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Items 1 to 58 of about 58
1. 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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2. Brethon B, Auvrignon A, Cayuela JM, Lapillonne H, Leverger G, Baruchel A: Molecular response in two children with relapsed acute myeloid leukemia treated with a combination of gemtuzumab ozogamicin and cytarabine. Haematologica; 2006 Mar;91(3):419-21
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  • [Title] Molecular response in two children with relapsed acute myeloid leukemia treated with a combination of gemtuzumab ozogamicin and cytarabine.
  • Phase I/II studies of gemtuzumab ozogamicin (GO) in pediatric refractory/relapsed acute myeloid leukemia (AML) have been reported.
  • We present the cases of two children with relapsed AML who were treated with GO plus cytarabine, leading to a decrease of minimal residual disease down to levels not previously obtained.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Leukemia, Myeloid, Acute / drug therapy. Neoplasm Recurrence, Local / prevention & control

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  • (PMID = 16531270.001).
  • [ISSN] 1592-8721
  • [Journal-full-title] Haematologica
  • [ISO-abbreviation] Haematologica
  • [Language] eng
  • [Publication-type] Comparative Study; Letter
  • [Publication-country] Italy
  • [Chemical-registry-number] 0 / Aminoglycosides; 0 / Antibodies, Monoclonal; 0 / Antibodies, Monoclonal, Humanized; 0 / gemtuzumab; 04079A1RDZ / Cytarabine
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3. Gesundheit B, Shapira MY, Resnick IB, Amar A, Kristt D, Dray L, Budowski E, Or R: Successful cell-mediated cytokine-activated immunotherapy for relapsed acute myeloid leukemia after hematopoietic stem cell transplantation. Am J Hematol; 2009 Mar;84(3):188-90
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  • [Title] Successful cell-mediated cytokine-activated immunotherapy for relapsed acute myeloid leukemia after hematopoietic stem cell transplantation.
  • Acute myeloid leukemia (AML) is an extremely aggressive disease with a high relapse rate even after allogeneic hematopoietic stem cell transplantation (HSCT).
  • We report the successful outcome of cell-mediated cytokine-activated immunotherapy in a high-risk pediatric AML patient who relapsed shortly after allogeneic HSCT.
  • Donor lymphocyte infusion along with interferon induced a graft-versus-leukemia effect, presenting as a reversible episode of graft-versus-host disease, which led to stable complete donor chimerism and total eradication of AML for over 24 months, at the time of this report.
  • [MeSH-major] Leukemia, Myeloid, Acute / immunology. Leukemia, Myeloid, Acute / therapy. Lymphocyte Transfusion. Neoplasm Recurrence, Local / immunology. Neoplasm Recurrence, Local / therapy
  • [MeSH-minor] Child. Graft vs Host Disease. Graft vs Leukemia Effect. Hematopoietic Stem Cell Transplantation. Humans. Immunologic Factors / administration & dosage. Immunotherapy. Interferon-alpha / administration & dosage. Male. Recombinant Proteins

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  • (PMID = 19105234.001).
  • [ISSN] 1096-8652
  • [Journal-full-title] American journal of hematology
  • [ISO-abbreviation] Am. J. Hematol.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Immunologic Factors; 0 / Interferon-alpha; 0 / Recombinant Proteins; 76543-88-9 / interferon alfa-2a
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4. O'Brien MM, Lacayo NJ, Lum BL, Kshirsagar S, Buck S, Ravindranath Y, Bernstein M, Weinstein H, Chang MN, Arceci RJ, Sikic BI, Dahl GV: Phase I study of valspodar (PSC-833) with mitoxantrone and etoposide in refractory and relapsed pediatric acute leukemia: a report from the Children's Oncology Group. Pediatr Blood Cancer; 2010 May;54(5):694-702
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  • [Title] Phase I study of valspodar (PSC-833) with mitoxantrone and etoposide in refractory and relapsed pediatric acute leukemia: a report from the Children's Oncology Group.
  • BACKGROUND: Valspodar, a non-immunosuppressive analog of cylosporine, is a potent P-glycoprotein (MDR1) inhibitor.
  • As MDR1-mediated efflux of chemotherapeutic agents from leukemic blasts may contribute to drug resistance, a phase 1 study of valspodar combined with mitoxantrone and etoposide in pediatric patients with relapsed or refractory leukemias was performed.
  • Three of 11 patients with acute lymphoblastic leukemia (ALL) had complete responses while no patient with acute myeloid leukemia (AML) had an objective response.

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  • (PMID = 20209646.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / R01 CA052168-12S1; United States / NCI NIH HHS / CA / U10 CA98413; United States / PHS HHS / / R01 52168; United States / NCI NIH HHS / CA / U10 CA098413; United States / NCRR NIH HHS / RR / M01 RR 00070; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / R01 CA052168; United States / NCRR NIH HHS / RR / M01 RR000070; United States / NCI NIH HHS / CA / U10 CA98543
  • [Publication-type] Clinical Trial, Phase I; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Cyclosporins; 0 / P-Glycoprotein; 121584-18-7 / valspodar; 6PLQ3CP4P3 / Etoposide; BZ114NVM5P / Mitoxantrone
  • [Other-IDs] NLM/ NIHMS155713; NLM/ PMC2838930
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5. Cloos J, Goemans BF, Hess CJ, van Oostveen JW, Waisfisz Q, Corthals S, de Lange D, Boeckx N, Hählen K, Reinhardt D, Creutzig U, Schuurhuis GJ, Zwaan ChM, Kaspers GJ: Stability and prognostic influence of FLT3 mutations in paired initial and relapsed AML samples. Leukemia; 2006 Jul;20(7):1217-20
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  • [Title] Stability and prognostic influence of FLT3 mutations in paired initial and relapsed AML samples.
  • In acute myeloid leukemia (AML), activating mutations in the fms-like tyrosine kinase 3 (FLT3) gene predict poor prognosis.
  • We determined FLT3 internal tandem duplications (FLT3/ITD) and D835 point mutations in paired initial and relapse samples from 80 pediatric and adult AML patients.
  • One D835 point mutation was found in an initial pediatric AML sample.
  • [MeSH-major] Leukemia, Myeloid, Acute / epidemiology. Leukemia, Myeloid, Acute / genetics. Point Mutation. fms-Like Tyrosine Kinase 3 / genetics
  • [MeSH-minor] Adolescent. Adult. Female. Genetic Markers. Genetic Predisposition to Disease / epidemiology. Humans. Leukemia, Erythroblastic, Acute / genetics. Leukemia, Megakaryoblastic, Acute / genetics. Leukemia, Monocytic, Acute / genetics. Leukemia, Myelomonocytic, Acute / genetics. Leukemia, Promyelocytic, Acute / genetics. Male. Neoplasm, Residual / epidemiology. Neoplasm, Residual / genetics. Prognosis. Recurrence. Risk Factors. Tandem Repeat Sequences

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  • (PMID = 16642044.001).
  • [ISSN] 0887-6924
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Genetic Markers; EC 2.7.10.1 / FLT3 protein, human; EC 2.7.10.1 / fms-Like Tyrosine Kinase 3
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6. Kaspers GJ, Reinhardt D, Fleischhack G, Armendariz H, Stark B, Zwaan CM, Zimmermann M, Creutzig U: Low efficacy of methotrexate in childhood acute myeloid leukemia (AML): single-agent therapeutic window study in relapsed AML. Pediatr Blood Cancer; 2006 Oct 15;47(5):539-42
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  • [Title] Low efficacy of methotrexate in childhood acute myeloid leukemia (AML): single-agent therapeutic window study in relapsed AML.
  • BACKGROUND: The efficacy in pediatric acute myeloid leukemia (AML) of single-agent methotrexate (MTX) at a higher dose than previously applied, 1,000 mg/m2, given as a theoretically beneficial 36-hr continuous infusion, is unknown, but may be beneficial based on preclinical data.
  • PROCEDURE: We performed a therapeutic window study in children with first relapsed AML treated in four different countries.
  • By that time, another four patients had been enrolled, of which one patient with a late relapsed AML FAB type M7 showed a good response.
  • CONCLUSIONS: This study shows that single-agent MTX in the applied regimen in pediatric relapsed AML has limited efficacy.
  • However, it also demonstrates the feasibility of an international and therapeutic window phase II study in pediatric relapsed AML.
  • [MeSH-major] Leukemia, Myeloid, Acute / drug therapy. Methotrexate / therapeutic use


