The results indicated that the FLT3-ITDs were detected in 8 out of 81 pediatric patients (9.88%) and all mutations detected were hybrid, while less frequently this mutation was detected in adult patients.
  • Although they were irrelevant with sex and immunophenotypes, the mutations seemed predominant in older pediatric patients.
  • The leukocyte counts and bone marrow blast cell counts in pediatric patients with FLT3-ITD at diagnosis were higher than those in pediatric patients without FLT3-ITD (p = 0.001 and p = 0.041 respectively), but the normal chromosomes were found in most pediatric patients with FLT-ITD.
  • The patients with FLT3-ITD had lower induction remission rate (only 25%), but the patients without FLT3-ITD had higher remission rate (76.1%).
  • According results detected by RT-PCR, the mdr1 gene was found in 27 pediatric patients, but only 3 out of 8 pediatric patients with FLT3-ITD were detected to express both FLT3-ITD and mdr1, which suggests unrelation between FLT3-ITD occurrence and mdr1 expression.
  • It is concluded that the FLT3-ITD is frequent mutation in pediatric patients with AML, the prognosis is worse and the induction remission rate is lower in these patients, but the FLT3-ITD not relates with the mdr1, which suggests that the common MDR modulators may be un effective for therapy of the patients with FLT3-ITD.

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  • (PMID = 19236740.001).
  • [ISSN] 1009-2137
  • [Journal-full-title] Zhongguo shi yan xue ye xue za zhi
  • [ISO-abbreviation] Zhongguo Shi Yan Xue Ye Xue Za Zhi
  • [Language] CHI
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] China
  • [Chemical-registry-number] 0 / ABCB1 protein, human; 0 / P-Glycoprotein; 0 / P-Glycoproteins; EC 2.7.10.1 / FLT3 protein, human; EC 2.7.10.1 / fms-Like Tyrosine Kinase 3
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48. Dluzniewska A, Balwierz W, Armata J, Balcerska A, Chybicka A, Kowalczyk J, Matysiak M, Ochocka M, Radwanska U, Rokicka-Milewska R, Sonta-Jakimczyk D, Wachowiak J, Wysocki M: Twenty years of Polish experience with three consecutive protocols for treatment of childhood acute myelogenous leukemia. Leukemia; 2005 Dec;19(12):2117-24
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  • [Title] Twenty years of Polish experience with three consecutive protocols for treatment of childhood acute myelogenous leukemia.
  • Until 1983, results of treatment of acute myelogenous leukemia (AML) in Poland with different regimens were very poor.
  • In 1983, the Polish Pediatric Leukemia/Lymphoma Study Group introduced a unified treatment protocol--a modified version of BFM-83 protocol.
  • This led to an increase in the curability of AML from 15% to approximately 32%.
  • A new treatment protocol employing idarubicin in place of daunorubicin was introduced in 1998 and produced better initial responses, increase in the number of patients attaining remission after induction therapy and proportional increase of standard-risk patients.
  • Unsatisfactory treatment results in children classified into the high-risk group are principally due to the low remission rate.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Antineoplastic Protocols / standards. Leukemia, Myeloid / therapy
  • [MeSH-minor] Acute Disease. Adolescent. Bone Marrow Transplantation. Cause of Death. Child. Child, Preschool. Cytarabine / administration & dosage. Female. Follow-Up Studies. Humans. Idarubicin / therapeutic use. Infant. Infant, Newborn. Male. Poland. Remission Induction / methods. Survival Analysis. Treatment Outcome

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  • (PMID = 16107894.001).
  • [ISSN] 0887-6924
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article; Multicenter Study
  • [Publication-country] England
  • [Chemical-registry-number] 04079A1RDZ / Cytarabine; ZRP63D75JW / Idarubicin
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49. Al-Shehri A, Al-Seraihy A, Owaidah TM, Belgaumi AF: Megakaryocytic blast crisis at presentation in a pediatric patient with chronic myeloid leukemia. Hematol Oncol Stem Cell Ther; 2010;3(1):42-6
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  • [Title] Megakaryocytic blast crisis at presentation in a pediatric patient with chronic myeloid leukemia.
  • Patients with chronic myeloid leukemia (CML) infrequently present in blast crisis (BC).
  • While most BC are of myeloid origin, megakaryocytic BC is rare, especially at the time of CML diagnosis.
  • We describe the first pediatric patient presenting with megakaryocytic leukemia and having BCR-ABL1 translocation as the single chromosomal abnormality.
  • Clinical features were more suggestive of CML in megakaryocytic blast crisis than Philadelphia chromosome positive de novo AML.
  • The patient was treated with AML-directed chemotherapy and imatinib mesylate followed by umbilical cord blood stem cell transplantation.
  • [MeSH-major] Blast Crisis / pathology. Leukemia, Megakaryoblastic, Acute / pathology. Leukemia, Myelogenous, Chronic, BCR-ABL Positive / pathology. Megakaryocytes / pathology
  • [MeSH-minor] Antineoplastic Agents / therapeutic use. Benzamides. Child. Cord Blood Stem Cell Transplantation. Diagnosis, Differential. Female. Humans. Imatinib Mesylate. Piperazines / therapeutic use. Pyrimidines / therapeutic use. Remission Induction. Treatment Outcome

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  • (PMID = 20231813.001).
  • [ISSN] 1658-3876
  • [Journal-full-title] Hematology/oncology and stem cell therapy
  • [ISO-abbreviation] Hematol Oncol Stem Cell Ther
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] Saudi Arabia
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Benzamides; 0 / Piperazines; 0 / Pyrimidines; 8A1O1M485B / Imatinib Mesylate
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50. Steinbach D, Schramm A, Eggert A, Onda M, Dawczynski K, Rump A, Pastan I, Wittig S, Pfaffendorf N, Voigt A, Zintl F, Gruhn B: Identification of a set of seven genes for the monitoring of minimal residual disease in pediatric acute myeloid leukemia. Clin Cancer Res; 2006 Apr 15;12(8):2434-41
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  • [Title] Identification of a set of seven genes for the monitoring of minimal residual disease in pediatric acute myeloid leukemia.
  • BACKGROUND: Monitoring of minimal residual disease (MRD) has become a strong diagnostic tool in acute lymphoblastic leukemia.
  • In acute myeloid leukemia (AML), there is still a need for more suitable MRD markers.
  • EXPERIMENTAL DESIGN: A stepwise approach which combined genome-wide expression profiling, TaqMan low density arrays, and a TaqMan real-time PCR-based screening was used to identify new markers for the monitoring of MRD in AML.
  • Leukemic cells from 52 children with AML and 145 follow-up samples from 25 patients were analyzed.
  • RESULTS: Seven genes were identified which are vastly overexpressed in many patients with AML compared with healthy bone marrow: CCL23, GAGED2, MSLN, SPAG6, and ST18 as well as the previously described markers WT1 and PRAME.
  • The expression of all genes decreased to normal levels in patients who achieved a continuous complete remission.
  • CONCLUSIONS: This set of genes should allow a sensitive and specific monitoring of MRD in AML.
  • [MeSH-major] Gene Expression Profiling. Leukemia, Myeloid / diagnosis. Neoplasm, Residual / diagnosis
  • [MeSH-minor] Acute Disease. Adolescent. Adult. Antigens, Neoplasm / blood. Antigens, Neoplasm / genetics. Biomarkers, Tumor / blood. Biomarkers, Tumor / genetics. Bone Marrow / metabolism. Chemokines, CC / blood. Chemokines, CC / genetics. Child. Child, Preschool. DNA-Binding Proteins / blood. DNA-Binding Proteins / genetics. Female. GPI-Linked Proteins. Humans. Infant. Infant, Newborn. Male. Membrane Glycoproteins / blood. Membrane Glycoproteins / genetics. Microtubule Proteins / blood. Microtubule Proteins / genetics. Oligonucleotide Array Sequence Analysis / methods. Repressor Proteins. Reverse Transcriptase Polymerase Chain Reaction. WT1 Proteins / blood. WT1 Proteins / genetics

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  • (PMID = 16638849.001).
  • [ISSN] 1078-0432
  • [Journal-full-title] Clinical cancer research : an official journal of the American Association for Cancer Research
  • [ISO-abbreviation] Clin. Cancer Res.
  • [Language] eng
  • [Grant] United States / Intramural NIH HHS / /
  • [Publication-type] Journal Article; Research Support, N.I.H., Intramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antigens, Neoplasm; 0 / Biomarkers, Tumor; 0 / CCL23 protein, human; 0 / Chemokines, CC; 0 / DNA-Binding Proteins; 0 / GPI-Linked Proteins; 0 / Membrane Glycoproteins; 0 / Microtubule Proteins; 0 / PRAME protein, human; 0 / Repressor Proteins; 0 / SPAG6 protein, human; 0 / ST18 protein, human; 0 / WT1 Proteins; 0 / XAGE1A protein, human; 0 / mesothelin
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51. O'Brien MM, Taub JW, Chang MN, Massey GV, Stine KC, Raimondi SC, Becton D, Ravindranath Y, Dahl GV, Children's Oncology Group Study POG 9421: Cardiomyopathy in children with Down syndrome treated for acute myeloid leukemia: a report from the Children's Oncology Group Study POG 9421. J Clin Oncol; 2008 Jan 20;26(3):414-20
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  • [Title] Cardiomyopathy in children with Down syndrome treated for acute myeloid leukemia: a report from the Children's Oncology Group Study POG 9421.
  • PURPOSE: To determine the outcomes, with particular attention to toxicity, of children with Down syndrome (DS) and acute myeloid leukemia (AML) treated on Pediatric Oncology Group (POG) protocol 9421.
  • PATIENTS AND METHODS: Children with DS and newly diagnosed AML (n = 57) were prospectively enrolled onto the standard-therapy arm of POG 9421 and were administered five cycles of chemotherapy, which included daunorubicin 135 mg/m(2) and mitoxantrone 80 mg/m(2).
  • Outcomes and toxicity were evaluated prospectively and were compared with the non-DS-AML cohort (n = 565).
  • RESULTS: In the DS-AML group, 54 patients (94.7%) entered remission.
  • Of the 54 who entered remission, three relapsed and six died as a result of other causes.
  • The remission induction rate was similar in the non-DS-French-American-British (FAB) M7 (91.7%) and non-DS-non-M7 (89.3%) groups.
  • The 5-year overall survival was significantly better in the DS-AML group (78.6%) than in the non-DS-M7 (36.3%) or the non-DS-non-M7 (51.8%) groups (P < .001).
  • Symptomatic cardiomyopathy developed in 10 patients (17.5%) with DS-AML during or soon after completion of treatment; three died as a result of congestive heart failure.
  • CONCLUSION: The POG 9421 treatment regimen was highly effective in both remission induction and disease-free survival for patients with DS-AML.

