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66. 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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67. 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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68. 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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  • [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


69. 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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70. Miller JS, Soignier Y, Panoskaltsis-Mortari A, McNearney SA, Yun GH, Fautsch SK, McKenna D, Le C, Defor TE, Burns LJ, Orchard PJ, Blazar BR, Wagner JE, Slungaard A, Weisdorf DJ, Okazaki IJ, McGlave PB: Successful adoptive transfer and in vivo expansion of human haploidentical NK cells in patients with cancer. Blood; 2005 Apr 15;105(8):3051-7
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  • A higher intensity inpatient regimen of high-dose cyclophosphamide and fludarabine (Hi-Cy/Flu) was tested in patients with poor-prognosis acute myeloid leukemia (AML).
  • In contrast, infusions after the more intense Hi-Cy/Flu resulted in a marked rise in endogenous IL-15, expansion of donor NK cells, and induction of complete hematologic remission in 5 of 19 poor-prognosis patients with AML.
  • [MeSH-major] Adoptive Transfer. Immunotherapy / methods. Killer Cells, Natural / transplantation. Leukemia, Myeloid / therapy
  • [MeSH-minor] Acute Disease. Carcinoma, Renal Cell / immunology. Carcinoma, Renal Cell / therapy. Cell Division / immunology. Haploidy. Hodgkin Disease / immunology. Hodgkin Disease / therapy. Humans. Kidney Neoplasms / immunology. Kidney Neoplasms / therapy. Melanoma / immunology. Melanoma / therapy. Skin Neoplasms / immunology. Skin Neoplasms / therapy. Treatment Outcome

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  • (PMID = 15632206.001).
  • [ISSN] 0006-4971
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Grant] United States / NCRR NIH HHS / RR / M01-RR00400; United States / NCI NIH HHS / CA / P01-CA-65493; United States / NCI NIH HHS / CA / R01-CA-72669; United States / NHLBI NIH HHS / HL / R01-HL-55417
  • [Publication-type] Clinical Trial; Journal Article; Research Support, U.S. Gov't, P.H.S.
  • [Publication-country] United States
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71. 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


72. 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


73. Savage NM, Kota V, Manaloor EJ, Kulharya AS, Pierini V, Mecucci C, Ustun C: Acute leukemia with PICALM-MLLT10 fusion gene: diagnostic and treatment struggle. Cancer Genet Cytogenet; 2010 Oct 15;202(2):129-32
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  • [Title] Acute leukemia with PICALM-MLLT10 fusion gene: diagnostic and treatment struggle.
  • Patients with various hematologic malignancies, including acute lymphoblastic leukemia (ALL), acute myeloblastic leukemia (AML), diffuse histiocytic lymphoma, and granulocytic sarcoma, have sometimes been shown to carry the PICALM-MLLT10 fusion gene (alias CALM-AF10) by various cytogenetic methodologies.
  • (1) the fusion gene occurs very rarely, (2) the cases do not have a distinct myeloid or lymphoid morphology and cells often appear immature, (3) cases usually have a mixed T-cell and myeloid phenotype, and (4) cases often have a mixed clinical presentation (e.g., mediastinal mass in a patient with AML).
  • A 27-year-old woman was diagnosed with AML with the PICALM-MLLT10 fusion gene.
  • The patient was treated on an AML regimen and achieved a complete remission.
  • Furthermore, central nervous system involvement at diagnosis and relapse are reported in pediatric populations.
  • Routine acute leukemia fluorescence in situ hybridization panels do not include a probe for the PICALM-MLLT10 fusion gene, and therefore diagnosis can be made only when karyotyping is available; that delay can result in initial misdiagnosis and mistreatment.
  • The case report and literature review here (including discussion of the poor prognosis and of management, including CNS prophylaxis) are intended to raise awareness and to inform about PICALM-MLLT10 in acute leukemia.
  • [MeSH-major] Leukemia, Myeloid, Acute / diagnosis. Leukemia, Myeloid, Acute / therapy. Oncogene Proteins, Fusion / genetics

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  • [Copyright] Copyright © 2010 Elsevier Inc. All rights reserved.
  • (PMID = 20875875.001).
  • [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 / Oncogene Proteins, Fusion; 0 / PICALM-MLLT10 fusion protein, human
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4. 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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75. 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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  • [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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76. 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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77. 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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78. 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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79. Gallegos-Castorena S, Medina-Sanson A, Gonzalez-Ramella O, Sanchez-Zubieta F, Martínez-Avalos A: Improved treatment results in Mexican children with acute myeloid leukemia using a Medical Research Council (MRC)-acute myeloid leukemia 10 modified protocol. Leuk Lymphoma; 2009 Jul;50(7):1132-7
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  • [Title] Improved treatment results in Mexican children with acute myeloid leukemia using a Medical Research Council (MRC)-acute myeloid leukemia 10 modified protocol.
  • Protocol III (2003-2005) on six cycles MRC AML 10 modified.
  • Patients with de novo acute myeloid leukemia 0 to 18 years were included.
  • We compared remission rate, overall and event-free survival.
  • Remission rate for Protocol I was 52%, for II 50% and for III 92% (p = 0.0001).
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid, Acute / drug therapy
  • [MeSH-minor] Adult. Child. Disease-Free Survival. Female. Humans. Male. Medical Oncology / methods. Mexico. Recurrence. Remission Induction. Treatment Outcome