7. Oda M, Isoyama K, Ito E, Inoue M, Tsuchida M, Kigasawa H, Kato K, Kato S: Survival after cord blood transplantation from unrelated donor as a second hematopoietic stem cell transplantation for recurrent pediatric acute myeloid leukemia. Int J Hematol; 2009 Apr;89(3):374-82
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  • [Title] Survival after cord blood transplantation from unrelated donor as a second hematopoietic stem cell transplantation for recurrent pediatric acute myeloid leukemia.
  • The Japan Cord Blood Bank Network (JCBBN) reports the treatment of 22 children with acute myeloid leukemia (AML) who received umbilical cord blood transplantation from unrelated donors (CBT) as their second hematopoietic stem cell transplantation (HSCT).
  • Second complete remission at second transplantation was favorable prognosis (58.3 +/- 18.6%, compared with 17.1 +/- 10.8% for the non-CR group.
  • In conclusion, CBT combined with RIC as second HSCT may be useful against a recurrence of AML in children after the initial HSCT.
  • [MeSH-major] Cord Blood Stem Cell Transplantation. Hematopoietic Stem Cell Transplantation. Leukemia, Myeloid, Acute / surgery. Tissue Donors


8. 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).
  • Twenty-nine children 1 to 16 years of age (relapsed disease, 19; refractory disease, 10) received gemtuzumab ozogamicin ranging from 6 to 9 mg/m2 per dose for 2 doses (separated by 2 weeks) infused over 2 hours.
  • Remissions were comparable in patients with refractory (30%) and relapsed (26%) disease.
  • Gemtuzumab ozogamicin was relatively well tolerated at 6 mg/m2 for 2 doses and was equally effective in patients with refractory and relapsed disease.
  • 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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9. Sander A, Zimmermann M, Dworzak M, Fleischhack G, von Neuhoff C, Reinhardt D, Kaspers GJ, Creutzig U: Consequent and intensified relapse therapy improved survival in pediatric AML: results of relapse treatment in 379 patients of three consecutive AML-BFM trials. Leukemia; 2010 Aug;24(8):1422-8
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  • [Title] Consequent and intensified relapse therapy improved survival in pediatric AML: results of relapse treatment in 379 patients of three consecutive AML-BFM trials.
  • Relapse remains the major cause of treatment failure in pediatric acute myeloid leukemia (AML).
  • We analyzed the clinical characteristics, treatment response to relapse treatment and overall survival (OS) of 379 children with AML relapse treated according to three consecutive frontline protocols of the AML-Berlin/Frankfurt/Muenster study group (AML-BFM-87/-93/-98).
  • Overall, one-third of children with relapsed AML can be cured today.
  • [MeSH-major] Leukemia, Myeloid, Acute / surgery

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  • (PMID = 20535146.001).
  • [ISSN] 1476-5551
  • [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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10. 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.
  • The frequent non-hematologic adverse events were gastrointestinal toxicities, such as vomiting, diarrhea and abdominal pain, as well as pyrexia and headache.
  • 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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11. Bonanomi S, Connor P, Webb D, Ancliff P, Amrolia P, Rao K, McCloskey D, Hemmatpour S, Goulden N, Veys P: Successful outcome of allo-SCT in high-risk pediatric AML using chemotherapy-only conditioning and post transplant immunotherapy. Bone Marrow Transplant; 2008 Aug;42(4):253-7
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  • [Title] Successful outcome of allo-SCT in high-risk pediatric AML using chemotherapy-only conditioning and post transplant immunotherapy.
  • We report successful outcome in 13 children (median age 2.2 years) with high-risk AML who received SCT from an unrelated (11) or identical sibling (2) donor after a preparative regimen consisting of BU, CY and melphalan.
  • There was no TRM, but three children relapsed and died 3, 4 and 17 months after SCT.
  • These encouraging early results warrant further studies in children with very high-risk AML.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid, Acute / drug therapy. Stem Cell Transplantation / methods. Transplantation Conditioning / methods

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  • [ErratumIn] Bone Marrow Transplant. 2008 Nov;42(9):635. Bonnanomi, S [corrected to Bonanomi, S]
  • (PMID = 18560412.001).
  • [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 / Antibodies, Monoclonal; 0 / Antibodies, Monoclonal, Humanized; 0 / Antibodies, Neoplasm; 3A189DH42V / alemtuzumab; 83HN0GTJ6D / Cyclosporine; 8N3DW7272P / Cyclophosphamide; G1LN9045DK / Busulfan; Q41OR9510P / Melphalan
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12. 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).
  • 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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13. 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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14. Tan RM, Quah TC, Aung L, Liang S, Kirk RC, Yeoh AE: Improved outcome in childhood acute myeloid leukemia in Singapore with the MRC AML 10 protocol. Pediatr Blood Cancer; 2007 Mar;48(3):262-7
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  • [Title] Improved outcome in childhood acute myeloid leukemia in Singapore with the MRC AML 10 protocol.
  • BACKGROUND: The introduction of the United Kingdom Medical Research Council's 10th AML trial (MRC AML 10) protocol incorporating high-dose anthracycline therapy has improved outcome of children with acute myeloid leukemia (AML).
  • In this study, we review the results of childhood AML therapy in a Singapore university hospital over the last 17 years emphasizing toxicity and outcome.
  • PROCEDURE: Retrospective analysis revealed 34 children with AML between 1988 and 2003.
  • From September 1996, all but one of 15 children received MRC AML 10 treatment.
  • RESULTS: At the time of analysis, 17 had died from disease, and 17 patients were alive among whom 2 had relapsed.
  • MRC AML 10-treated patients (n = 14) had significantly better 3-year overall, event-free, and disease-free survival (74% vs. 35%, 77% vs. 20%, 83% vs. 31%; P = 0.019, P = 0.002, and P = 0.010, respectively) and were likelier to achieve complete remission (CR) than non-MRC AML 10 patients (P = 0.102).
  • Among patients who achieved CR, MRC AML 10-treated patients were significantly more likely to achieve CR after only one cycle of chemotherapy (P = 0.016).
  • CONCLUSIONS: These findings suggest that MRC AML 10 treatment results in significantly superior survival, without excess toxicity.
  • Future studies should attempt to elucidate the relative importance of individual MRC AML 10 components and reduce the high cumulative anthracycline dose without compromising outcome.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid / drug therapy
  • [MeSH-minor] 6-Mercaptopurine / administration & dosage. 6-Mercaptopurine / adverse effects. Acute Disease. Amsacrine / administration & dosage. Amsacrine / adverse effects. Azacitidine / administration & dosage. Azacitidine / adverse effects. Child. Child, Preschool. Cytarabine / administration & dosage. Cytarabine / adverse effects. Daunorubicin / administration & dosage. Daunorubicin / adverse effects. Developing Countries. Disease-Free Survival. Drug Evaluation. Drug-Induced Liver Injury / etiology. Etoposide / administration & dosage. Etoposide / adverse effects. Female. Gastrointestinal Diseases / chemically induced. Heart Diseases / chemically induced. Hematologic Diseases / chemically induced. Humans. Infant. Infection / etiology. Kaplan-Meier Estimate. Male. Methotrexate / administration & dosage. Methotrexate / adverse effects. Mitoxantrone / administration & dosage. Mitoxantrone / adverse effects. Prednisone / administration & dosage. Prednisone / adverse effects. Remission Induction. Retrospective Studies. Singapore / epidemiology. Survival Analysis. Thioguanine / administration & dosage. Thioguanine / adverse effects. Treatment Outcome. Vincristine / administration & dosage. Vincristine / adverse effects