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  • (PMID = 18202418.001).
  • [ISSN] 1527-7755
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / R01 CA090916; United States / NCI NIH HHS / CA / R01 CA90916
  • [Publication-type] Journal Article; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] BZ114NVM5P / Mitoxantrone; ZS7284E0ZP / Daunorubicin
  • [Other-IDs] NLM/ NIHMS515321; NLM/ PMC3897300
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52. Huang LB, Guan XQ, Zhang YC, Zhang XL, Ke ZY, Luo XQ: Current status of diagnosis and prognosis of infant acute leukemia in China. Pediatr Blood Cancer; 2009 Dec;53(6):973-7
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  • [Title] Current status of diagnosis and prognosis of infant acute leukemia in China.
  • OBJECTIVE: Treatment and outcome of infant acute leukemia (IAL) in developed countries have been well documented.
  • METHODS: Five hundred ninety seven pediatric patients were diagnosed with acute leukemia in our hospital between January 1997 and June 2008, of which 19 were younger than 12 months.
  • RESULTS: Of the 19 cases, 14 had acute lymphoblastic leukemia (ALL) and 5 had acute myeloid leukemia (AML) based on FAB classification.
  • Two infants with immunophenotypic AML who abandoned treatment achieved spontaneous remission without chemotherapy within 2 and 4 months respectively.
  • Combining our data with those from Chinese literature, less than one third of the infants had immunophenotypic and genetic verification of leukemia and 29% (18/63) of them received treatment.
  • [MeSH-major] Leukemia / diagnosis
  • [MeSH-minor] Acute Disease. China / epidemiology. Female. Humans. Infant. Leukemia, Myeloid, Acute / diagnosis. Leukemia, Myeloid, Acute / genetics. Male. Precursor Cell Lymphoblastic Leukemia-Lymphoma / diagnosis. Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics. Prognosis. Treatment Outcome

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  • (PMID = 19588516.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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53. Takeda M, Yamaguchi S, Eguchi K, Kajiume T, Nishimura S, Kobayashi M, Kurisu K: Spinal epidural granulocytic sarcoma in a child precedent to clinical manifestation of acute myeloid lymphoma: case report. Neurol Med Chir (Tokyo); 2009 May;49(5):221-4
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  • [Title] Spinal epidural granulocytic sarcoma in a child precedent to clinical manifestation of acute myeloid lymphoma: case report.
  • A 13-year-old boy presented with an epidural thoracic granulocytic sarcoma manifesting as rapidly progressive paraplegia preceding clinical manifestation of acute myeloid leukemia (AML).
  • The correct diagnosis of epidural granulocytic sarcoma and AML was established based on cell-surface markers and a chromosomal study of the bone marrow cells.
  • A combination of chemotherapy and bone marrow transfusion achieved complete remission of leukemia.
  • No evidence of AML has emerged over the 18-month follow-up period.
  • Granulocytic sarcoma should be considered in the differential diagnosis of an epidural mass in pediatric patients with or without acute leukemia.
  • [MeSH-major] Epidural Neoplasms / surgery. Leukemia, Myeloid, Acute / diagnosis. Sarcoma, Myeloid / surgery. Spinal Neoplasms / surgery
  • [MeSH-minor] Adolescent. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Bone Marrow Transplantation. Combined Modality Therapy. Exophthalmos / etiology. Humans. Laminectomy. Magnetic Resonance Imaging. Male. Methylprednisolone / therapeutic use. Orbit / pathology. Paraplegia / etiology. Remission Induction. Temporal Lobe / pathology. Thoracic Vertebrae / surgery

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  • (PMID = 19465795.001).
  • [ISSN] 1349-8029
  • [Journal-full-title] Neurologia medico-chirurgica
  • [ISO-abbreviation] Neurol. Med. Chir. (Tokyo)
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] Japan
  • [Chemical-registry-number] X4W7ZR7023 / Methylprednisolone
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54. Gassas A, Ishaqi MK, Afzal S, Finkelstein-Shechter T, Dupuis A, Doyle J: A comparison of the outcomes of children with acute myelogenous leukemia in either first or second complete remission (CR1 vs CR2) following allogeneic hematopoietic stem cell transplantation at a single transplant center. Bone Marrow Transplant; 2008 Jun;41(11):941-5
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  • [Title] A comparison of the outcomes of children with acute myelogenous leukemia in either first or second complete remission (CR1 vs CR2) following allogeneic hematopoietic stem cell transplantation at a single transplant center.
  • We reviewed 70 consecutive children with AML who received hematopoietic stem cell transplantation (HSCT) in our institution between 1994 and 2005.
  • Expectedly, there was a significant increase in acute GVHD incidence in CR2 patients (40 vs 25% for grades I-II and 30 vs 10% for grades III-IV; P=0.02) and a significant increase in transplant-related mortality (38 vs 11%; P=0.01).
  • Children with relapsed AML who achieve and maintain remission until HSCT, have a reasonable survival, but the outcome of children receiving HSCT in CR1 remains superior.
  • [MeSH-major] Hematopoietic Stem Cell Transplantation. Leukemia, Myeloid, Acute / therapy. Neoplasm Recurrence, Local / therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Disease-Free Survival. Female. Hospitals, Pediatric. Humans. Infant. Male. Remission Induction. Retrospective Studies. Transplantation Conditioning. Transplantation, Homologous

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  • (PMID = 18264141.001).
  • [ISSN] 0268-3369
  • [Journal-full-title] Bone marrow transplantation
  • [ISO-abbreviation] Bone Marrow Transplant.
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article
  • [Publication-country] England
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55. Walter RB, Alonzo TA, Gerbing RB, Ho PA, Smith FO, Raimondi SC, Hirsch BA, Gamis AS, Franklin JL, Hurwitz CA, Loken MR, Meshinchi S: High expression of the very late antigen-4 integrin independently predicts reduced risk of relapse and improved outcome in pediatric acute myeloid leukemia: a report from the children's oncology group. J Clin Oncol; 2010 Jun 10;28(17):2831-8
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  • [Title] High expression of the very late antigen-4 integrin independently predicts reduced risk of relapse and improved outcome in pediatric acute myeloid leukemia: a report from the children's oncology group.
  • PURPOSE: To evaluate the prognostic significance of the integrin cell adhesion molecule very late antigen-4 (VLA-4) in acute myeloid leukemia (AML).
  • In low- and high-expression groups, rates of remission (89% v 80%, respectively; P = .137) and minimal residual disease (29% v 25%, respectively; P = .700) were similar.
  • Subgroup analyses indicated that the prognostic role of VLA-4 expression was most prominent in patients with standard-risk AML, in whom low VLA-4 expression was associated with inferior DFS (34% +/- 16% v 69% +/- 14% for high expression; P = .011) and higher RR (61% +/- 16% v 26% +/- 14% for high expression; P = .009).
  • CONCLUSION: High VLA-4 expression is associated with better clinical outcome in pediatric AML and is an independent predictor of relapse that may refine our abilities to stratify patients without identifiable cytogenetic or molecular risk factors.

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  • (PMID = 20421533.001).
  • [ISSN] 1527-7755
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / U10 CA098413; United States / NCI NIH HHS / CA / U24 CA114766; United States / NCI NIH HHS / CA / K23 CA137161; United States / NCI NIH HHS / CA / R01 CA114563; United States / NCI NIH HHS / CA / U10 CA098543
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  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Integrin alpha4beta1
  • [Other-IDs] NLM/ PMC2903318
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56. Liang DC, Chan TT, Lin KH, Lin DT, Lu MY, Chen SH, Liu HC, Lin MT, Lee MT, Shu SG, Chang TK, Chen JS, Hsiao CC, Hung IJ, Hsieh YL, Chen RL, Cheng SN, Chang WH, Lee CH, Lin KS: Improved treatment results for childhood acute myeloid leukemia in Taiwan. Leukemia; 2006 Jan;20(1):136-41
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  • [Title] Improved treatment results for childhood acute myeloid leukemia in Taiwan.
  • To improve treatment results for children with de novo acute myeloid leukemia (AML), we introduced a novel protocol, Taiwan Pediatric Oncology Group-AML-97A, for AML other than acute promyelocytic leukemia (APL), for which modified conventional protocols were used.
  • From January 1, 1997, to December 31, 2002, 141 children younger than 17 years old with de novo AML were enrolled.
  • In total, 117 patients with non-APL AML were treated with induction therapy of idarubicin and cytarabine (Ara-C), postremission therapy with high-dose Ara-C - containing regimens for four monthly courses, and moderate-dose therapy with idarubicin and Ara-C for four monthly courses.
  • Stem cell transplantation was performed in 29 patients in first remission with a similar outcome as chemotherapy alone.
  • The remission rate in the AML-97A study was 90%, the 5-year survival 51 +/- 5.3% (s.e.) and the 5-year event-free survival 50 +/- 4.8%; for APL, these were 100%, 86 +/- 7.0, and 75 +/- 9.8%.
  • The AML-97A regimen was well tolerated.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid, Acute / therapy. Leukemia, Promyelocytic, Acute / therapy. Stem Cell Transplantation
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Disease-Free Survival. Female. Follow-Up Studies. Humans. Infant. Infant, Newborn. Male. Remission Induction. Taiwan. Treatment Outcome

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  • (PMID = 16281075.001).
  • [ISSN] 0887-6924
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
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57. Larson RA: Micro-RNAs and copy number changes: new levels of gene regulation in acute myeloid leukemia. Chem Biol Interact; 2010 Mar 19;184(1-2):21-5
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  • [Title] Micro-RNAs and copy number changes: new levels of gene regulation in acute myeloid leukemia.
  • Together with epigenetic alterations, these result in dysplasia, clonal expansion, and ultimately myeloid leukemia.
  • Combinations of lesions are required to induce overt leukemia.
  • Recent studies have shown that cytogenetically normal (CN-) AML is quite heterogeneous at the molecular level.
  • Patients with CN-AML harboring mutations in NPM1, FLT3, CEBPA, WT1 or expressing high levels of BAALC, ERG, or MN1 have distinctly different clinical outcomes.
  • NPM1 mutations are independently associated with higher remission rates and longer disease-free and overall survival in AML.
  • Fewer CNAs have been detected in AML than in pediatric ALL.
  • Distinctive genome-wide miR expression profiles have been associated with different subsets of AML.
  • A miR signature that is associated with clinical outcome in patients with high-risk molecular features of AML (those who have FLT3-ITD or wild-type NPM1) has been reported.
  • This subgroup constitutes approximately 65% of patients with CN-AML and one-third of all patients with AML <60 years old.
  • Down-regulation of the miR-181 family contributes to an aggressive leukemia phenotype through mechanisms associated with the activation of pathways of innate immunity mediated by toll-like receptors and interleukin-1beta.

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  • [Copyright] Copyright (c) 2009 Elsevier Ireland Ltd. All rights reserved.
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  • (PMID = 19822134.001).
  • [ISSN] 1872-7786
  • [Journal-full-title] Chemico-biological interactions
  • [ISO-abbreviation] Chem. Biol. Interact.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / CA40046; United States / NCI NIH HHS / CA / P01 CA040046; United States / NCI NIH HHS / CA / CA014599-34; United States / NCI NIH HHS / CA / P30 CA014599; United States / NCI NIH HHS / CA / U10 CA031946; United States / NCI NIH HHS / CA / U10 CA031946-28; United States / NCI NIH HHS / CA / P30 CA014599-34; United States / NCI NIH HHS / CA / P01 CA040046-22; United States / NCI NIH HHS / CA / CA031946-28; United States / NCI NIH HHS / CA / CA040046-22; United States / NCI NIH HHS / CA / CA14599; United States / NCI NIH HHS / CA / CA31946
  • [Publication-type] Journal Article; Research Support, N.I.H., Extramural
  • [Publication-country] Ireland
  • [Chemical-registry-number] 0 / MicroRNAs; 0 / Nuclear Proteins; 117896-08-9 / nucleophosmin
  • [Other-IDs] NLM/ NIHMS156571; NLM/ PMC2846194
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58. Pigazzi M, Ricotti E, Germano G, Faggian D, Aricò M, Basso G: cAMP response element binding protein (CREB) overexpression CREB has been described as critical for leukemia progression. Haematologica; 2007 Oct;92(10):1435-7
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  • [Title] cAMP response element binding protein (CREB) overexpression CREB has been described as critical for leukemia progression.
  • CREB has been described as critical for leukemia progress.
  • We investigated CREB expression in ALL and AML pediatric patients.
  • CREB protein was significantly high (p<0.001) at diagnosis but not during remission.
  • This study underlines the role of CREB in leukemia and suggests new insights into the transformation process.
  • [MeSH-major] Cyclic AMP Response Element-Binding Protein / metabolism. Leukemia / metabolism. Leukemia / pathology

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  • (PMID = 18024382.001).
  • [ISSN] 1592-8721
  • [Journal-full-title] Haematologica
  • [ISO-abbreviation] Haematologica
  • [Language] eng
  • [Publication-type] Letter; Research Support, Non-U.S. Gov't
  • [Publication-country] Italy
  • [Chemical-registry-number] 0 / Cyclic AMP Response Element-Binding Protein
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59. Hiçsönmez G: The effect of steroid on myeloid leukemic cells: the potential of short-course high-dose methylprednisolone treatment in inducing differentiation, apoptosis and in stimulating myelopoiesis. Leuk Res; 2006 Jan;30(1):60-8
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  • [Title] The effect of steroid on myeloid leukemic cells: the potential of short-course high-dose methylprednisolone treatment in inducing differentiation, apoptosis and in stimulating myelopoiesis.
  • Several in vitro studies have shown that dexamethasone (Dex) and prednisolone can induce differentiation of some mouse and human myeloid leukemic cells to macrophages and granulocytes.
  • Based on in vitro experiments, we have shown that short-course (3-7 days) high-dose methylprednisolone (HDMP) (20-30 mg/kg/day) treatment can induce differentiation of myeloid leukemic cells in vivo in children with different subtypes of acute myeloblastic leukemia (AML) (AML-M1, -M2, -M3, -M4, -M7).
  • We have also shown that induction of apoptosis of myeloid leukemic cells with or without differentiation is possible by short-course HDMP treatment.
  • Addition of HDMP to mild cytotoxic chemotherapy (low-dose cytosine arabinoside (LD-Ara-c), weekly mitoxantrone and Ara-c or 6-thioguanine) increased the remission rate (87-89%) and improved the outcome of AML children.
  • We believe that the results of our 17-year clinical experience will provide important benefits to AML patients.
  • [MeSH-major] Anti-Inflammatory Agents / pharmacology. Apoptosis / drug effects. Cell Differentiation / drug effects. Leukemia, Myeloid, Acute / metabolism. Methylprednisolone / pharmacology. Myelopoiesis / drug effects