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  • (PMID = 19557634.001).
  • [ISSN] 1029-2403
  • [Journal-full-title] Leukemia & lymphoma
  • [ISO-abbreviation] Leuk. Lymphoma
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
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80. 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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81. 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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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [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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82. 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


83. 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


84. 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


85. 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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86. Children's Oncology Group, Aplenc R, Alonzo TA, Gerbing RB, Smith FO, Meshinchi S, Ross JA, Perentesis J, Woods WG, Lange BJ, Davies SM: Ethnicity and survival in childhood acute myeloid leukemia: a report from the Children's Oncology Group. Blood; 2006 Jul 1;108(1):74-80
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  • [Title] Ethnicity and survival in childhood acute myeloid leukemia: a report from the Children's Oncology Group.
  • We evaluated differences in outcome by ethnicity among children with acute myeloid leukemia (AML).
  • In conclusion, Hispanic and black children with AML have worse survival than white children.
  • Access to chemotherapy, differences in supportive care or leukemia phenotype, and reduced compliance are unlikely explanations for this difference because therapy was given intravenously according to CCG protocols.


87. 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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88. 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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89. 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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90. 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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91. 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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  • [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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92. 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
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Integrin alpha4beta1
  • [Other-IDs] NLM/ PMC2903318
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93. 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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94. 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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  • [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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95. 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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96. 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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97. Ünal E, Sahdev I: Use of gemtuzumab ozogamicin in the treatment of pediatric relapsed/ refractory Acute Myeloid Leukemia. Turk J Haematol; 2008 Mar 5;25(1):36-41
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  • [Title] Use of gemtuzumab ozogamicin in the treatment of pediatric relapsed/ refractory Acute Myeloid Leukemia.
  • [Transliterated title] Pediatrik relaps/refraktor akut myeloid lösemi tedavisinde gemtuzumab ozogamisin kullanımı.
  • Gemtuzumab ozogamicin (GO, MylotargTM) is an antibody-targeted chemotherapy agent that has been studied in acute myeloid leukemia (AML) at first relapse in adults.
  • There is limited experience in pediatric patients.
  • We report six patients with refractory/relapsed CD33+AML who were treated with GO on compassionate-use basis.
  • One patient attained remission.
  • One patient is still alive following hematopoietic stem cell transplantation (HSCT), and one patient died in remission.
  • GO should be used cautiously in chemotherapy-refractory AML pediatric patients due to the high incidence of VOD.

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  • (PMID = 27264148.001).
  • [ISSN] 1300-7777
  • [Journal-full-title] Turkish journal of haematology : official journal of Turkish Society of Haematology
  • [ISO-abbreviation] Turk J Haematol
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Turkey
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98. Yuan J, McDonough C, Kulharya A, Ramalingam P, Manaloor E: Isolated trisomy 10 in an infant with acute myeloid leukemia: a case report and review of literature. Int J Clin Exp Pathol; 2010;3(7):718-22
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  • [Title] Isolated trisomy 10 in an infant with acute myeloid leukemia: a case report and review of literature.
  • Trisomy 10 as the sole cytogenetic abnormality in AML is rare, with an incidence rate of < 0.5%.
  • It tends to affect the elderly and is extremely rare in pediatric patients.
  • The patient received induction chemotherapy and achieved complete clinical and hematologic remission at day 28.
  • Compared to the two other reported pediatric cases, our patient has some unique features such as much younger age and additional findings such as bilineage dysplasia and bone marrow fibrosis.
  • Both reported cases and our case were classified as AML-M2 indicating that this may be a common subtype in pediatric patients.
  • These findings suggest that isolated trisomy 10 may be associated with distinct clinicopathologic features in pediatric AML.
  • [MeSH-major] Chromosomes, Human, Pair 10 / genetics. Leukemia, Myeloid, Acute / genetics. Leukemia, Myeloid, Acute / pathology. Trisomy / genetics. Trisomy / pathology

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  • (PMID = 20830243.001).
  • [ISSN] 1936-2625
  • [Journal-full-title] International journal of clinical and experimental pathology
  • [ISO-abbreviation] Int J Clin Exp Pathol
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article; Review
  • [Publication-country] United States
  • [Other-IDs] NLM/ PMC2933392
  • [Keywords] NOTNLM ; CD13 / CD33 / CD34 / CD7 / Trisomy 10 / acute myeloid leukemia / infant / review
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99. 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


100. Tilak V, Sookmane DD, Gupta V, Shukla J: Myelodysplastic syndrome. Indian J Pediatr; 2008 Jul;75(7):729-32
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • 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






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