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  • [Copyright] (c) 2006 Wiley-Liss, Inc.
  • (PMID = 16602120.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 00DPD30SOY / Amsacrine; 04079A1RDZ / Cytarabine; 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; BZ114NVM5P / Mitoxantrone; E7WED276I5 / 6-Mercaptopurine; FTK8U1GZNX / Thioguanine; M801H13NRU / Azacitidine; VB0R961HZT / Prednisone; YL5FZ2Y5U1 / Methotrexate; ZS7284E0ZP / Daunorubicin; MRC AML 10 protocol; POG-8498 protocol
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15. Styczynski J, Wysocki M, Debski R, Czyzewski K, Kolodziej B, Rafinska B, Kubicka M, Koltan S, Koltan A, Pogorzala M, Kurylak A, Olszewska-Slonina D, Balwierz W, Juraszewska E, Wieczorek M, Olejnik I, Krawczuk-Rybak M, Kuzmicz M, Kowalczyk J, Stefaniak J, Badowska W, Sonta-Jakimczyk D, Szczepanski T, Matysiak M, Malinowska I, Stanczak E, Wachowiak J, Konatkowska B, Gil L, Balcerska A, Maciejka-Kapuscinska L: Predictive value of multidrug resistance proteins and cellular drug resistance in childhood relapsed acute lymphoblastic leukemia. J Cancer Res Clin Oncol; 2007 Nov;133(11):875-93
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  • [Title] Predictive value of multidrug resistance proteins and cellular drug resistance in childhood relapsed acute lymphoblastic leukemia.
  • PURPOSE: Cellular resistance in childhood acute leukemias might be related to profile and function of multidrug resistance proteins and apoptosis regulating proteins.
  • (2) correlation with ex vivo drug resistance, and (3) analysis of their prognostic impact on clinical outcome in childhood acute lymphoblastic (ALL) and acute myeloid (AML) leukemia.
  • METHODS: Total number of 787 children diagnosed for initial ALL (n = 527), relapsed ALL (n = 104), initial AML (n = 133) and relapsed AML (n = 23) were included into the study.
  • RESULTS: Both initial AML and relapsed ALL samples showed higher drug resistance than initial ALL samples.
  • No significant differences were found in drug resistance between initial and relapsed AML samples.
  • CONCLUSIONS: The phenomenon of cellular drug resistance in childhood acute leukemias is multifactorial and plays an important role in response to therapy.
  • Expression of MRP1, PGP and LRP proteins, as well as their co-expression play possible role in childhood relapsed ALL.
  • [MeSH-major] Drug Resistance, Multiple. Drug Resistance, Neoplasm. Multidrug Resistance-Associated Proteins / metabolism. P-Glycoprotein / metabolism. Precursor Cell Lymphoblastic Leukemia-Lymphoma / metabolism. Vault Ribonucleoprotein Particles / metabolism

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  • [ErratumIn] J Cancer Res Clin Oncol. 2007 Nov;133(11):895. Wachowiak, Jacek [added]; Konatkowska, Benigna [added]; Gil, Lidia [added]; Balcerska, Anna [added]; Maciejka-Kapuscinska, Lucyna [added]
  • (PMID = 17671794.001).
  • [ISSN] 0171-5216
  • [Journal-full-title] Journal of cancer research and clinical oncology
  • [ISO-abbreviation] J. Cancer Res. Clin. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] Germany
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Multidrug Resistance-Associated Proteins; 0 / P-Glycoprotein; 0 / Vault Ribonucleoprotein Particles; 0 / major vault protein
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16. Castellino SM, Alonzo TA, Buxton A, Gold S, Lange BJ, Woods WG: Outcomes in childhood AML in the absence of transplantation in first remission--Children's Cancer Group (CCG) studies 2891 and CCG 213. Pediatr Blood Cancer; 2008 Jan;50(1):9-16
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  • [Title] Outcomes in childhood AML in the absence of transplantation in first remission--Children's Cancer Group (CCG) studies 2891 and CCG 213.
  • BACKGROUND: The majority of childhood acute myeloid leukemia (AML) patients lack a matched-related bone marrow transplant (BMT) donor in first remission.
  • PROCEDURE: Disease-free survival (DFS), overall survival (OS), relapse-free survival (RFS), and post-relapse outcome were evaluated for children with de novo AML on CCG 213 and the standard timing (ST) and intensive timing (IT) induction arms of CCG 2891 who were randomized to (intent-to-treat, ITT) or who received (as-treated, AT) only chemotherapy intensification.
  • Overall 48% of patients on both trials relapsed and 19.1% of patients who relapsed on these trials survived.
  • Overall survival for childhood AML in the absence of BMT in CR1 is influenced by duration of CR1 and by race.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid, Acute / drug therapy

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  • [Copyright] (c) 2007 Wiley-Liss, Inc.
  • (PMID = 17252564.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA 13539; United States / NCI NIH HHS / CA / CA 98543
  • [Publication-type] Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural
  • [Publication-country] United States
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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.
  • 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.
  • One patient relapsed before autoSCT.
  • Three patients relapsed following autoSCT; none relapsed after alloSCT.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Leukemia, Myeloid, Acute / therapy. Stem Cell Transplantation

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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. 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

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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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19. Ustün C, Kalla A, Bollag RJ, Manaloo E, Kulharya A, Jillella A: Relapsed acute myelogenous leukemia occurring after 18 years with recurrent novel chromosomal abnormality t(18;22)(q23;q11.2). Cancer Genet Cytogenet; 2007 Sep;177(2):135-8
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  • [Title] Relapsed acute myelogenous leukemia occurring after 18 years with recurrent novel chromosomal abnormality t(18;22)(q23;q11.2).
  • At age 4, she was diagnosed with acute myelogenous leukemia (AML) with t(18;22)(q23;q11.2) and received chemotherapy until age 6 under a pediatric study protocol.
  • The patient was treated for relapsed AML and at writing had been disease-free for 9 months.
  • Translocation between chromosomes 18 and 22 has been reported in indolent lymphoproliferative disorders, but not in AML.
  • Although we do not know the precise molecular etiology of this leukemia, the uncommon presentation for AML and late relapse with the same chromosomal abnormality may indicate a causal relationship between this novel chromosomal abnormality and the AML.
  • [MeSH-major] Chromosome Aberrations. Chromosomes, Human, Pair 18 / genetics. Chromosomes, Human, Pair 22 / genetics. Leukemia, Myeloid, Acute / genetics. Neoplasm Recurrence, Local / genetics

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  • (PMID = 17854669.001).
  • [ISSN] 0165-4608
  • [Journal-full-title] Cancer genetics and cytogenetics
  • [ISO-abbreviation] Cancer Genet. Cytogenet.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
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20. 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.
  • [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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21. Angiolillo AL, Whitlock J, Chen Z, Krailo M, Reaman G, Children's Oncology Group: Phase II study of gemcitabine in children with relapsed acute lymphoblastic leukemia or acute myelogenous leukemia (ADVL0022): a Children's Oncology Group Report. Pediatr Blood Cancer; 2006 Feb;46(2):193-7
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  • [Title] Phase II study of gemcitabine in children with relapsed acute lymphoblastic leukemia or acute myelogenous leukemia (ADVL0022): a Children's Oncology Group Report.
  • BACKGROUND: To determine the response rate and toxicity to gemcitabine administered as 10 mg/m2/min x 360 min weekly for 3 weeks in children with relapsed acute lymphoblastic leukemia (ALL) or acute myelogenous leukemia (AML).
  • RESULTS: One of 20 evaluable patients with ALL and none of 10 evaluable patients with AML had complete responses to gemcitabine; there were no partial responses.
  • CONCLUSIONS: Gemcitabine at the dose and schedule in this trial was not effective for children with relapsed AML or ALL.
  • [MeSH-major] Antimetabolites, Antineoplastic / administration & dosage. Deoxycytidine / analogs & derivatives. Leukemia, Myeloid, Acute / prevention & control. Precursor Cell Lymphoblastic Leukemia-Lymphoma / prevention & control


22. 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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23. 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.
  • 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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24. Parikh SH, Mendizabal A, Martin PL, Prasad VK, Szabolcs P, Driscoll TA, Kurtzberg J: Unrelated donor umbilical cord blood transplantation in pediatric myelodysplastic syndrome: a single-center experience. Biol Blood Marrow Transplant; 2009 Aug;15(8):948-55
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  • [Title] Unrelated donor umbilical cord blood transplantation in pediatric myelodysplastic syndrome: a single-center experience.
  • Between 1995 and 2006, 23 pediatric patients with MDS were transplanted with unrelated donor umbilical cord blood (UUCB) at our center.
  • Patients with acute myelogenous leukemia (AML) were excluded.
  • Three patients developed acute GVHD (aGVHD) grades II-IV with a cumulative incidence at 100 days of 13% (95% CI 0.0%-27.1.0%).
  • Four patients relapsed with a cumulative incidence of relapse at 3 years of 13.0% (95% CI 0.0%-27.1%).
  • UUCB should be actively considered for pediatric MDS patients lacking matched related or unrelated adult donors.