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  • (PMID = 15979702.001).
  • [ISSN] 0145-2126
  • [Journal-full-title] Leukemia research
  • [ISO-abbreviation] Leuk. Res.
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Anti-Inflammatory Agents; 0 / Antigens, CD34; X4W7ZR7023 / Methylprednisolone
  • [Number-of-references] 111
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60. Kang HJ, Hong SH, Kim IH, Park BK, Han KS, Cho HI, Shin HY, Ahn HS: Prognostic significance of FLT3 mutations in pediatric non-promyelocytic acute myeloid leukemia. Leuk Res; 2005 Jun;29(6):617-23
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  • [Title] Prognostic significance of FLT3 mutations in pediatric non-promyelocytic acute myeloid leukemia.
  • This is the first study of FLT3 mutations in pediatric non-promyelocytic AML patients that received the same treatment scheme in single institute.
  • Patients with FLT3/ITD relapsed early after complete remission even after receiving bone marrow transplantation from a matched related donor with little BuCy conditioning.
  • New therapeutic scheme such as stem cell transplantation with more intensive conditioning just after complete remission could be applied in pediatric non-promyelocytic AML patients with the FLT3/ITD mutation.
  • [MeSH-major] Leukemia, Myeloid, Acute / genetics. Proto-Oncogene Proteins / genetics. Receptor Protein-Tyrosine Kinases / genetics

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  • (PMID = 15863200.001).
  • [ISSN] 0145-2126
  • [Journal-full-title] Leukemia research
  • [ISO-abbreviation] Leuk. Res.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Proto-Oncogene Proteins; EC 2.7.10.1 / FLT3 protein, human; EC 2.7.10.1 / Receptor Protein-Tyrosine Kinases; EC 2.7.10.1 / fms-Like Tyrosine Kinase 3
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61. Langebrake C, Klusmann JH, Wortmann K, Kolar M, Puhlmann U, Reinhardt D: Concomitant aberrant overexpression of RUNX1 and NCAM in regenerating bone marrow of myeloid leukemia of Down's syndrome. Haematologica; 2006 Nov;91(11):1473-80
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  • [Title] Concomitant aberrant overexpression of RUNX1 and NCAM in regenerating bone marrow of myeloid leukemia of Down's syndrome.
  • BACKGROUND AND OBJECTIVES: Myeloid leukemia of Down's syndrome (ML-DS) has characteristic biological features (e.g. expression of the truncated GATA1s), which are different from those of non-DS childhood acute myeloid leukemias (AML).
  • DESIGN AND METHODS: We analyzed 134 bone marrow specimens from 64 children with ML-DS and non-DS AML during chemotherapy and 7 specimens from DS children with- out leukemia,who did not receive any chemotherapy,The specimens were analyzed by multiparameter flow cytometry and quantitative reverse transcriptase polymerase chain reaction for transcription factors involved in hematopoiesis.
  • RESULTS: Samples taken from children with ML-DS in complete remission during chemotherapy aberrantly expressed CD56 (NCAM) at the surface of monocytic and granulocytic cells.
  • Compared to non-DS AML cases,children with ML-DS had a statistically significant higher proportion of CD56+ cells in the CD33+ fraction: 71%+/-6% vs. 4%+/-1% (p<0.00001).
  • INTERPRETATION AND CONCLUSIONS: The combined overexpression of RUNX1 and NCAM during stress hematopoiesis in children with DS might be a key factor in the development of overt leukemia and/or in the growth advantage of the malignant GATA1s clone in ML- DS.
  • [MeSH-major] Bone Marrow Cells / metabolism. Core Binding Factor Alpha 2 Subunit / biosynthesis. Down Syndrome / metabolism. Leukemia, Myeloid / metabolism. Neural Cell Adhesion Molecules / biosynthesis

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  • (PMID = 17043020.001).
  • [ISSN] 1592-8721
  • [Journal-full-title] Haematologica
  • [ISO-abbreviation] Haematologica
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] Italy
  • [Chemical-registry-number] 0 / Core Binding Factor Alpha 2 Subunit; 0 / Neural Cell Adhesion Molecules; 0 / RUNX1 protein, human
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62. Tirado CA, Valdez F, Klesse L, Karandikar NJ, Uddin N, Arbini A, Fustino N, Collins R, Patel S, Smart RL, Garcia R, Doolittle J, Chen W: Acute myeloid leukemia with inv(16) with CBFB-MYH11, 3'CBFB deletion, variant t(9;22) with BCR-ABL1, and del(7)(q22q32) in a pediatric patient: case report and literature review. Cancer Genet Cytogenet; 2010 Jul 1;200(1):54-9
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  • [Title] Acute myeloid leukemia with inv(16) with CBFB-MYH11, 3'CBFB deletion, variant t(9;22) with BCR-ABL1, and del(7)(q22q32) in a pediatric patient: case report and literature review.
  • Coexistence of inv(16) and t(9;22) is a rare chromosomal aberration, one that has been described in chronic myelogenous leukemia (CML), mainly in myeloid blast crisis, and de novo acute myeloid leukemia (AML).
  • Approximately 14 cases have been reported, including only 1 pediatric case.
  • Here we present the case of a 13-year-old boy with a new diagnosis of AML with some features of monocytic differentiation.
  • The patient was treated with standard AML chemotherapy plus gemtuzumab as part of a clinical trial.
  • At 10-months follow-up, he was in remission.
  • To the best of our knowledge, this is the first description of a pediatric case of de novo AML with a stemline showing inv(16) along with 3'CBFB deletion, an abnormal clone revealing in addition a del(7)(q22q32), and another clone with a t(9;22;19)(q34;q11.2;p13.1) as an additional abnormality.
  • [MeSH-major] Chromosome Deletion. Chromosome Inversion. Chromosomes, Human, Pair 16. Chromosomes, Human, Pair 22. Chromosomes, Human, Pair 7. Chromosomes, Human, Pair 9. Fusion Proteins, bcr-abl / genetics. Leukemia, Myeloid, Acute / genetics. Oncogene Proteins, Fusion / genetics. Translocation, Genetic

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  • [Copyright] 2010 Elsevier Inc. All rights reserved.
  • (PMID = 20513535.001).
  • [ISSN] 1873-4456
  • [Journal-full-title] Cancer genetics and cytogenetics
  • [ISO-abbreviation] Cancer Genet. Cytogenet.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article; Review
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / CBFbeta-MYH11 fusion protein; 0 / Oncogene Proteins, Fusion; EC 2.7.10.2 / Fusion Proteins, bcr-abl
  • [Number-of-references] 16
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63. Kawamura M, Kaku H, Ito T, Funata N, Taki T, Shimada A, Hayashi Y: FLT3-internal tandem duplication in a pediatric patient with t(8;21) acute myeloid leukemia. Cancer Genet Cytogenet; 2010 Dec;203(2):292-6
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  • [Title] FLT3-internal tandem duplication in a pediatric patient with t(8;21) acute myeloid leukemia.
  • Patients diagnosed with t(8;21)-acute myeloid leukemia (AML) are currently considered to have good prognoses, but about half of these patients relapse.
  • FLT3-internal tandem duplication (ITD) is generally thought to be strongly associated with poor prognosis in AML, but is rarely reported in patients with t(8;21)-AML.
  • Expression of the neural cell-adhesion molecule (CD56) is also associated with a significantly shorter complete remission duration and survival in patients with t(8;21)-AML.
  • Patients with t(8;21)-AML expressing CD56 have been reported to exhibit a higher incidence of granulocytic sarcoma (GS), and t(8;21)-AML with GS results in a less favorable prognosis than AML with this translocation alone.
  • Here, we report on a 15-year-old girl with t(8;21)-AML having both CD56 expression and FLT3-ITD.
  • This patient underwent unrelated donor bone marrow transplantation and achieved complete remission, but thereafter presented with obstructive jaundice caused by GS compression of the common bile duct without bone marrow invasion at relapse.
  • For earlier detection of relapse, we suggest that it would be useful to examine existence of GS in CD56-positive t(8;21)-AML patients at diagnosis and hematologic remission.
  • Even though t(8;21)-AML is less likely to co-occur with FLT3-ITD in pediatric patients, this report suggests that prognostic factors, including FLT3 and KIT genes and the surface marker CD56, should be analyzed in these patients.
  • [MeSH-major] Chromosomes, Human, Pair 21. Chromosomes, Human, Pair 8. Gene Duplication. Leukemia, Myeloid, Acute / genetics. Translocation, Genetic
  • [MeSH-minor] Adolescent. Adult. Aged. Antigens, CD56 / biosynthesis. Fatal Outcome. Female. Humans. Jaundice, Obstructive / mortality. Jaundice, Obstructive / pathology. Male. Middle Aged. Proto-Oncogene Proteins c-kit / metabolism. Recurrence. Remission Induction

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  • [Copyright] Copyright © 2010 Elsevier Inc. All rights reserved.
  • (PMID = 21156247.001).
  • [ISSN] 1873-4456
  • [Journal-full-title] Cancer genetics and cytogenetics
  • [ISO-abbreviation] Cancer Genet. Cytogenet.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antigens, CD56; EC 2.7.10.1 / Proto-Oncogene Proteins c-kit
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64. Rheingold SR: Acute myeloid leukemia in a child with hereditary thrombocytopenia. Pediatr Blood Cancer; 2007 Jan;48(1):105-7
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  • [Title] Acute myeloid leukemia in a child with hereditary thrombocytopenia.
  • A child with a known diagnosis of an autosomal dominant macrothrombocytopenia, Fechtner Syndrome, developed acute myeloid leukemia (AML).
  • MYH9 has never been associated with the development of acute leukemia, but MYH11 is disrupted in the M4 eosinophilia sub-type of AML (inv16).
  • The patients leukemic blasts did carry the common t(8;21) which yields an AML1-ETO fusion protein that inhibits AML-1.
  • Despite his thrombocytopenia, the patient successfully completed intensive bone marrow cytoreduction without significant bleeding complications and is now in remission for over 3 years.
  • [MeSH-major] Blood Coagulation Disorders, Inherited / genetics. Chromosome Disorders / genetics. Leukemia, Myeloid, Acute / genetics. Thrombocytopenia / genetics
  • [MeSH-minor] Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Child, Preschool. Chromosomes, Human, Pair 21 / genetics. Chromosomes, Human, Pair 8 / genetics. Core Binding Factor Alpha 2 Subunit / genetics. Humans. Male. Molecular Motor Proteins / genetics. Myosin Heavy Chains / genetics. Oncogene Proteins, Fusion / genetics. Remission Induction. Translocation, Genetic

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  • [Copyright] (c) 2006 Wiley-Liss, Inc.
  • (PMID = 16276527.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / AML1-ETO fusion protein, human; 0 / Core Binding Factor Alpha 2 Subunit; 0 / MYH9 protein, human; 0 / Molecular Motor Proteins; 0 / Oncogene Proteins, Fusion; EC 3.6.4.1 / Myosin Heavy Chains
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65. Winter SS, Holdsworth MT, Devidas M, Raisch DW, Chauvenet A, Ravindranath Y, Ducore JM, Amylon MD: Antimetabolite-based therapy in childhood T-cell acute lymphoblastic leukemia: a report of POG study 9296. Pediatr Blood Cancer; 2006 Feb;46(2):179-86
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  • [Title] Antimetabolite-based therapy in childhood T-cell acute lymphoblastic leukemia: a report of POG study 9296.
  • PURPOSE: A previous Pediatric Oncology Group (POG) study showed high incidence of secondary acute myelogenous leukemia (AML) in children treated for T-cell acute lymphoblastic leukemia (T-ALL) or higher-stage lymphoblastic lymphoma.
  • Forty-two of 45 patients achieved complete remission (CR), and 27 completed the therapy in continuous CR.
  • One patient taken off study for asparaginase toxicity was treated with multiagent therapy that contained teniposide, and died from secondary myelodysplasia (sMDS)/AML.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy
  • [MeSH-minor] Adolescent. Anthracyclines / administration & dosage. Antimetabolites, Antineoplastic / administration & dosage. Antineoplastic Agents, Alkylating / administration & dosage. Asparaginase / administration & dosage. Asparaginase / adverse effects. Child. Child, Preschool. Disease-Free Survival. Drug Hypersensitivity / etiology. Female. Follow-Up Studies. Humans. Lymphoma, Non-Hodgkin / drug therapy. Lymphoma, Non-Hodgkin / mortality. Male. Pilot Projects. Remission Induction. Sepsis / etiology. Sepsis / mortality