25. Horton TM, Thompson PA, Berg SL, Adamson PC, Ingle AM, Dolan ME, Delaney SM, Hedge M, Weiss HL, Wu MF, Blaney SM, Children's Oncology Group Study: Phase I pharmacokinetic and pharmacodynamic study of temozolomide in pediatric patients with refractory or recurrent leukemia: a Children's Oncology Group Study. J Clin Oncol; 2007 Nov 1;25(31):4922-8
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  • [Title] Phase I pharmacokinetic and pharmacodynamic study of temozolomide in pediatric patients with refractory or recurrent leukemia: a Children's Oncology Group Study.
  • PURPOSE: To determine the tolerability, pharmacokinetics, and mechanisms of temozolomide resistance in children with relapsed or refractory leukemia.
  • Pretreatment leukemia cell O6-methylguanine-DNA methyltransferase (MGMT) activity, tumor and plasma MGMT promoter methylation, and microsatellite instability (MSI) were examined in 14 of 16 study patients and in tissue bank samples from children with acute leukemia not treated with temozolomide (MGMT, n = 67; MSI, n = 65).
  • RESULTS: Sixteen patients (nine female, seven male; acute lymphoblastic leukemia [ALL], n = 8; acute myeloid leukemia [AML], n = 8), median age 11 years (range, 1 to 19 years), received either 200 mg/m2/d (nine enrolled, three assessable for toxicity) or 260 mg/m2/d (seven enrolled, three assessable for toxicity) of temozolomide.
  • MGMT activity in leukemia cells was quite variable and was highest in patients with relapsed ALL.
  • CONCLUSION: Temozolomide was well tolerated at doses as high as 260 mg/m2/d for 5 days in children with relapsed or refractory leukemia.
  • Increased MGMT activity may account for the temozolomide resistance in children with relapsed leukemia.
  • Leukemia cell MGMT activity was higher in pediatric ALL than AML (P < .0001).
  • [MeSH-major] Antineoplastic Agents, Alkylating / pharmacokinetics. Dacarbazine / analogs & derivatives. Drug Resistance, Neoplasm / genetics. Leukemia / drug therapy. Neoplasm Recurrence, Local / drug therapy

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  • (PMID = 17971589.001).
  • [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 / K12CA90433; United States / NCI NIH HHS / CA / P30 CA14599; United States / NCI NIH HHS / CA / U01CA63187; United States / NCI NIH HHS / CA / UO1-CA97452
  • [Publication-type] Clinical Trial, Phase I; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents, Alkylating; 0 / Tumor Suppressor Proteins; 7GR28W0FJI / Dacarbazine; 85622-93-1 / temozolomide; EC 2.1.1.- / DNA Modification Methylases; EC 2.1.1.63 / MGMT protein, human; EC 6.5.1.- / DNA Repair Enzymes
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26. 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.
  • 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


27. 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%).
  • 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.
  • 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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28. 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.
  • Elevated levels of at least one gene were found prior to relapse in 7 out of 10 patients who relapsed.
  • 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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29. Zidan H, Lo S, Wiebe D, Talano J, Alemzadeh R: Severe hypercholesterolemia mediated by lipoprotein X in a pediatric patient with chronic graft-versus-host disease of the liver. Pediatr Blood Cancer; 2008 Jun;50(6):1280-1
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  • [Title] Severe hypercholesterolemia mediated by lipoprotein X in a pediatric patient with chronic graft-versus-host disease of the liver.
  • We describe a case of extreme hypercholesterolemia, mediated by lipoprotein X, in a 12-year-old Caucasian female who underwent an unrelated allogenic bone marrow transplant for relapsed acute myelocytic leukemia (AML).


30. 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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31. Styczynski J, Gil L, Derwich K, Wachowiak J, Balwierz W, Badowska W, Krawczuk-Rybak M, Matysiak M, Wieczorek M, Balcerska A, Sonta-Jakimczyk D, Stefaniak J, Kowalczyk J, Urasinski T, Sobol G, Komarnicki M, Wysocki M: Comparison of clofarabine activity in childhood and adult acute leukemia: individual tumor response study. Anticancer Res; 2009 May;29(5):1643-50
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  • [Title] Comparison of clofarabine activity in childhood and adult acute leukemia: individual tumor response study.
  • The aim of the study was the analysis of ex vivo activity of clofarabine and 14 other anticancer drugs in pediatric and adult acute lymphoblastic (ALL) and myeloid (AML) leukemia.
  • PATIENTS AND METHODS: The ex vivo drug resistance profile was analyzed in 282 patients, including 201 children with ALL de novo, 24 children with relapsed ALL, 25 children with AML de novo and 32 adults with AML.
  • Its activity in acute myeloid leukemia was independent of patient age.
  • No significant differences in drug resistance to clofarabine between pediatric age-based subgroups of ALL were detected, while it was observed for most of other drugs.
  • An activity of clofarabine in relapsed pediatric ALL patients was as good as in newly-diagnosed ones.
  • CONCLUSION: In comparison to childhood acute lymphoblastic leukemia, lack of differences in ex vivo activity gives rationale for use of clofarabine in refractory and relapsed pediatric and adult patients with acute myeloid leukemia.
  • [MeSH-major] Adenine Nucleotides / therapeutic use. Antineoplastic Agents / therapeutic use. Arabinonucleosides / therapeutic use. Leukemia / drug therapy
  • [MeSH-minor] Acute Disease. Adolescent. Adult. Child. Child, Preschool. Humans. Infant. Middle Aged. Treatment Outcome. Young Adult

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  • (PMID = 19443380.001).
  • [ISSN] 0250-7005
  • [Journal-full-title] Anticancer research
  • [ISO-abbreviation] Anticancer Res.
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] Greece
  • [Chemical-registry-number] 0 / Adenine Nucleotides; 0 / Antineoplastic Agents; 0 / Arabinonucleosides; 762RDY0Y2H / clofarabine
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32. Styczynski J, Wysocki M, Debski R, Czyzewski K, Balwierz W, Juraszewska E, Matysiak M, Malinowska I, Stanczak E, Sońta-Jakimczyk D, Szczepanski T, Wachowiak J, Konatkowska B, Balcerska A, Ploszynska A, Kowalczyk J, Stefaniak J, Badowska W, Wieczorek M, Olejnik I, Krawczuk-Rybak M, Kuzmicz M: In vitro sensitivity of leukemic cells to nucleoside derivatives in childhood acute leukemias: good activity in leukemic relapses. Neoplasma; 2005;52(1):74-8
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  • [Title] In vitro sensitivity of leukemic cells to nucleoside derivatives in childhood acute leukemias: good activity in leukemic relapses.
  • Nucleoside analogues such as fludarabine and cladribine are used in therapy of indolent lymphomas and leukemias in adults, while cytarabine is used mainly in protocols for acute leukemias.
  • The objective of the study was the analysis of in vitro cellular drug sensitivity in childhood acute lymphoblastic (ALL) and myeloid (AML) leukemia.
  • Isolated leukemic cells obtained from 264 patients, including 152 initial ALL, 45 relapsed ALL, 54 initial AML and 13 relapsed AML were tested for cytotoxicity for fludarabine, cladribine, and cytarabine by the MTT assay.
  • Samples of relapsed ALL and initial AML were more resistant than ALL de novo ones.
  • Unexpectedly, no differences were observed between initial and relapsed AML samples for all tested drugs, what suggests that nucleoside analogues are active drugs in relapsed AML, which is commonly regarded as a resistant disease.
  • In summary, tested nucleoside analogues presented relatively good activity against childhood leukemias at relapse stage.
  • [MeSH-major] Antineoplastic Agents / pharmacology. Cladribine / pharmacology. Cytarabine / pharmacology. Leukemia, Myeloid / pathology. Precursor Cell Lymphoblastic Leukemia-Lymphoma / pathology. Vidarabine / analogs & derivatives. Vidarabine / pharmacology

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  • (PMID = 15739031.001).
  • [ISSN] 0028-2685
  • [Journal-full-title] Neoplasma
  • [ISO-abbreviation] Neoplasma
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Slovakia
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 04079A1RDZ / Cytarabine; 47M74X9YT5 / Cladribine; FA2DM6879K / Vidarabine; P2K93U8740 / fludarabine
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33. Quezada G, Kopp L, Estey E, Wells RJ: All-trans-retinoic acid and arsenic trioxide as initial therapy for acute promyelocytic leukemia. Pediatr Blood Cancer; 2008 Jul;51(1):133-5
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  • [Title] All-trans-retinoic acid and arsenic trioxide as initial therapy for acute promyelocytic leukemia.
  • Acute promyelocytic leukemia (APL) is a rare subtype of acute myeloid leukemia (AML).
  • Treatment of pediatric APL is based on the combination of all-trans-retinoic acid (ATRA), an anthracycline and cytosine arabinoside.
  • Arsenic trioxide (ATO) has been studied in adults with newly diagnosed or relapsed APL with excellent response rates both when used as a single agent or in combination with ATRA or ATRA plus chemotherapy.
  • There is little data on combination therapy with ATRA and ATO in pediatric APL.
  • [MeSH-major] Arsenicals / therapeutic use. Leukemia, Promyelocytic, Acute / diagnosis. Leukemia, Promyelocytic, Acute / drug therapy. Oxides / therapeutic use. Tretinoin / therapeutic use

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  • [Copyright] (c) 2008 Wiley-Liss, Inc.
  • (PMID = 18293388.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Arsenicals; 0 / Oxides; 5688UTC01R / Tretinoin; S7V92P67HO / arsenic trioxide
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34. Xie XT, Jiang SY, Li BS, Yang LL: [Relationship between the expression of the genes encoding the key enzymes for cytarabine metabolism and the pharmacokinetics of cytarabine in the treatment of childhood acute leukemia with high-dose cytarabine]. Zhonghua Er Ke Za Zhi; 2008 Apr;46(4):276-80
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  • [Title] [Relationship between the expression of the genes encoding the key enzymes for cytarabine metabolism and the pharmacokinetics of cytarabine in the treatment of childhood acute leukemia with high-dose cytarabine].
  • OBJECTIVE: It has been reported that high-dose cytarabine (HD-AraC) was very effective for childhood hematological malignancies, especially for improving the long-term survival of high-risk acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), and T-cell lymphoid malignancies (T-ALL, T-cell non-Hodgkin's lymphoma).
  • This study aimed to evaluate the pharmacokinetics of HD-AraC for childhood hematological malignancies, and the relationship between the expression of the genes coding the key enzymes for Ara-C metabolism with the outcome of the patients.
  • METHODS: The drug levels of Ara-C in plasma and cerebrospinal fluid were detected with HPLC while HD-AraC was used, the expression of deoxycytidine kinase (dCK) and cytidine deaminase (CDA) mRNA in human leukemia cell lines and the bone marrow cells were investigated in 48 cases of childhood hematological malignancies with RT-PCR methods, and the relationship between the expression of these enzymes mRNA and the outcome of the patients was analyzed. RESULTS:.
  • (1) When HD-AraC was used, the plasma levels of Ara-C and Ara-U could be respectively about 50 times and 25 times higher than those obtained when the patients were treated with regular dose of Ara-C treatment, and the level of Ara-C in cerebrospinal fluid could reach about 10% of plasma level of Ara-C. (2) There were significantly different expressions of dCK mRNA in different childhood acute leukemia (AL) patients, which were markedly related to the chemotherapy results.
  • The expression of dCK in ALL was much higher than that in AML and relapsed AL cases.
  • There were no significant differences in expressions of dCK in T-ALL and B lineage ALL. (3) In vitro study found that the expressions of dCK and CDA mRNA did not change in leukemia cell lines incubated at different doses and times of Ara-C.
  • CONCLUSIONS: HD-AraC was a very effective protocol for childhood hematological malignancies for it could significantly elevate the plasma and cerebrospinal fluid drug levels.
  • Good long-term outcomes of the childhood T-ALL could be achieved as the B lineage ALL had been treated with HD-AraC regimen.
  • As the expression levels of dCK were much lower, it may be necessary for the treatment of AML with HD-AraC for consecutive three days.
  • [MeSH-major] Antimetabolites, Antineoplastic / pharmacokinetics. Cytarabine / pharmacokinetics. Leukemia / genetics. Leukemia / metabolism
  • [MeSH-minor] Child. Cytidine Deaminase / genetics. Deoxycytidine Kinase / genetics. Gene Expression. Humans. Leukemia, Myeloid, Acute / drug therapy. Leukemia, Myeloid, Acute / genetics. Leukemia, Myeloid, Acute / metabolism. Lymphoma, Non-Hodgkin / drug therapy. Lymphoma, Non-Hodgkin / genetics. Lymphoma, Non-Hodgkin / metabolism. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy. Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics. Precursor Cell Lymphoblastic Leukemia-Lymphoma / metabolism

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  • (PMID = 19099730.001).
  • [ISSN] 0578-1310
  • [Journal-full-title] Zhonghua er ke za zhi = Chinese journal of pediatrics
  • [ISO-abbreviation] Zhonghua Er Ke Za Zhi
  • [Language] chi
  • [Publication-type] English Abstract; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] China
  • [Chemical-registry-number] 0 / Antimetabolites, Antineoplastic; 04079A1RDZ / Cytarabine; EC 2.7.1.74 / Deoxycytidine Kinase; EC 3.5.4.5 / Cytidine Deaminase
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35. Yildirim I, Uçkan D, Cetin M, Tuncer M, Tezcan I: Isolated testicular and bone relapse in children with acute myeloblastic leukemia and chronic graft versus host disease after allogeneic BMT. Turk J Pediatr; 2007 Apr-Jun;49(2):206-9
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  • [Title] Isolated testicular and bone relapse in children with acute myeloblastic leukemia and chronic graft versus host disease after allogeneic BMT.
  • Isolated extramedullary relapse after allogeneic bone marrow transplantation (BMT) for acute myeloblastic leukemia (AML) is very unusual, particularly in patients with graft versus host disease (GVHD) known to be associated with decreased incidence of leukemic relapses.
  • Here we report two unusual post-transplant cases with AML: the first developed testicular relapse during the treatment of chronic GVHD (cGVHD) and bronchiolitis obliterans and the second relapsed as granulocytic sarcoma in the proximal tibia two years after BMT.
  • [MeSH-major] Bone Marrow Transplantation. Graft vs Host Disease / pathology. Leukemia, Myeloid, Acute / pathology. Leukemia, Myeloid, Acute / therapy

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  • (PMID = 17907524.001).
  • [ISSN] 0041-4301
  • [Journal-full-title] The Turkish journal of pediatrics
  • [ISO-abbreviation] Turk. J. Pediatr.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] Turkey
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36. Horton TM, Ames MM, Reid JM, Krailo MD, Pendergrass T, Mosher R, Reaman GH, Seibel NL, Children's Oncology Group: A Phase 1 and pharmacokinetic clinical trial of paclitaxel for the treatment of refractory leukemia in children: a Children's Oncology Group study. Pediatr Blood Cancer; 2008 Apr;50(4):788-92
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  • [Title] A Phase 1 and pharmacokinetic clinical trial of paclitaxel for the treatment of refractory leukemia in children: a Children's Oncology Group study.
  • BACKGROUND: This report summarizes a phase 1 study conducted by the Children's Cancer Group (CCG) to determine the maximum tolerated dose (MTD), dose-limiting toxicities (DLTs), pharmacokinetics, and anti-leukemia activity of paclitaxel in children with advanced stage leukemias.
  • RESULTS: Sixty-three patients (median 10 years) with refractory or relapsed leukemia (ALL) (n = 39), acute myeloid leukemia (AML) (n = 19), biphenotypic (n = 4), and JCML (n = 1)) were enrolled.
  • CONCLUSIONS: Paclitaxel was tolerated at 430 mg/m(2) every 21 days and at 182 mg/m(2)/dose weekly x 3 every 28 days in pediatric patients.