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  • (PMID = 16007607.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / 5 U10 CA5312; United States / NCI NIH HHS / CA / CA29139
  • [Publication-type] Clinical Trial; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Anthracyclines; 0 / Antimetabolites, Antineoplastic; 0 / Antineoplastic Agents, Alkylating; EC 3.5.1.1 / Asparaginase
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66. Jeha S, Razzouk B, Rytting M, Rheingold S, Albano E, Kadota R, Luchtman-Jones L, Bomgaars L, Gaynon P, Goldman S, Ritchey K, Arceci R, Altman A, Stine K, Steinherz L, Steinherz P: Phase II study of clofarabine in pediatric patients with refractory or relapsed acute myeloid leukemia. J Clin Oncol; 2009 Sep 10;27(26):4392-7
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  • [Title] Phase II study of clofarabine in pediatric patients with refractory or relapsed acute myeloid leukemia.
  • PURPOSE: To determine the efficacy and safety of clofarabine in pediatric patients with refractory or relapsed acute myeloid leukemia (AML).
  • PATIENTS AND METHODS: A phase II, open-label, multicenter study was conducted with single-agent clofarabine in pediatric patients with refractory or relapsed AML.
  • Clofarabine was administered intravenously over 2 hours at the pediatric maximum-tolerated dose (MTD) of 52 mg/m(2) daily for 5 consecutive days.
  • CONCLUSION: Clofarabine is active in pediatric patients with multiply relapsed or refractory AML.
  • [MeSH-major] Adenine Nucleotides / therapeutic use. Arabinonucleosides / therapeutic use. Leukemia, Myeloid / drug therapy
  • [MeSH-minor] Acute Disease. Adolescent. Antineoplastic Agents / adverse effects. Antineoplastic Agents / therapeutic use. Child. Child, Preschool. Drug Resistance, Neoplasm. Female. Fever / etiology. Hematopoietic Stem Cell Transplantation / adverse effects. Hematopoietic Stem Cell Transplantation / methods. Humans. Male. Nausea / etiology. Neutropenia / etiology. Recurrence. Remission Induction. Treatment Outcome. Young Adult

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  • (PMID = 19652076.001).
  • [ISSN] 1527-7755
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / P30 CA021765; United States / NCI NIH HHS / CA / CA21765
  • [Publication-type] Clinical Trial, Phase II; Journal Article; Multicenter Study; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Adenine Nucleotides; 0 / Antineoplastic Agents; 0 / Arabinonucleosides; 762RDY0Y2H / clofarabine
  • [Other-IDs] NLM/ PMC2744276
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67. Lin CH, Wu KH, Lin WC, Tsai JD, Peng CT, Chen AC: Granulocytic sarcoma of the colon in a child with acute myeloid leukemia presenting as hematochezia. J Pediatr Hematol Oncol; 2008 Dec;30(12):981-3
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  • [Title] Granulocytic sarcoma of the colon in a child with acute myeloid leukemia presenting as hematochezia.
  • Granulocytic sarcoma (GS), an extramedullary myeloid tumor composed of immature cells of the granulocytic series, can occur in patients with acute myeloid leukemia (AML), myelodysplastic syndrome, or chronic myelogenous leukemia.
  • We report here an extremely rare case of GS in the colon of a 10-year-old boy with AML presenting with hematochezia.
  • His hematochezia responded rapidly to induction chemotherapy and the patient remained in complete remission after 3-month follow-up.
  • In conclusion, hematochezia may be due to colonic involvement of GS, which should be considered in the differentials in addition to thrombocytopenia, as it is usually encountered in AML patients.
  • [MeSH-major] Colonic Neoplasms / complications. Gastrointestinal Hemorrhage / complications. Gastrointestinal Hemorrhage / diagnosis. Leukemia, Myeloid, Acute / complications. Neoplasms, Multiple Primary / pathology. Sarcoma, Myeloid / complications


68. Tadmor T, Vadazs Z, Dar H, Laor R, Attias D: Hemophagocytic syndrome preceding acute myeloid leukemia with der t [7:17][q12; q11], monosomy, 17 and 5p-. J Pediatr Hematol Oncol; 2006 Aug;28(8):544-6
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  • [Title] Hemophagocytic syndrome preceding acute myeloid leukemia with der t [7:17][q12; q11], monosomy, 17 and 5p-.
  • Hemophagocytic syndrome (HS) is a severe and acute proliferative process of histiocytes, often associated with infection or malignancy.
  • We report a case of a child presented with HS, who progressed later to acute myeloid leukemia (AML)-M4, associated with a clonal evolution, from normal to a complex karyotype consisting of t [7:17] and deletions in chromosomes 7, 17, and 5.
  • This is the second report of involvement of 7q rearrangement in a child with HS that has progressed to AML.
  • Additional studies are required to establish the association reported here, between HS with progression to AML and chromosome rearrangements that involve chromosome 7q.
  • [MeSH-major] Chromosome Aberrations. Chromosomes, Human, Pair 17 / genetics. Chromosomes, Human, Pair 5 / genetics. Chromosomes, Human, Pair 7 / genetics. Leukemia, Myeloid / genetics. Lymphohistiocytosis, Hemophagocytic / genetics
  • [MeSH-minor] Acute Disease. Child, Preschool. Fatal Outcome. Humans. Male. Remission Induction. Treatment Outcome

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  • (PMID = 16912598.001).
  • [ISSN] 1077-4114
  • [Journal-full-title] Journal of pediatric hematology/oncology
  • [ISO-abbreviation] J. Pediatr. Hematol. Oncol.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
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69. Rodriguez V, Anderson PM, Litzow MR, Erlandson L, Trotz BA, Arndt CA, Khan SP, Wiseman GA: Marrow irradiation with high-dose 153Samarium-EDTMP followed by chemotherapy and hematopoietic stem cell infusion for acute myelogenous leukemia. Leuk Lymphoma; 2006 Aug;47(8):1583-92
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  • [Title] Marrow irradiation with high-dose 153Samarium-EDTMP followed by chemotherapy and hematopoietic stem cell infusion for acute myelogenous leukemia.
  • In four patients, aged 15 - 20 years, with high-risk acute myeloid leukemia (AML), high-dose samarium 153-labelled ethylenediaminetetramethylenephosphonate (153Sm-EDTMP) was used for targeted marrow irradiation before preparative chemotherapy conditioning regimens and allogeneic (three patients) or autologous (one patient) hematopoietic stem cell transplantation.
  • Complete cytogenetic and morphologic remission of AML was evident on follow-up marrow aspirate and biopsy specimens from all patients.
  • In two of the four study patients, the disease remains in complete remission and the patients have an excellent quality of life (Eastern Cooperative Oncology Group performance status 0; no medications) and no organ toxicity more than 2 years and more than 4 years, respectively, after their blood and bone marrow transplantations.
  • Thus, in adolescents and adults, 153Sm-EDTMP may provide a relatively simple and effective means for using irradiation to eliminate AML within the marrow.
  • [MeSH-major] Hematopoietic Stem Cell Transplantation / methods. Leukemia, Myeloid, Acute / radiotherapy. Radioisotopes / therapeutic use. Samarium / therapeutic use
  • [MeSH-minor] Adolescent. Adult. Bone Marrow / pathology. Bone Marrow / radiation effects. Dose-Response Relationship, Radiation. Humans. Organometallic Compounds / administration & dosage. Organometallic Compounds / therapeutic use. Organophosphorus Compounds / administration & dosage. Organophosphorus Compounds / therapeutic use. Quality of Life. Remission Induction / methods. Transplantation Conditioning / adverse effects. Transplantation Conditioning / methods

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  • (PMID = 16966270.001).
  • [ISSN] 1042-8194
  • [Journal-full-title] Leukemia & lymphoma
  • [ISO-abbreviation] Leuk. Lymphoma
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Organometallic Compounds; 0 / Organophosphorus Compounds; 0 / Radioisotopes; 122575-21-7 / samarium ethylenediaminetetramethylenephosphonate; 42OD65L39F / Samarium
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70. Veltroni M, Sainati L, Zecca M, Fenu S, Tridello G, Testi AM, Merlone AD, Buldini B, Leszl A, Lo Nigro L, Longoni D, Bernini G, Basso G, AIEOP-MDS Study Group: Advanced pediatric myelodysplastic syndromes: can immunophenotypic characterization of blast cells be a diagnostic and prognostic tool? Pediatr Blood Cancer; 2009 Mar;52(3):357-63
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  • [Title] Advanced pediatric myelodysplastic syndromes: can immunophenotypic characterization of blast cells be a diagnostic and prognostic tool?
  • The current article provides data concerning the blast phenotype in pediatric MDS.
  • PROCEDURE: We evaluated by multiparameter flow cytometry 26 MDS pediatric patients with more than 2% of blast cells at bone marrow morphological examination (17 de novo MDS and 9 secondary MDS) and 145 pediatric de novo acute myeloid leukemia (AML) cases (M3 excluded).
  • As control group, 12 healthy age-matched donors for allogenic bone marrow transplantation (BMD) and 6 regenerating bone marrow samples, collected from children with acute lymphoblastic leukemia (ALL) in remission after induction chemotherapy, were studied.
  • CONCLUSIONS: Our data suggest that the blasts phenotypic features can constitute a diagnostic and prognostic tool also for pediatric MDS.

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  • (PMID = 19061215.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antigens, CD34; 0 / Antigens, CD7
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71. Afzal S, Ishaqi MK, Dupuis A, Doyle J, Gassas A: Early lymphocyte recovery after allogeneic hematopoietic SCT is associated with significant GVL effect in pediatric ALL but not acute myelogenous leukemia-Update study. Bone Marrow Transplant; 2009 Dec;44(12):799-804
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  • [Title] Early lymphocyte recovery after allogeneic hematopoietic SCT is associated with significant GVL effect in pediatric ALL but not acute myelogenous leukemia-Update study.
  • To answer the question whether this is true for AML, we extended our cohort to 207 consecutive children with acute leukemia by adding 71 children with AML who received 75 HSCT's between 1994 and 2005.
  • For the AML cohort, all patients at time of HSCT were in complete morphological remission (CR) except for one patient in CR1, who had 8% blasts in the BM before HSCT.
  • In AML, absolute lymphocyte count <0.3 x 10(9)/l or >0.3 x 10(9)/l on days 21 and 30 were not predictive of relapse with a hazard ratio at day 21=0.88; P=0.8, and hazard ratio at day 30=0.5; P=0.2.
  • [MeSH-major] Graft vs Leukemia Effect. Hematopoietic Stem Cell Transplantation. Leukemia, Myeloid, Acute / blood. Leukemia, Myeloid, Acute / therapy. Lymphocytes. Precursor Cell Lymphoblastic Leukemia-Lymphoma / blood. Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy. Recovery of Function
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Disease-Free Survival. Female. Humans. Infant. Lymphocyte Count. Male. Remission Induction. Retrospective Studies. Survival Rate. Transplantation, Homologous

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  • (PMID = 19421173.001).
  • [ISSN] 1476-5365
  • [Journal-full-title] Bone marrow transplantation
  • [ISO-abbreviation] Bone Marrow Transplant.
  • [Language] eng
  • [Publication-type] Clinical Trial; Comparative Study; Journal Article
  • [Publication-country] England
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72. Horan JT, Alonzo TA, Lyman GH, Gerbing RB, Lange BJ, Ravindranath Y, Becton D, Smith FO, Woods WG, Children's Oncology Group: Impact of disease risk on efficacy of matched related bone marrow transplantation for pediatric acute myeloid leukemia: the Children's Oncology Group. J Clin Oncol; 2008 Dec 10;26(35):5797-801
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  • [Title] Impact of disease risk on efficacy of matched related bone marrow transplantation for pediatric acute myeloid leukemia: the Children's Oncology Group.
  • PURPOSE: There is considerable variation in the use of HLA-matched related bone marrow transplantation (BMT) for the treatment of pediatric patients with newly diagnosed acute myeloid leukemia (AML).
  • Some oncologists have argued that BMT should be offered to most patients in first complete remission (CR).
  • Others have maintained that transplantation in first remission should be reserved for patients with high-risk disease.
  • METHODS: We combined data from four cooperative group clinical trials: Pediatric Oncology Group 8821, Children's Cancer Group (CCG) 2891, CCG 2961, and Medical Research Council 10.
  • RESULTS: The data set included 1,373 pediatric patients with AML in first CR.
  • CONCLUSION: Our study indicates that HLA-matched related BMT is an effective treatment for pediatric patients with intermediate-risk AML in first CR.

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  • (PMID = 18955460.001).
  • [ISSN] 1527-7755
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / CA 98543
  • [Publication-type] Journal Article; Meta-Analysis; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / HLA Antigens
  • [Other-IDs] NLM/ PMC2645105
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73. de Jonge HJ, Weidenaar AC, Ter Elst A, Boezen HM, Scherpen FJ, Bouma-Ter Steege JC, Kaspers GJ, Goemans BF, Creutzig U, Zimmermann M, Kamps WA, de Bont ES: Endogenous vascular endothelial growth factor-C expression is associated with decreased drug responsiveness in childhood acute myeloid leukemia. Clin Cancer Res; 2008 Feb 1;14(3):924-30
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  • [Title] Endogenous vascular endothelial growth factor-C expression is associated with decreased drug responsiveness in childhood acute myeloid leukemia.
  • PURPOSE: We hypothesized that downstream effects of endogenous vascular endothelial growth factor (VEGF)/VEGF receptor signaling on acute myelogenous leukemia (AML) cell survival resulted in increased in vitro cellular drug resistance and a longer time to kill most leukemic cells in vivo upon drug exposure.
  • EXPERIMENTAL DESIGN: In primary AML cells from pediatric patients, VEGFA and VEGFC mRNA expression and in vitro cellular resistance to nine cytotoxic drugs were studied.
  • As in vivo equivalents for in vitro drug resistance, in vivo AML blast reduction upon drug exposure, measured as blast cell reduction on day 15 in the bone marrow and as time in days from diagnosis to complete remission (CR) were used.
  • RESULTS: Increased endogenous VEGFC levels significantly correlated with increased in vitro resistance for six typical AML drugs in primary AML cells from pediatric patients.
  • CONCLUSIONS: These results suggest for the first time that higher endogenous VEGFC levels of AML cells are related to decreased in vitro and in vivo drug responsiveness.
  • [MeSH-major] Leukemia, Myeloid, Acute / genetics. Vascular Endothelial Growth Factor C / genetics

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  • (PMID = 18245556.001).
  • [ISSN] 1078-0432
  • [Journal-full-title] Clinical cancer research : an official journal of the American Association for Cancer Research
  • [ISO-abbreviation] Clin. Cancer Res.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / RNA, Messenger; 0 / VEGFA protein, human; 0 / Vascular Endothelial Growth Factor A; 0 / Vascular Endothelial Growth Factor C
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74. Oliansky DM, Rizzo JD, Aplan PD, Arceci RJ, Leone L, Ravindranath Y, Sanders JE, Smith FO 3rd, Wilmot F, McCarthy PL Jr, Hahn T: The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of acute myeloid leukemia in children: an evidence-based review. Biol Blood Marrow Transplant; 2007 Jan;13(1):1-25
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  • [Title] The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of acute myeloid leukemia in children: an evidence-based review.
  • Clinical research examining the role of hematopoietic stem cell transplantation (SCT) in the therapy of acute myeloid leukemia (AML) in children is presented and critically evaluated in this systematic evidence-based review.
  • Treatment recommendations based on the evidence are presented in the table entitled "Summary of Treatment Recommendations Made by the Expert Panel for Pediatric Acute Myeloid Leukemia" and were reached unanimously by a panel of experts in AML.
  • The identified priority areas of needed future research in pediatric AML include: What is the role of risk group stratification, including the role of cytogenetics, in selection of patients for allogeneic SCT, especially those in first CR?
  • and What is the role of biologically targeted agents (ie, tyrosine kinase inhibitors, farnesyl transferase inhibitors, Flt-3 inhibitors, etc) in the treatment of AML, including induction, consolidation, conditioning regimens, and after SCT?
  • [MeSH-major] Hematopoietic Stem Cell Transplantation / methods. Leukemia, Myeloid / therapy. Leukemia, Myeloid, Acute / therapy
  • [MeSH-minor] Acute Disease. Adolescent. Bone Marrow Transplantation. Child. Child, Preschool. Evidence-Based Medicine. Humans. Remission Induction / methods. Transplantation Conditioning / methods. Transplantation, Autologous. Transplantation, Homologous

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  • (PMID = 17222748.001).
  • [ISSN] 1083-8791
  • [Journal-full-title] Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
  • [ISO-abbreviation] Biol. Blood Marrow Transplant.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Review
  • [Publication-country] United States
  • [Number-of-references] 69
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75. Anoop P, Lancaster DL, Ethell ME, Potter MN, Wotherspoon A: Relapse of primitive mediastinal lymphoma as a myeloid mass following beam autologous transplant and subsequent refractoriness to matched unrelated umbilical cord blood allograft. Pediatr Hematol Oncol; 2009 Mar;26(2):93-9
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  • [Title] Relapse of primitive mediastinal lymphoma as a myeloid mass following beam autologous transplant and subsequent refractoriness to matched unrelated umbilical cord blood allograft.
  • At relapse, there was differentiation into myeloid lineage.
  • Though remission was achieved with AML-type reinduction chemotherapy, the mass recurred post allogenic cord blood stem cell transplant.
  • [MeSH-minor] Cell Differentiation. Child. Humans. Male. Myeloid Cells / pathology. Recurrence. Transplantation, Autologous. Transplantation, Homologous. Treatment Failure

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  • (PMID = 19322740.001).
  • [ISSN] 1521-0669
  • [Journal-full-title] Pediatric hematology and oncology
  • [ISO-abbreviation] Pediatr Hematol Oncol
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] England
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76. Tilak V, Sookmane DD, Gupta V, Shukla J: Myelodysplastic syndrome. Indian J Pediatr; 2008 Jul;75(7):729-32
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  • Pediatric myelodysplastic syndrome (MDS), though rare, constitutes a distinct entity quite different from adult MDS.
  • A pediatric approach to the WHO classification has become necessary since the WHO classification of MDS has failed to address the uniqueness of pediatric MDS.
  • Intensive chemotherapy such as the one used in de novo-acute myeloid leukemia (AML) leads to complete remission in some children and this may be the treatment of choice in pediatric MDS.
  • [MeSH-minor] Age Factors. Child. Diagnosis, Differential. Humans. Leukemia, Myeloid, Acute / diagnosis. Prognosis


77. Styczynski J, Toporski J, Wysocki M, Debski R, Chybicka A, Boruczkowski D, Wachowiak J, Wojcik B, Kowalczyk J, Gil L, Balwierz W, Matysiak M, Krawczuk-Rybak M, Balcerska A, Sonta-Jakimczyk D: Fludarabine, treosulfan and etoposide sensitivity and the outcome of hematopoietic stem cell transplantation in childhood acute myeloid leukemia. Anticancer Res; 2007 May-Jun;27(3B):1547-51
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  • [Title] Fludarabine, treosulfan and etoposide sensitivity and the outcome of hematopoietic stem cell transplantation in childhood acute myeloid leukemia.
  • BACKGROUND: The prognostic role of the ex vivo drug resistance profile has not yet been proved in childhood acute myeloid leukemia (AML).
  • The aim of the study was the analysis of the impact of the ex vivo drug resistance profile in a cohort of 44 children with AML undergoing hematopoietic stem cell transplantation (HSCT).
  • RESULTS: Children who relapsed after transplantation showed higher ex vivo resistance of the leukemic blasts to etoposide, mercaptopurine, thioguanine, fludarabine, mitoxantrone and treosulfan than those who stayed in remission.
  • CONCLUSION: The combined drug resistance profile to fludarabine, treosulfan and etoposide may be useful for better stratification of children with AML undergoing stem cell transplantation or to indicate the necessity for additional post-transplant therapy.
  • [MeSH-major] Antineoplastic Agents / pharmacology. Drug Resistance, Neoplasm. Granulocyte Precursor Cells / drug effects. Hematopoietic Stem Cell Transplantation. Leukemia, Myeloid / surgery
  • [MeSH-minor] Acute Disease. Adolescent. Busulfan / analogs & derivatives. Busulfan / pharmacology. Child. Child, Preschool. Etoposide / pharmacology. Female. Humans. Infant. Male. Prognosis. Recurrence. Treatment Outcome. Vidarabine / analogs & derivatives. Vidarabine / pharmacology

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  • (PMID = 17595774.001).
  • [ISSN] 0250-7005
  • [Journal-full-title] Anticancer research
  • [ISO-abbreviation] Anticancer Res.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] Greece
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 6PLQ3CP4P3 / Etoposide; CO61ER3EPI / treosulfan; FA2DM6879K / Vidarabine; G1LN9045DK / Busulfan; P2K93U8740 / fludarabine
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78. Strunk CJ, Platzbecker U, Thiede C, Schaich M, Illmer T, Kang Z, Leahy P, Li C, Xie X, Laughlin MJ, Lazarus HM, Gerson SL, Bunting KD, Ehninger G, Tse W: Elevated AF1q expression is a poor prognostic marker for adult acute myeloid leukemia patients with normal cytogenetics. Am J Hematol; 2009 May;84(5):308-9
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  • [Title] Elevated AF1q expression is a poor prognostic marker for adult acute myeloid leukemia patients with normal cytogenetics.
  • Nearly half of the patients with newly diagnosed acute myeloid leukemia have normal cytogenetics (NC-AML) and are classified as intermediate risk, but their 5-year overall survival (OS) ranges from 24 to 42%.
  • Elevated AF1q expression in the absence of specific poor cytogenetics is associated with poor outcomes in pediatric patients with AML and adult patients with myelodysplastic syndrome.
  • We examined AF1q expression in 290 patients with NC-AML.
  • We found that patients with low AF1q (n = 73) expression (AF1q(low)) have better OS (P = 0.026), disease-free survival (P = 0.1), and complete remission rate (P = 0.06) when compared with patients with high AF1q expression (AF1q(high) n = 217).
  • This study suggests that high AF1q expression is a poor prognostic marker for adult patients with NC-AML.
  • [MeSH-major] Biomarkers, Tumor. Blood Proteins / metabolism. Leukemia, Myeloid, Acute / metabolism. Leukemia, Myeloid, Acute / therapy. Neoplasm Proteins / metabolism

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  • (PMID = 19396856.001).
  • [ISSN] 1096-8652
  • [Journal-full-title] American journal of hematology
  • [ISO-abbreviation] Am. J. Hematol.
  • [Language] eng
  • [Grant] United States / PHS HHS / / NIH/K12; United States / PHS HHS / / NIH/T32
  • [Publication-type] Letter; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Biomarkers, Tumor; 0 / Blood Proteins; 0 / MLLT11 protein, human; 0 / Neoplasm Proteins; 0 / Proto-Oncogene Proteins; EC 2.7.10.1 / fms-Like Tyrosine Kinase 3
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79. Lange BJ, Smith FO, Feusner J, Barnard DR, Dinndorf P, Feig S, Heerema NA, Arndt C, Arceci RJ, Seibel N, Weiman M, Dusenbery K, Shannon K, Luna-Fineman S, Gerbing RB, Alonzo TA: Outcomes in CCG-2961, a children's oncology group phase 3 trial for untreated pediatric acute myeloid leukemia: a report from the children's oncology group. Blood; 2008 Feb 01;111(3):1044-53
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  • [Title] Outcomes in CCG-2961, a children's oncology group phase 3 trial for untreated pediatric acute myeloid leukemia: a report from the children's oncology group.
  • CCG-2961 incorporated 3 new agents, idarubicin, fludarabine and interleukin-2, into a phase 3 AML trial using intensive-timing remission induction/consolidation and related donor marrow transplantation or high-dose cytarabine intensification.
  • Partial replacement of daunomycin with idarubicin in the 5-drug induction combination achieved a remission rate of 88%, similar to historical controls.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Idarubicin / therapeutic use. Interleukin-2 / therapeutic use. Leukemia, Myeloid, Acute / drug therapy. Vidarabine / analogs & derivatives