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  • [Copyright] (c) 2008 Wiley-Liss, Inc.
  • (PMID = 17668866.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / CA98543; United States / NCI NIH HHS / CA / K23 CA113775; United States / NCI NIH HHS / CA / U01 CA097452; United States / NCRR NIH HHS / RR / M01 RR000188; United States / NCI NIH HHS / CA / U01-CA97452; United States / NCI NIH HHS / CA / K23-CA113775; United States / NCI NIH HHS / CA / K12-CA90433; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCRR NIH HHS / RR / MO1 RR00108
  • [Publication-type] Clinical Trial, Phase I; Journal Article; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents, Phytogenic; P88XT4IS4D / Paclitaxel
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37. Mantadakis E, Danilatou V, Stiakaki E, Paterakis G, Papadhimitriou S, Kalmanti M: T-cell acute lymphoblastic leukemia relapsing as acute myelogenous leukemia. Pediatr Blood Cancer; 2007 Mar;48(3):354-7
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  • [Title] T-cell acute lymphoblastic leukemia relapsing as acute myelogenous leukemia.
  • We present the unusual case of a 16-year-old girl with T-cell acute lymphoblastic leukemia (ALL) with an early thymocyte immunophenotype without myeloid markers, who after 13 months of complete hematological remission relapsed as acute myelogenous leukemia (AML) with minimal differentiation and died of her disease.
  • Whether the AML represented a relapse with lineage switch of the original immature T-cell clone or a new secondary malignancy, could not be proven due to the absence of molecular or clonal markers.
  • This report suggests that a subset of CD7+ T-cell leukemias without mature T-cell antigens (CD4-, CD8-) are minimally differentiated and can relapse as AML.
  • [MeSH-major] Antigens, Differentiation, T-Lymphocyte / analysis. Antigens, Neoplasm / analysis. Leukemia, Myeloid / pathology. Leukemia-Lymphoma, Adult T-Cell / pathology. Neoplastic Stem Cells / pathology. T-Lymphocyte Subsets / pathology
  • [MeSH-minor] 6-Mercaptopurine / administration & dosage. Acute Disease. Adolescent. Antigens, CD7 / analysis. Antineoplastic Combined Chemotherapy Protocols / adverse effects. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Asparaginase / administration & dosage. Bone Marrow / pathology. Cell Differentiation. Cell Lineage. Core Binding Factor Alpha 2 Subunit / genetics. Cyclophosphamide / administration & dosage. Cytarabine / administration & dosage. Daunorubicin / administration & dosage. Dexamethasone / administration & dosage. Diagnosis, Differential. Doxorubicin / administration & dosage. Etoposide / administration & dosage. Etoposide / adverse effects. Fatal Outcome. Female. Gene Dosage. Histone-Lysine N-Methyltransferase. Humans. Immunophenotyping. Karyotyping. Methotrexate / administration & dosage. Myeloid-Lymphoid Leukemia Protein / genetics. Neoplasms, Second Primary / diagnosis. Proto-Oncogenes. Recurrence. Vincristine / administration & dosage

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  • [Copyright] (c) 2006 Wiley-Liss, Inc.
  • (PMID = 16206214.001).
  • [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 / Antigens, CD7; 0 / Antigens, Differentiation, T-Lymphocyte; 0 / Antigens, Neoplasm; 0 / Core Binding Factor Alpha 2 Subunit; 0 / MLL protein, human; 0 / RUNX1 protein, human; 04079A1RDZ / Cytarabine; 149025-06-9 / Myeloid-Lymphoid Leukemia Protein; 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 7S5I7G3JQL / Dexamethasone; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide; E7WED276I5 / 6-Mercaptopurine; EC 2.1.1.43 / Histone-Lysine N-Methyltransferase; EC 3.5.1.1 / Asparaginase; YL5FZ2Y5U1 / Methotrexate; ZS7284E0ZP / Daunorubicin
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38. Gregory J, Feusner J: Acute promyelocytic leukemia in childhood. Curr Oncol Rep; 2009 Nov;11(6):439-45
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  • [Title] Acute promyelocytic leukemia in childhood.
  • Acute promyelocytic leukemia (APL) is a relatively rare form of acute myelogenous leukemia (AML).
  • In the United States, APL in children constitutes only 5% to 10% of AML.
  • Molecularly, the disease is characterized by a fusion protein, promyelocytic leukemia (PML)-retinoic acid receptor (RAR)-alpha that results from a balanced reciprocal translocation between the PML gene on chromosome 15 and the RAR-alpha (RARA) gene on chromosome 17.
  • Advances in the treatment of APL have taken this form of AML from a disease with significant morbidity and mortality to one with an excellent outcome.
  • Recent trials have shown a role for arsenic trioxide in both newly diagnosed and relapsed APL.
  • [MeSH-major] Antineoplastic Agents / therapeutic use. Arsenicals / therapeutic use. Leukemia, Promyelocytic, Acute / drug therapy. Oxides / therapeutic use. Tretinoin / therapeutic use

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  • (PMID = 19840521.001).
  • [ISSN] 1534-6269
  • [Journal-full-title] Current oncology reports
  • [ISO-abbreviation] Curr Oncol Rep
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Anthracyclines; 0 / Antineoplastic Agents; 0 / Arsenicals; 0 / Oxides; 5688UTC01R / Tretinoin; S7V92P67HO / arsenic trioxide
  • [Number-of-references] 47
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39. 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.
  • A highly undifferentiated lymphomatous mass that relapsed in spite of intense chemotherapy and autologous transplant is reported.
  • 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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40. Franklin JL, Seibel NL, Krailo M, Fu C, Adamson PC, Reaman G, Children's Oncology Group: Phase 2 study of docetaxel in the treatment of childhood refractory acute leukemias: a Children's Oncology Group report. Pediatr Blood Cancer; 2008 Mar;50(3):533-6
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  • [Title] Phase 2 study of docetaxel in the treatment of childhood refractory acute leukemias: a Children's Oncology Group report.
  • BACKGROUND: To determine the response rate and toxicity of docetaxel when administered as a 60 mg/m(2) dose by 1 hr intravenous (IV) infusion weekly x 3 weeks in children with relapsed acute lymphoblastic leukemia (ALL) or acute myeloid leukemia (AML).
  • PROCEDURE: Patients who were under the age of 22-year-old at the time of the original ALL or AML diagnosis and in a second relapse were accrued from August 2002 to May 2005 for this Children's Oncology Group (COG) phase 2 study (ADVL0023).
  • Ten patients with ALL and two patients with AML were enrolled.
  • CONCLUSIONS: Docetaxel was not effective therapy for children with relapsed ALL at the dose and schedule tested.
  • [MeSH-major] Antineoplastic Agents, Phytogenic / therapeutic use. Leukemia / drug therapy. Salvage Therapy. Taxoids / therapeutic use
  • [MeSH-minor] Acute Disease. Adolescent. Adult. Bone Marrow Diseases / chemically induced. Child. Child, Preschool. Drug Administration Schedule. Female. Fever / etiology. Humans. Infant. Infusions, Intravenous. Leukemia, Myeloid / drug therapy. Male. Neutropenia / chemically induced. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy. Treatment Failure

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  • [Copyright] (c) 2007 Wiley-Liss, Inc.
  • (PMID = 17668867.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA 98543
  • [Publication-type] Clinical Trial, Phase II; Journal Article; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents, Phytogenic; 0 / Taxoids; 15H5577CQD / docetaxel
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41. 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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42. Spanaki A, Perdikogianni C, Linardakis E, Kalmanti M: Quantitative assessment of PRAME expression in diagnosis of childhood acute leukemia. Leuk Res; 2007 May;31(5):639-42
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  • [Title] Quantitative assessment of PRAME expression in diagnosis of childhood acute leukemia.
  • The purpose of this study was to investigate PRAME expression levels in children with acute leukemia with real-time PCR analysis.
  • Seventeen children with newly diagnosed or relapsed acute leukemia (11 ALL, 4 AML, 1 acute myeloblastic leukemia secondary to MDS, 1 ALL at relapse) and a control group of seven children were studied.
  • Overexpression of PRAME was found in 52.9% (3 AML, 6 ALL) of the patients studied.
  • The above findings indicate that PRAME expression in acute leukemia does not seem to be of prognostic significance, whereas it might represent a candidate marker for the monitoring of minimal residual disease.
  • [MeSH-major] Antigens, Neoplasm / genetics. Biomarkers, Tumor / genetics. Leukemia, Myeloid / genetics. Neoplasm, Residual / genetics. Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics
  • [MeSH-minor] Acute Disease. Adolescent. Child. Child, Preschool. Humans. Prognosis. RNA, Messenger. Reverse Transcriptase Polymerase Chain Reaction