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  • (PMID = 18000167.001).
  • [ISSN] 0006-4971
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / CA 13538; United States / NCI NIH HHS / CA / CA 13539; United States / NCI NIH HHS / CA / CA 98543
  • [Publication-type] Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Interleukin-2; FA2DM6879K / Vidarabine; P2K93U8740 / fludarabine; ZRP63D75JW / Idarubicin
  • [Other-IDs] NLM/ PMC2214754
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80. Balamurugan S, Sugapriya D, Shanthi P, Thilaka V, Venkatadesilalu S, Pushpa V, Madhavan M: Multidrug resistance 1 gene expression and AgNOR in childhood acute leukemias. Indian J Hematol Blood Transfus; 2007 Dec;23(3-4):73-8

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Multidrug resistance 1 gene expression and AgNOR in childhood acute leukemias.
  • We have studied MDR1 expression and AgNORS in 41 cases of acute leukemia in children.
  • In this study, AgNOR counts in patients with acute lymphoblastic leukemia (ALL) L2 subtype (FAB classification) were significantly higher as compared to the ALL L1 subtype.
  • Similarly, mean AgNOR count in the acute myeloid Leukemia (AML) M2 subtype was significantly higher as compared to the ALL L1 subtype.
  • However, there was no correlation between AgNOR and treatment outcome or between AgNOR counts and MDR1 expression in any of the subtypes of acute leukemia included in this series.
  • In AML, MDR1 gene expression was found to be related to reduced remission induction rates and hence poorer prognosis.
  • In ALL, our study has shown no difference in remission induction between MDR1 positive and MDR1 negative cases.
  • This would suggest that factors other than MDR1 may be of relevance in Pediatric ALL.

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  • (PMID = 23100919.001).
  • [ISSN] 0971-4502
  • [Journal-full-title] Indian journal of hematology & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion
  • [ISO-abbreviation] Indian J Hematol Blood Transfus
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] India
  • [Other-IDs] NLM/ PMC3453125
  • [Keywords] NOTNLM ; Acute leukemia / AgNOR / Multidrug Resistance 1 / P-glycoprotein
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81. Gamis AS: Acute myeloid leukemia and Down syndrome evolution of modern therapy--state of the art review. Pediatr Blood Cancer; 2005 Jan;44(1):13-20
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  • [Title] Acute myeloid leukemia and Down syndrome evolution of modern therapy--state of the art review.
  • Over the past decade, a series of clinical reports have described the experience of Down syndrome (DS) children treated for acute myeloid leukemia (AML).
  • Whereas prior to the first reports in the early 1980's it was felt that DS children with AML had poor outcomes, these clinical trials concluded that DS had a better outcome than non-DS (NDS) children with AML.
  • This review focuses upon the six multi-institutional reports that described the DS and AML experience in order to better ascertain the chemotherapy combinations that may be useful in the future for these children.
  • In general, the remission rates are approximately 90% with event-free survival (EFS) approximating 70-80%.
  • AML in DS is unique and these differences in comparison to NDS children are highlighted.
  • The significantly better outcomes for DS children likely represents a combination of the unique AML seen in DS children and the heightened sensitivity to cytarabine that DS AML cells have.
  • Future trials should focus on age-stratified approaches that exploit the greater sensitivity of DS AML to cytarabine.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Down Syndrome / complications. Leukemia, Myeloid / drug therapy. Leukemia, Myeloid / etiology
  • [MeSH-minor] Acute Disease. Child. Humans. Prognosis. Risk Assessment

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  • [Copyright] (c) 2004 Wiley-Liss, Inc.
  • (PMID = 15534881.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] United States
  • [Number-of-references] 22
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82. Kobayashi R, Tawa A, Hanada R, Horibe K, Tsuchida M, Tsukimoto I, Japanese childhood AML cooperative study group: Extramedullary infiltration at diagnosis and prognosis in children with acute myelogenous leukemia. Pediatr Blood Cancer; 2007 Apr;48(4):393-8
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  • [Title] Extramedullary infiltration at diagnosis and prognosis in children with acute myelogenous leukemia.
  • BACKGROUND: Extramedullary infiltration (EMI) is an occasional clinical symptom in childhood acute myelogenous leukemia (AML), but there is considerable controversy regarding the prognostic significance of EMI in AML.
  • PROCEDURE: We evaluated the frequency and prognostic significance of EMI at diagnosis of AML in children.
  • RESULTS: Of 240 cases of de novo AML excluding children with Down syndrome and acute promyelocytic leukemia, 56 (23.3%) showed EMI at diagnosis.
  • The complete remission rate following induction chemotherapy was lower in patients with EMI.
  • CONCLUSIONS: CNS leukemia and EMI together with a WBC count of >100 x 10(9)/L at diagnosis of AML are high risk factors for relapse, and alternative treatment approaches for patients with these characteristics should be explored.
  • [MeSH-major] Leukemia, Myeloid / pathology. Leukemic Infiltration / epidemiology. Sarcoma, Myeloid / epidemiology
  • [MeSH-minor] Acute Disease. Adolescent. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Bone and Bones / pathology. Central Nervous System / pathology. Child. Child, Preschool. Cytarabine / administration & dosage. Disease-Free Survival. Etoposide / administration & dosage. Female. Follow-Up Studies. Gingiva / pathology. Humans. Hydrocortisone / administration & dosage. Idarubicin / administration & dosage. Infant. Infant, Newborn. Japan / epidemiology. Kaplan-Meier Estimate. Male. Methotrexate / administration & dosage. Orbit / pathology. Prognosis. Remission Induction. Skin / pathology. Testis / pathology

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  • (PMID = 16550530.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 04079A1RDZ / Cytarabine; 6PLQ3CP4P3 / Etoposide; WI4X0X7BPJ / Hydrocortisone; YL5FZ2Y5U1 / Methotrexate; ZRP63D75JW / Idarubicin
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83. Inaba H, Fan Y, Pounds S, Geiger TL, Rubnitz JE, Ribeiro RC, Pui CH, Razzouk BI: Clinical and biologic features and treatment outcome of children with newly diagnosed acute myeloid leukemia and hyperleukocytosis. Cancer; 2008 Aug 1;113(3):522-9
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Clinical and biologic features and treatment outcome of children with newly diagnosed acute myeloid leukemia and hyperleukocytosis.
  • BACKGROUND: Acute myeloid leukemia (AML) with hyperleukocytosis often is associated with early complications.
  • To the authors' knowledge, no recently published study has evaluated the management and clinical course in this regard, especially in relation to pediatric patients.
  • METHODS: The authors reviewed 579 patients with newly diagnosed pediatric AML who were treated at St. Jude Children's Research Hospital from 1968 to 2002 and carefully examined 106 patients with initial leukocyte counts > or = 100 x 10(9)/L and French-American-British (FAB) AML subtypes other than M3.
  • These patients with hyperleukocytosis were divided into 2 groups-'before' (early period; 70 patients) and 'after' (late period; 36 patients) the initiation of the AML-83 protocol-to address potential differences in supportive measures (including leukoreduction).
  • In the late period, patients with or without hyperleukocytosis had similar complete remission rates.
  • CONCLUSIONS: With improved management, including supportive care, early mortality in patients with AML and hyperleukocytosis decreased remarkably in the more recent period.
  • [MeSH-major] Leukemia, Myeloid, Acute / diagnosis. Leukemia, Myeloid, Acute / therapy. Leukocytosis / diagnosis. Leukocytosis / therapy

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  • [Copyright] (c) 2008 American Cancer Society
  • (PMID = 18484648.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA21765
  • [Publication-type] Evaluation Studies; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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84. Bierings M, Nachman JB, Zwaan CM: Stem cell transplantation in pediatric leukemia and myelodysplasia: state of the art and current challenges. Curr Stem Cell Res Ther; 2007 Jan;2(1):53-63
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  • [Title] Stem cell transplantation in pediatric leukemia and myelodysplasia: state of the art and current challenges.
  • The role of stem cell transplantation in the treatment of leukemia and myelodysplasia (MDS) in children has changed over the past decade.
  • In pediatric acute lymphoblastic leukemia (ALL), the overall cure-rate is high with conventional chemotherapy.
  • However, selected patients with a high-risk of relapse are often treated with allogeneic hematopoietic stem cell transplantation (allo-HSCT) in first remission (CR1).
  • Patients with a bone-marrow relapse who attain a second remission frequently receive HSCT.
  • In pediatric acute myeloid leukemia (AML) the role of allo-HSCT in CR1 is declining, due to better outcome with modern multi-agent chemotherapy.
  • In relapsed AML patients, allo-HSCT still seems indispensable.
  • Targeted therapy may change the role of HSCT, in particular in chronic myeloid leukemia, where the role of allografting is changing in the imatinib era.
  • [MeSH-major] Leukemia / therapy. Myelodysplastic Syndromes / therapy. Stem Cell Transplantation / trends
  • [MeSH-minor] Child. Humans. Leukemia, Myelogenous, Chronic, BCR-ABL Positive / therapy. Leukemia, Myeloid, Acute / therapy


85. Abdel Rahman H, Farrag SA, El-Attar IA: AML1/ETO Fusion Gene in de novo Pediatric Acute Myeloid Leukemia: Clinical Significance and Prognostic Implications. J Egypt Natl Canc Inst; 2007 Mar;19(1):39-47

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  • [Title] AML1/ETO Fusion Gene in de novo Pediatric Acute Myeloid Leukemia: Clinical Significance and Prognostic Implications.
  • The characterization of leukemia-associated chromosome translocations has contributed relevant insights into our understanding of leukemia pathogenesis and has provided new specific tumor markers essential in prognostic assessment and minimal residual disease studies.
  • The aim of this work is to study the frequency of AML1/ETO fusion gene in a series of Egyptian childhood AML cases.
  • The clinical significance and prognostic implications of this aberration, including CR rate, duration of first CR, extramedullary leukemia (EML), and survival are investigated as well.
  • Peripheral blood and/or bone marrow mononuclear cells were available for analysis from 78 children, all newly diagnosed with AML.
  • Patients with de novo AML were treated by 2 courses of induction chemotherapy, followed by 4 courses of consolidation treatment if the patient achieved complete remission (CR).
  • Lymph nodes were enlarged in 8/15 cases (53.34%), hepatomegly was observed in 4/15 cases (26.67%), splenomegaly in 8/15 cases (53.34%), purpura in 6/15 cases (40%), while pallor was observed in all fifteen cases.Extramedullary leukemia occurred in 4/15 cases (26.67%).
  • As regards the fate of the positive cases, thirteen cases (86.67%) attained complete remission (CR) following induction chemotherapy.
  • Eight patients were in complete continuous remission (CCR), four patients (26.67%) relapsed and died during relapse, and one patient (6.67%) died in complete remission due to severe neutropenia and infection.
  • In conclusion, we report a frequency of 19.2% of AML1/ETO fusion gene in our newly diagnosed pediatric AML cases.
  • Positive cases showed good response to induction therapy, as well as high complete remission rates, which are features of good prognosis.
  • Key Words: Pediatric acute myeloid leukemia , AML1/ETO fusion gene , RT-PCR , Clinical outcome , Prognostic significance.

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  • (PMID = 18839034.001).
  • [ISSN] 1110-0362
  • [Journal-full-title] Journal of the Egyptian National Cancer Institute
  • [ISO-abbreviation] J Egypt Natl Canc Inst
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Egypt
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86. Lee DH, Kwon YJ, Lim J, Kim Y, Han K, Chung NG, Jeong DC, Cho B, Kim HK: Comparable outcomes of HLA-matched unrelated and HLA-identical sibling donor bone marrow transplantation for childhood acute myeloid leukemia in first remission. Pediatr Transplant; 2009 Mar;13(2):210-6
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  • [Title] Comparable outcomes of HLA-matched unrelated and HLA-identical sibling donor bone marrow transplantation for childhood acute myeloid leukemia in first remission.
  • We retrospectively investigated the outcomes of HLA-matched unrelated BMT (MU-BMT, n = 13) and HLA-identical sibling donor BMT (MS-BMT, n = 17) for childhood AML in CR1 between June 2002 and August 2005.
  • The cumulative incidence of grade II-IV acute GVHD and any chronic GVHD at three yr was not different between MS-BMT and MU-BMT.
  • The outcome of HLA-matched unrelated BMT is comparable to that of HLA-identical sibling BMT for childhood AML in CR1.
  • HLA-matched unrelated BMT may be recommended for patients who have AML in CR1 without an HLA-matched sibling donor.
  • [MeSH-major] Bone Marrow Transplantation / methods. HLA Antigens / metabolism. Leukemia, Myeloid, Acute / therapy
  • [MeSH-minor] Adolescent. Adult. Blood Platelets / metabolism. Child. Child, Preschool. Female. Humans. Living Donors. Male. Neutrophils / metabolism. Remission Induction. Retrospective Studies. Siblings. Treatment Outcome


87. Gorman MF, Ji L, Ko RH, Barnette P, Bostrom B, Hutchinson R, Raetz E, Seibel NL, Twist CJ, Eckroth E, Sposto R, Gaynon PS, Loh ML: Outcome for children treated for relapsed or refractory acute myelogenous leukemia (rAML): a Therapeutic Advances in Childhood Leukemia (TACL) Consortium study. Pediatr Blood Cancer; 2010 Sep;55(3):421-9
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  • [Title] Outcome for children treated for relapsed or refractory acute myelogenous leukemia (rAML): a Therapeutic Advances in Childhood Leukemia (TACL) Consortium study.
  • BACKGROUND: Current event-free survival (EFS) rates for children with newly diagnosed acute myeloid leukemia (AML) approach 50-60%.
  • This report highlights efforts to assess the response rates and survival outcomes after first or greater relapse in children with AML.
  • PROCEDURE: We performed a retrospective cohort review of pediatric patients with relapsed and refractory AML (rAML) previously treated at TACL institutions between the years of 1995 and 2004.
  • [MeSH-major] Leukemia, Myeloid, Acute / drug therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Disease-Free Survival. Female. Humans. Infant. Male. Prognosis. Recurrence. Remission Induction. Retreatment. Survival Rate. Treatment Outcome. Young Adult

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  • [Copyright] 2010 Wiley-Liss, Inc.
  • (PMID = 20658611.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / K22 CA113557
  • [Publication-type] Journal Article; Multicenter Study; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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88. Kelly MJ, Meloni-Ehrig AM, Manley PE, Altura RA: Poor outcome in a pediatric patient with acute myeloid leukemia associated with a variant t(8;21) and trisomy 6. Cancer Genet Cytogenet; 2009 Feb;189(1):48-52
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  • [Title] Poor outcome in a pediatric patient with acute myeloid leukemia associated with a variant t(8;21) and trisomy 6.
  • RUNX1T1/RUNX1 (formerly ETO/AML1) is a molecular marker that is usually associated with a favorable outcome in both pediatric and adult patients with acute myeloid leukemia (AML).
  • We describe a 10-year-old girl with AML associated with an RUNX1T1/RUNX1 fusion.
  • She had an early relapse while being treated on a standard protocol and had significant difficulty in attaining a second remission.
  • Trisomy 6 is an uncommon cytogenetic abnormality in myeloid diseases.
  • As a sole abnormality, it has been associated mainly with myelodysplastic syndrome and AML.
  • The presence of this novel variant of t(8;21)(q22;q22) associated with trisomy 6 may have abrogated the usual favorable prognosis associated with RUNX1T1/RUNX1 in AML.
  • [MeSH-major] Chromosomes, Human, Pair 21 / genetics. Chromosomes, Human, Pair 6 / genetics. Chromosomes, Human, Pair 8 / genetics. Leukemia, Myeloid, Acute / genetics. Translocation, Genetic / genetics. Trisomy / genetics

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  • (PMID = 19167612.001).
  • [ISSN] 1873-4456
  • [Journal-full-title] Cancer genetics and cytogenetics
  • [ISO-abbreviation] Cancer Genet. Cytogenet.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / AML1-ETO fusion protein, human; 0 / Core Binding Factor Alpha 2 Subunit; 0 / Oncogene Proteins, Fusion
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89. Rubnitz JE, Gibson B, Smith FO: Acute myeloid leukemia. Pediatr Clin North Am; 2008 Feb;55(1):21-51, ix
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  • [Title] Acute myeloid leukemia.
  • Acute myeloid leukemia (AML) is a heterogeneous group of leukemias that result from clonal transformation of hematopoietic precursors through the acquisition of chromosomal rearrangements and multiple gene mutations.
  • As a result of highly collaborative clinical research by pediatric cooperative cancer groups worldwide, disease-free survival has improved significantly during the past 3 decades.
  • Further improvements in outcomes of children who have AML probably will reflect continued progress in understanding the biology of AML and the concomitant development of new molecularly targeted agents for use in combination with conventional chemotherapy drugs.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid, Acute / therapy. Stem Cell Transplantation
  • [MeSH-minor] Adolescent. Anthracyclines / administration & dosage. Child. Child, Preschool. Clinical Trials as Topic. Cytarabine / administration & dosage. Humans. Infant. Pharmacogenetics. Prognosis. Remission Induction. Risk Factors

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  • (PMID = 18242314.001).
  • [ISSN] 0031-3955
  • [Journal-full-title] Pediatric clinics of North America
  • [ISO-abbreviation] Pediatr. Clin. North Am.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Review
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Anthracyclines; 04079A1RDZ / Cytarabine
  • [Number-of-references] 229
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90. Adachi S, Manabe A, Imaizumi M, Taga T, Tawa A, Tsurusawa M, Kikuchi A, Masunaga A, Tsuchida M, Nakahata T, MDS Committee of the Japanese Society of Pediatric Hematology: Acute myeloid leukemia with multilineage dysplasia in children. Int J Hematol; 2007 Nov;86(4):358-63
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  • [Title] Acute myeloid leukemia with multilineage dysplasia in children.
  • We retrospectively surveyed pediatric acute myeloid leukemia (AML) patients with multilineage dysplasia treated with the AML 99 and the Children's Cancer and Leukemia Study Group (CCLSG) AML 9805 protocols.
  • We found only 9 AML patients (2.6%) with multilineage dysplasia among the 341 patients with newly diagnosed de novo AML.
  • Eight of the 9 patients obtained complete remission (CR) following the intensive AML-oriented treatments.
  • No reports have described AML with multilineage dysplasia in children, and the incidence of the disease is expected to be very low.
  • We plan to conduct a prospective pathologic review to select cases with this disease entity in the next Japanese Pediatric Leukemia/Lymphoma Study Group (JPLSG) AML-05 protocol.
  • [MeSH-major] Cell Lineage. Leukemia, Myeloid, Acute / pathology

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  • (PMID = 18055345.001).
  • [ISSN] 0925-5710
  • [Journal-full-title] International journal of hematology
  • [ISO-abbreviation] Int. J. Hematol.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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91. Hu SY, Gu WY, Chen ZX, Wang XL, Cen JN, He HL, Chai YH, Chen CS: The significance of detecting WT1 expression in childhood acute leukemias. Pediatr Hematol Oncol; 2010 Nov;27(8):581-91
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  • [Title] The significance of detecting WT1 expression in childhood acute leukemias.
  • WT1 (Wilms' tumor gene 1) overexpression is implicated in the prognosis of acute leukemia.
  • The purpose of this study was to investigate WT1 expression and its clinical implication in childhood acute leukemia (AL) in Chinese population.
  • Bone marrow specimen from 200 children at different stages of acute leukemia and from 21 children without leukemia were studied.
  • The WT1 expression at diagnostic marrow specimen in both acute myeloid leukemia (AML) and acute lymphoid leukemia (ALL) was higher than control group, whereas WT1 expression in AML was higher than in ALL, and WT1 expression level in relapse in ALL increased more significantly than in AML.
  • The WT1 expression level showed positive correlation with the hypodiploidy and BCR-ABL fusion gene in acute leukemia.
  • A rapidly decrease of WT1 expression level predicted a good response to the induction therapy and low expression of WT1 correlates with remission status.
  • This study suggested that WT1 expression levels in acute leukemia can potentially be a marker for evaluating therapeutic efficacy, correlating with monitoring minimal residue disease, and predicting hematological relapse in children acute leukemia.
  • [MeSH-major] Leukemia, Myeloid, Acute / genetics. Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics. WT1 Proteins / genetics

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  • (PMID = 20863155.001).
  • [ISSN] 1521-0669
  • [Journal-full-title] Pediatric hematology and oncology
  • [ISO-abbreviation] Pediatr Hematol Oncol
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 0 / WT1 Proteins; EC 2.7.10.2 / Fusion Proteins, bcr-abl
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92. Johnston DL, Alonzo TA, Gerbing RB, Lange BJ, Woods WG: The presence of central nervous system disease at diagnosis in pediatric acute myeloid leukemia does not affect survival: a Children's Oncology Group study. Pediatr Blood Cancer; 2010 Sep;55(3):414-20
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  • [Title] The presence of central nervous system disease at diagnosis in pediatric acute myeloid leukemia does not affect survival: a Children's Oncology Group study.
  • BACKGROUND: The presence of central nervous system (CNS) disease in pediatric acute myeloid leukemia (AML) is often thought to confer a worse prognosis.
  • This study examined the outcome of children with AML who had CNS disease at diagnosis.
  • METHODS: Patients enrolled on Children's Cancer Group protocols 2861, 2891, 2941, and 2961 being treated for de novo AML were classified for the presence of CNS disease at diagnosis as CNS1 (<5 WBC in the CSF without blasts), CNS2 (<5 WBC in the CSF with blasts), or CNS3 (> or =5 WBC in the CSF with blasts).
  • There were no significant differences in overall survival, event free survival, or remission rates between the groups; however, a significant difference was seen between the CNS1 and CNS3 groups in disease free survival and isolated CNS relapse risk.

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  • [Copyright] 2010 Wiley-Liss, Inc.
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  • (PMID = 20658610.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / U10 CA98413; United States / NCI NIH HHS / CA / U10 CA098413; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / U10 CA098543-01; United States / NCI NIH HHS / CA / U10 CA98543
  • [Publication-type] Journal Article; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Other-IDs] NLM/ NIHMS218661; NLM/ PMC2990693
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93. Mandrell BN, Pritchard M: Understanding the clinical implications of minimal residual disease in childhood leukemia. J Pediatr Oncol Nurs; 2006 Jan-Feb;23(1):38-44

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  • [Title] Understanding the clinical implications of minimal residual disease in childhood leukemia.
  • Improved laboratory techniques now allow a more sensitive detection of leukemia cells at designated intervals throughout therapy.
  • Using flow cytometry and polymerase chain reaction, it is possible to detect 1 leukemic cell among 10(4) normal cells (1 leukemia cell in 10,000 normal cells), representing a 100-fold greater sensitivity than morphological examination in acute lymphoblastic leukemia (ALL).
  • Recently, it has been shown that the molecular presence of persistent acute lymphoblastic leukemia at the end of remission therapy is a poor indicator of clinical outcome.
  • Now similar studies are being performed in acute myeloid leukemia (AML).
  • While the sensitivity using flow cytometry is less in AML than in ALL (able to detect 1 leukemic cell among 1000 normal cells in AML), persistent or minimal residual AML provides the clinician guidance with future treatment recommendations.
  • Therefore, it is imperative that the nurse have an understanding of the newer techniques to study residual leukemia and their clinical implications for patients and their families.
  • [MeSH-major] Leukemia, Myeloid / pathology. Neoplasm, Residual / pathology. Precursor Cell Lymphoblastic Leukemia-Lymphoma / pathology

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  • (PMID = 16689404.001).
  • [ISSN] 1043-4542
  • [Journal-full-title] Journal of pediatric oncology nursing : official journal of the Association of Pediatric Oncology Nurses
  • [ISO-abbreviation] J Pediatr Oncol Nurs
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] United States
  • [Number-of-references] 28
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94. Lange BJ, Gerbing RB, Feusner J, Skolnik J, Sacks N, Smith FO, Alonzo TA: Mortality in overweight and underweight children with acute myeloid leukemia. JAMA; 2005 Jan 12;293(2):203-11
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  • [Title] Mortality in overweight and underweight children with acute myeloid leukemia.
  • CONTEXT: Current treatment for acute myeloid leukemia (AML) in children cures about half the patients.
  • Of the other half, most succumb to leukemia, but 5% to 15% die of treatment-related complications.
  • Overweight children with AML seem to experience excess life-threatening and fatal toxicity.
  • Nothing is known about how weight affects outcomes in pediatric AML.
  • OBJECTIVE: To compare survival rates in children with AML who at diagnosis are underweight (body mass index [BMI] < or =10th percentile), overweight (BMI > or =95th percentile), or middleweight (BMI = 11th-94th percentiles).
  • DESIGN, SETTING, AND PARTICIPANTS: Retrospective review of BMI and survival in 768 children and young adults aged 1 to 20 years enrolled in Children's Cancer Group-2961, an international cooperative group phase 3 trial for previously untreated AML conducted August 30, 1996, through December 4, 2002.
  • CONCLUSION: Treatment-related complications significantly reduce survival in overweight and underweight children with AML.