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  • (PMID = 16860864.001).
  • [ISSN] 0145-2126
  • [Journal-full-title] Leukemia research
  • [ISO-abbreviation] Leuk. Res.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Antigens, Neoplasm; 0 / Biomarkers, Tumor; 0 / PRAME protein, human; 0 / RNA, Messenger
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43. Styczynski J, Wysocki M, Dluzniewska A, Juraszewska E, Balwierz W, Czyzewski K, Wieczorek M, Olejnik I, Krawczuk-Rybak M, Kuzmicz M, Kowalczyk J, Stefaniak J, Badowska W, Sonta-Jakimczyk D, Szczepanski T, Matysiak M, Stanczak E, Malinowska I, Wachowiak J, Konatkowska B, Gil L, Balcerska A, Kapuscinska L, Szczepanek J, Kolodziej B, Rafinska B, Kubicka M: Prognostic impact of combined fludarabine, treosulfan and mitoxantrone resistance profile in childhood acute myeloid leukemia. Anticancer Res; 2008 May-Jun;28(3B):1927-31
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  • [Title] Prognostic impact of combined fludarabine, treosulfan and mitoxantrone resistance profile in childhood acute myeloid leukemia.
  • BACKGROUND: The role of cellular drug resistance in childhood acute myeloid leukemia (AML) has not yet been established.
  • The aim of the study was the analysis of the clinical value of ex vivo drug resistance in pediatric AML.
  • PATIENTS AND METHODS: A cohort of 90 children with de novo AML were assayed for drug resistance profile by the 3-4,5-dimethylthiazol-2-yl-2,5-difenyl tetrazolium bromide (MTT) assay and prognostic model of in vitro drug sensitivity was analyzed.
  • RESULTS: Children who relapsed during follow-up showed higher in vitro resistance of leukemic blasts to most of the drugs tested, except for cytarabine, cladribine, vincristine, mercaptopurine and thioguanine.
  • A combined in vitro drug resistance profile to fludarabine, treosulfan and mitoxantrone (FTM score) was defined and it had an independent prognostic significance for disease free survival in pediatric AML.
  • CONCLUSION: The combined fludarabine, treosulfan and mitoxantrone resistance profile to possibly may be used for better stratification of children with AML or indicate the necessity for additional therapy.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Leukemia, Myeloid, Acute / drug therapy

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  • (PMID = 18630483.001).
  • [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] BZ114NVM5P / Mitoxantrone; CO61ER3EPI / treosulfan; FA2DM6879K / Vidarabine; G1LN9045DK / Busulfan; P2K93U8740 / fludarabine
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44. Tan PL, Wagner JE, Auerbach AD, Defor TE, Slungaard A, Macmillan ML: Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia patients undergoing genotypically identical donor hematopoietic cell transplantation. Pediatr Blood Cancer; 2006 May 1;46(5):630-6
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  • The patient with MDS relapsed with AML and a maternal donor recipient experienced secondary graft failure.
  • CONCLUSIONS: In summary, a FLU-based, non-irradiation approach is effective for FA patients with AA undergoing HLA-identical sibling donor HCT.

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  • (PMID = 16078221.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NHLBI NIH HHS / HL / R37 HL32987
  • [Publication-type] Comparative Study; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antilymphocyte Serum; 0 / Immunosuppressive Agents; 83HN0GTJ6D / Cyclosporine; 8N3DW7272P / Cyclophosphamide; FA2DM6879K / Vidarabine; P2K93U8740 / fludarabine; X4W7ZR7023 / Methylprednisolone
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45. Small D: Targeting FLT3 for the treatment of leukemia. Semin Hematol; 2008 Jul;45(3 Suppl 2):S17-21
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  • [Title] Targeting FLT3 for the treatment of leukemia.
  • It is frequently overexpressed in acute leukemias and is frequently mutated in acute myeloid leukemia (AML).
  • FLT3 internal tandem duplication (ITD) mutations in AML portend poor prognosis in both adult and pediatric patients.
  • Many of these are still in preclinical development, but several have entered clinical phase I and II trials as monotherapy in patients with relapsed AML.
  • Several combination trials are ongoing or planned in both relapsed and newly diagnosed FLT3-mutant AML patients.
  • Anti-FLT3 antibodies may also prove to be an excellent way of targeting FLT3 in AML and acute lymphocytic leukemia (ALL) by inhibiting signaling and through antibody-dependent cell-mediated cytotoxicity.

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  • (PMID = 18760705.001).
  • [ISSN] 0037-1963
  • [Journal-full-title] Seminars in hematology
  • [ISO-abbreviation] Semin. Hematol.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / R01 CA090668; United States / NCI NIH HHS / CA / P01 CA070970; United States / NCI NIH HHS / CA / R01 CA090668-01A1; United States / NCI NIH HHS / CA / CA090668-01A1; United States / NCI NIH HHS / CA / P01 CA070970-10A16432; United States / NCI NIH HHS / CA / CA070970-10A16432
  • [Publication-type] Journal Article; Review
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antibodies; 0 / Protein Kinase Inhibitors; EC 2.7.10.1 / FLT3 protein, human; EC 2.7.10.1 / fms-Like Tyrosine Kinase 3
  • [Number-of-references] 34
  • [Other-IDs] NLM/ NIHMS69701; NLM/ PMC2597087
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46. 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.
  • However, he relapsed after three cycles of chemotherapy.
  • 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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47. 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.
  • Six patients (23%) relapsed, four of them died.
  • Including the two relapsed patients who were salvaged by DLI and second transplantation, three-yr OS was 0.61 (95% CI; 0.41-0.78) with a median follow-up of three and a half yr (range 1.5-11.2 yr).
  • 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

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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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48. Korycka A, Lech-Marańda E, Robak T: Novel purine nucleoside analogues for hematological malignancies. Recent Pat Anticancer Drug Discov; 2008 Jun;3(2):123-36
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  • However, all these drugs have demonstrated promising activity in patients with relapsed and refractory acute lymphoblastic leukemia (ALL).
  • CAFdA was approved for the therapy of relapsed or refractory ALL in the third line of treatment.
  • It has proved promising in pediatric patients as well as in some patients who are able to proceed to allogenic hematopietic stem cell transplantation (HSCT).
  • Moreover, the drug exhibits an efficacy in acute myeloid leukemia (AML), blast crisis of chronic myelogenous leukemia (CML-BP) and myelodysplastic syndrome (MDS).
  • Forodesine is a purine nucleoside phosphorylase (PNP) inhibitor and it has shown activity in relapsed and refractory T- and B-cells leukemias as well as in cutaneous T-cell lymphoma (CTCL).
  • Great hopes are currently set on the use of these drugs in the treatment of lymphoid and myeloid malignancies in adult and in pediatric patients, however ongoing studies will help to define their role in the standard therapy.

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  • (PMID = 18537755.001).
  • [ISSN] 1574-8928
  • [Journal-full-title] Recent patents on anti-cancer drug discovery
  • [ISO-abbreviation] Recent Pat Anticancer Drug Discov
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Review
  • [Publication-country] United Arab Emirates
  • [Chemical-registry-number] 0 / Adenine Nucleotides; 0 / Antineoplastic Agents; 0 / Arabinonucleosides; 0 / Purine Nucleosides; 0 / Pyrimidinones; 426X066ELK / forodesine; 60158CV180 / nelarabine; 762RDY0Y2H / clofarabine
  • [Number-of-references] 102
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49. 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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50. Harned TM, Gaynon PS: Treating refractory leukemias in childhood, role of clofarabine. Ther Clin Risk Manag; 2008 Apr;4(2):327-36
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  • [Title] Treating refractory leukemias in childhood, role of clofarabine.
  • Approximately 4000 children and adolescents under the age of 20 years develop acute leukemia per year in the US.
  • Acute lymphoblastic leukemia (ALL) is the most common pediatric cancer.
  • Despite impressive improvements in outcome, relapsed ALL is the fourth most common pediatric malignancy.
  • Therapy for relapsed ALL remains unsatisfactory, and the majority of relapse patients still succumb to leukemia.
  • Between one-third and one-half of patients with acute myelogenous leukemia (AML) relapse, and no standard therapy is recognized for patients with relapsed and/or refractory AML.
  • Novel therapeutic agents are needed to improve the cure rate for relapsed ALL and AML.
  • Phase I and II single-agent trials in children have shown that clofarabine is safe and active in both myeloid and lymphoid relapsed/refractory acute leukemias.
  • Clofarabine has been approved by the FDA for pediatric patients with relapsed/refractory ALL after at least 2 prior therapeutic attempts.