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  • (PMID = 15644547.001).
  • [ISSN] 1538-3598
  • [Journal-full-title] JAMA
  • [ISO-abbreviation] JAMA
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / CA098413; United States / NCI NIH HHS / CA / CA098543
  • [Publication-type] Clinical Trial; Clinical Trial, Phase III; Comparative Study; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.
  • [Publication-country] United States
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95. Yeh TC, Liu HC, Wang LY, Chen SH, Lin WY, Liang DC: The development of a novel protocol for the treatment of de novo childhood acute myeloid leukemia in a single institution in Taiwan. J Pediatr Hematol Oncol; 2007 Dec;29(12):826-31
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] The development of a novel protocol for the treatment of de novo childhood acute myeloid leukemia in a single institution in Taiwan.
  • From November 1, 1995 to July 31, 2004, 49 children with de novo acute myeloid leukemia (AML) were treated at our institution.
  • In total, 48 patients with de novo AML were enrolled in this study.
  • Forty-two patients with AML other than acute promyelocytic leukemia (non-APL) were treated consecutively with 2 novel protocols: Mackay Memorial Hospital (MMH)-AML-96, designed as a pilot phase, and Taiwan Pediatric Oncology Group (TPOG)-AML-97A, on the basis of MMH-AML-96 with minor modifications.
  • As of July 31, 2006, the remission rates were 79%, 92%, and 98% after 1, 2, and 3 courses of induction therapy, respectively.
  • The 5-year overall survival was 64%+/-6.9% (SE), and the 5-year event-free survival was 60%+/-7.1%; for non-APL AML, the rates were 62%+/-7.5% and 59%+/-7.6%; for APL, 83+/-15.2 and 67+/-19.3%.
  • Among the factors analyzed, a complete remission achieved after 1 course of induction therapy, lactate dehydrogenase <500 IU/L at diagnosis, patients without invasive fungal infection during chemotherapy, and male sex were associated with a favorable outcome.
  • [MeSH-major] Antineoplastic Agents / therapeutic use. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid, Acute / drug therapy. Tretinoin / therapeutic use


96. Roberson JR, Onciu M, Pounds S, Rubnitz JE, Pui CH, Razzouk BI: Prognostic significance of myeloperoxidase expression in childhood acute myeloid leukemia. Pediatr Blood Cancer; 2008 Mar;50(3):542-8
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Prognostic significance of myeloperoxidase expression in childhood acute myeloid leukemia.
  • BACKGROUND: The percentage of myeloperoxidase (MPO)-positive blast cells is associated with prognosis in adult acute myeloid leukemia (AML), but this association is unsubstantiated in pediatric AML.
  • PROCEDURE: We retrospectively compared cytochemical MPO results with outcome in 154 patients younger than 21 years treated on three consecutive institutional protocols for newly diagnosed AML (1987-2001).
  • Patients with FAB M0 and M7 AML (no MPO expression) or M3 AML (100% MPO expression) and Down's syndrome were excluded.
  • The percentage of MPO-positive blasts was not significantly associated with the probability of complete remission (P = 0.97), event-free survival (P = 0.72), or survival (P = 0.76) in multivariate analyses that accounted for age, FAB subtype, presenting WBC count, cytogenetic and protocol treatment risk group.
  • In analysis limited to patients with intermediate-risk cytogenetics, higher MPO expression appeared to be associated with improved EFS (P = 0.06) but was not associated with remission induction rate (P = 0.16) or overall survival (P = 0.38).
  • CONCLUSIONS: The percentage of MPO-positive blast cells is related to FAB subtype in pediatric AML but has limited prognostic relevance.
  • [MeSH-major] Leukemia, Myeloid / blood. Myeloid Cells / enzymology. Neoplastic Stem Cells / enzymology. Peroxidase / blood
  • [MeSH-minor] Acute Disease. Adolescent. Adult. Biomarkers. Child. Child, Preschool. Disease-Free Survival. Female. Humans. Infant. Kaplan-Meier Estimate. Karyotyping. Male. Prognosis. Retrospective Studies. Risk. Survival Analysis

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  • [Copyright] (c) 2007 Wiley-Liss, Inc.
  • (PMID = 17763467.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA-21765
  • [Publication-type] Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Biomarkers; EC 1.11.1.7 / Peroxidase
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97. Tavil B, Aytac S, Balci YI, Unal S, Kuskonmaz B, Yetgin S, Gurgey A, Tuncer M, Gumruk F, Uckan D, Cetin M: Fludarabine, cytarabine, granulocyte colony-stimulating factor, and idarubicin (FLAG-IDA) for the treatment of children with poor-prognosis acute leukemia: the Hacettepe experience. Pediatr Hematol Oncol; 2010 Oct;27(7):517-28
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Fludarabine, cytarabine, granulocyte colony-stimulating factor, and idarubicin (FLAG-IDA) for the treatment of children with poor-prognosis acute leukemia: the Hacettepe experience.
  • Fludarabine, cytarabine, granulocyte colony-stimulating factor (G-CSF), and idarubicin (FLAG-IDA) regimen has been proven to be a potentially useful chemotherapy regimen for relapsed or poor-prognosis childhood leukemia.
  • The aim of the study was to evaluate complete remission (CR) rate, toxicity, and overall survival of children with poor-prognosis acute leukemia who received the FLAG-IDA regimen.
  • Between January 2002 and April 2007, 25 children with poor-prognosis acute leukemia were treated with FLAG-IDA regimen in our center.
  • Of the 25 children (16 AML, 9 ALL) with poor-prognosis acute leukemia, 7 (28.0%) received 1 cycle, 17 (68.0%) received 2 cycles, and 1 (4%) received 3 cycles of FLAG or FLAG-IDA regimen.
  • Five (20%) of these patients in CR who underwent allogeneic HSCT are still in remission.
  • The CR rate was quite high in the present study using the FLAG-IDA regimen, and the authors believe this regimen is a possible option prior to allogeneic HSCT in children with poor-prognosis acute leukemia.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Cytarabine / therapeutic use. Granulocyte Colony-Stimulating Factor / therapeutic use. Idarubicin / therapeutic use. Leukemia, Myeloid, Acute / drug therapy. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy. Vidarabine / analogs & derivatives

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  • (PMID = 20677923.001).
  • [ISSN] 1521-0669
  • [Journal-full-title] Pediatric hematology and oncology
  • [ISO-abbreviation] Pediatr Hematol Oncol
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 04079A1RDZ / Cytarabine; 143011-72-7 / Granulocyte Colony-Stimulating Factor; FA2DM6879K / Vidarabine; P2K93U8740 / fludarabine; ZRP63D75JW / Idarubicin
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98. Chen YM, Liu TF, Ruan M, Zou Y, Chen XJ, Guo Y, Wang SC, Zhu XF: [Prognosis and chromosomal abnormalities in 79 children with t (8;21) acute myeloid leukemia]. Zhongguo Yi Xue Ke Xue Yuan Xue Bao; 2009 Oct;31(5):542-6
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] [Prognosis and chromosomal abnormalities in 79 children with t (8;21) acute myeloid leukemia].
  • OBJECTIVE: To investigate the chromosomal abnormalities and evaluate the prognostic value of post-remission chemotherapy in children with t (8;21) acute myeloid leukemia (AML).
  • METHODS: The diagnosis of AML and its subtyping were performed using morphological, immunological, and cytogenetic methodologies in 79 children.
  • Allogeneic stem cell transplantation or 5-6 cycles of intensive chemotherapy was performed after remission therapy.
  • The complete remission (CR) rates were 81.7% (49/60) and 94.8% (55/58), respectively, after one and two cycles of induction chemotherapy.
  • Post-remission consolidation by high dose cytarabine (HDAC) was significantly superior to standard chemotherapy (66.7% vs. 27.3%, P = 0.03).
  • CONCLUSION: Most children with t (8;21) AML have additional chromosomal abnormalities, although they do not affect the prognosis and long-term survival.
  • Childhood t (8;21) AML usually has high CR rate with relatively good prognosis, and post-remission consolidation by HDAC can improve the survival.
  • [MeSH-major] Chromosome Aberrations. Leukemia, Myeloid, Acute / genetics

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  • (PMID = 19968066.001).
  • [ISSN] 1000-503X
  • [Journal-full-title] Zhongguo yi xue ke xue yuan xue bao. Acta Academiae Medicinae Sinicae
  • [ISO-abbreviation] Zhongguo Yi Xue Ke Xue Yuan Xue Bao
  • [Language] chi
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] China
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99. Corral Mdel P, Villa O, Alfaro EM, Alonso CN, Baro C, Felice MS, Rossi J, Solé F, Gallego MS: Complex chromosome 8;21 translocation with associated hyperdiploidy in acute myeloid leukemia (FAB-M2). Pediatr Blood Cancer; 2008 Mar;50(3):651-4
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Complex chromosome 8;21 translocation with associated hyperdiploidy in acute myeloid leukemia (FAB-M2).
  • We present a case of acute myeloblastic leukemia (AML-M2) with a complex t(8;21) translocation and additional acquired chromosomes yielding a hyperdiploid karyotype.
  • The patient was treated according to our current protocol for AML.
  • He remains in complete remission +11 months from diagnosis.
  • Further follow-up of this patient and the analysis of a larger number of children are needed to define whether the gains of the specific extra chromosomes modify the good prognosis that t(8;21) confers to this subgroup of AML.
  • [MeSH-major] Aneuploidy. Chromosomes, Human, Pair 21 / ultrastructure. Chromosomes, Human, Pair 8 / ultrastructure. Core Binding Factor Alpha 2 Subunit / genetics. Leukemia, Myeloid, Acute / genetics. Oncogene Proteins, Fusion / genetics. Translocation, Genetic

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  • [Copyright] (c) 2007 Wiley-Liss, Inc.
  • (PMID = 17405156.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Case Reports; Comparative Study; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / AML1-ETO fusion protein, human; 0 / Core Binding Factor Alpha 2 Subunit; 0 / Oncogene Proteins, Fusion; 04079A1RDZ / Cytarabine; 6PLQ3CP4P3 / Etoposide; ZRP63D75JW / Idarubicin; ICE protocol 4
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100. Rubnitz JE, Lensing S, Razzouk BI, Pounds S, Pui CH, Ribeiro RC: Effect of race on outcome of white and black children with acute myeloid leukemia: the St. Jude experience. Pediatr Blood Cancer; 2007 Jan;48(1):10-5
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Effect of race on outcome of white and black children with acute myeloid leukemia: the St. Jude experience.
  • BACKGROUND: The association between race and outcome of treatment for childhood acute myeloid leukemia (AML) has not been adequately studied.
  • PROCEDURE: We compared the clinical characteristics, biological features, and outcomes between white and black children with AML who were treated on five consecutive clinical protocols (1980-2002) at St. Jude Children's Research Hospital.
  • However, on our most recent trial (AML-97), there was a trend towards inferior outcome among black patients: the 5-year survival estimates were 55.6% +/- 12.3% and 27.3% +/- 13.5% for whites and blacks, respectively.
  • CONCLUSIONS: Although we detected no differences in treatment outcome between white and black children with AML over the entire study period, black children appear to have worse outcomes than white children during more recent studies.
  • Improved treatment is needed for all children with AML.
  • [MeSH-major] African Americans. European Continental Ancestry Group. Leukemia, Myeloid, Acute / mortality
  • [MeSH-minor] Adolescent. Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Child. Child, Preschool. Clinical Trials as Topic. Disease-Free Survival. Female. Hospitals, Pediatric. Humans. Infant. Male. Remission Induction. Retrospective Studies. Survival Rate. Treatment Outcome

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  • [Copyright] (c) 2006 Wiley-Liss, Inc.
  • (PMID = 16642489.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / P30 CA-21765
  • [Publication-type] Comparative Study; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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