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  • (PMID = 18728851.001).
  • [ISSN] 1176-6336
  • [Journal-full-title] Therapeutics and clinical risk management
  • [ISO-abbreviation] Ther Clin Risk Manag
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] New Zealand
  • [Other-IDs] NLM/ PMC2504075
  • [Keywords] NOTNLM ; childhood / clofarabine / leukemia / pediatric / refractory
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51. Chen SH, Hung IJ, Yang CP, Jaing TH, Fang EC, Yang SH, Tseng CK: Allogeneic related bone marrow transplantation in children--a single center experience. Acta Paediatr Taiwan; 2005 Nov-Dec;46(6):352-5
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  • We report our single-center experience with related allogeneic bone marrow transplantation (BMT) in pediatric recipients between April 1998 and December 2004.
  • Allogeneic bone marrow grafts from 19 donors (18 human leukocyte antigen (HLA)-matched sibling donors and 1 one antigen-mismatched related donor) were transplanted into patients aged 3-17 years (16 with leukemia and 3 with non-malignant disease).
  • Acute graft versus host disease (GVHD) grade II to IV developed in 8 patients (42.
  • The underlying diseases were acute lymphoblastic leukemia (ALL; 4 cases: 3 CR2, 1 Ph+), acute myeloid leukemia (AML; 2 cases: 1 CR2, 1 refractory), and juvenile chronic myelogenous leukemia (JCML; 1 case).
  • Relapsed leukemia at an extramedullary site occurred in 2 patients with ALL after BMT.
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Female. Humans. Leukemia / therapy. Male. Recurrence. Retrospective Studies. Transplantation, Homologous

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  • (PMID = 16640037.001).
  • [ISSN] 1608-8115
  • [Journal-full-title] Acta paediatrica Taiwanica = Taiwan er ke yi xue hui za zhi
  • [ISO-abbreviation] Acta Paediatr Taiwan
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] China (Republic : 1949- )
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52. 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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  • [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.
  • 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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53. van Be T, van Binh T, Binh N, Tuan T, Nghia H, Hien B: Current status of hematopoietic stem cell transplantations in Vietnam. Bone Marrow Transplant; 2008 Aug;42 Suppl 1:S146-S148
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  • Today, Vietnam has five SCT centers; in the north, there are three centers: 108 Military Hospital, Pediatric Institute and Blood transfusion and Hematology Institute; in the middle of Vietnam is Hue Hospital and in the south, the BT-H Hospital of Ho Chi Minh City.
  • With Allo-SCT we performed 19 cases with 3 procedures: BMT (4 cases), PBSC (6 cases) and cord blood transplantation (9 cases); patients were diagnosed with AML (n=7), ALL (n=1), CML (n=5) and beta-thalassemia (n=6).
  • Following transplantation, 7 patients (36.84%) relapsed, 12 (63.16%) remained alive and overall survival times: 6.81+/-1.35 years, disease-free survival times: 6.69+/-1.4 years (range 0.5-12 years).
  • With Auto-SCT: since November 1996, we have performed 33 cases of autologous PBSC transplantation consisting of without cryopreservation (24 cases) and with cryopreservation (9 cases); patients were diagnosed with AML in CR1 (n=21), ALL in CR1 (n=6), CML in CP (n=5) and non-Hodgkin's lymphoma in CR1 (n=1).
  • Following transplantation, 18 patients (54.50%) relapsed, 15 (45.45%) remained alive and overall survival times: 5.74+/-0.82 years and disease-free survival times: 5.48+/-0.92 years.

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  • (PMID = 18724291.001).
  • [ISSN] 0268-3369
  • [Journal-full-title] Bone marrow transplantation
  • [ISO-abbreviation] Bone Marrow Transplant.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
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54. Xu XJ, Tang YM, Song H, Yang SL, Shi SW, Wei J: Long-term outcome of childhood acute myeloid leukemia in a developing country: experience from a children's hospital in China. Leuk Lymphoma; 2010 Dec;51(12):2262-9
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  • [Title] Long-term outcome of childhood acute myeloid leukemia in a developing country: experience from a children's hospital in China.
  • Data on childhood acute myeloid leukemia (AML) in developing countries are limited.
  • Herein we report the outcome of childhood AML treated with modified NPCLC-AML97 in our institution from 1997 to 2005.
  • One hundred and eighty-five children with newly diagnosed AML were admitted.
  • APL was more curable than non-APL (7-year EFS: 63.5 ± 7.9% vs. 35.9 ± 6.3%, p = 0.005).
  • Thirty-one patients (25.2%) relapsed, but no central nervous system leukemia was observed.
  • Although the cure rate of childhood AML in China was low, the treatment outcome for patients who could adhere to the treatment protocol was satisfactory.
  • [MeSH-major] Developing Countries. Hospitals, Pediatric / statistics & numerical data. Leukemia, Myeloid, Acute / mortality. Leukemia, Myeloid, Acute / therapy


55. Kantarjian HM, Jeha S, Gandhi V, Wess M, Faderl S: Clofarabine: past, present, and future. Leuk Lymphoma; 2007 Oct;48(10):1922-30
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  • Intravenous clofarabine showed significant efficacy in pediatric leukemias (specifically, acute lymphoblastic leukemia (ALL)) and, in 2004, it was approved by the United States Food and Drug Administration (FDA) for the treatment of pediatric relapsed/refractory ALL after at least two regimens.
  • In adults, clofarabine has shown significant efficacy in hematologic malignancies including acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) alone and in combinations.
  • Ongoing and future studies will examine the use of clofarabine in elderly patients with AML for whom standard regimens are too toxic, and in MDS intravenous and oral forms of the drug.
  • [MeSH-minor] Aged. Animals. Child. Clinical Trials as Topic. DNA Damage. Drug Approval. Humans. Leukemia, Myeloid, Acute / drug therapy. Middle Aged. Models, Biological. Models, Chemical. Myelodysplastic Syndromes / drug therapy. Prognosis. Purines / chemistry

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  • (PMID = 17852710.001).
  • [ISSN] 1042-8194
  • [Journal-full-title] Leukemia & lymphoma
  • [ISO-abbreviation] Leuk. Lymphoma
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA57629
  • [Publication-type] Journal Article; Research Support, N.I.H., Extramural; Review
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Adenine Nucleotides; 0 / Arabinonucleosides; 0 / Purines; 762RDY0Y2H / clofarabine
  • [Number-of-references] 37
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56. Yang MH, Zhao MY, He YL, Wang MH, Wang Z, Xie M, Wu XS, Cao LZ: [Interaction of WAVE1 and genes involved in multiple drug resistance in children with acute myeloblastic leukemia]. Zhonghua Er Ke Za Zhi; 2010 Mar;48(3):175-9
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  • [Title] [Interaction of WAVE1 and genes involved in multiple drug resistance in children with acute myeloblastic leukemia].
  • OBJECTIVE: Multidrug resistance (MDR) is one of the primary causes of suboptimal outcomes in chemotherapy of children with acute myeloblastic leukemia (AML).
  • Expression and/or activity of P-glycoprotein (P-gp), multiple resistance-associated protein-1 (MRP1), lung-resistance related protein (LRP) and breast cancer resistance protein (BCRP) have been considered to be associated with unfavourable outcomes in pediatric AML patients.
  • In previous studies, we found WASP-family verprolin-homologous protein-1 (WAVE1) was involved in the MDR mechanisms in K562/A02 leukemia cells.
  • To investigate the expression of WAVE1, P-gp, MRP1, LRP/MVP and BCRP; and if WAVE1 is involved in MDR of human leukemia cell.
  • METHODS: WAVE1, P-gp, MRP1, LRP, BCRP mRNA and protein expression in bone marrow mononuclear cells (BMMCs) were measured by real-time fluorescence quantitative PCR (RQ-PCR) and Western blot in a cohort of 52 children with acute myeloblastic leukemia.
  • (1) The expression levels of WAVE1, P-gp, MRP, LRP and BCRP in refractory/relapsing group were much higher than that in complete continuous remission (CCR) group. (2) WAVE1 mRNA and protein expression in BMMCs of children were at higher levels when they were newly diagnosed or relapsed, compared with complete continuous remission. (3) The WAVE1 expression at mRNA and protein level in HL60/ADR cells was increased by about 353% and 95% respectively as compared with that in HL60 cells. (4) Overexpression of WAVE1 in HL60 cell lines upregulated the expression levels of MRP and BCRP (MRP mRNA and protein level were increased by about 16.54 times and 129% respectively, BCRP was increased by 4.93 times and 96%); whereas suppression of WAVE1 expression by RNA interference downregulated the expression levels of MRP1 and BCRP (MRP mRNA and protein level was only 11% and 43% of pre-disturbance respectively, BCRP was 14% and 71%).
  • CONCLUSIONS: Higher levels of WAVE1 in the BM indicate an unfavorable prognosis in children with AML.
  • WAVE1 is related to the development of AML and involved in the MDR mechanisms, and regulates the level of MRP1 and BCRP.
  • [MeSH-major] Drug Resistance, Neoplasm / genetics. Leukemia, Myeloid, Acute / genetics. Wiskott-Aldrich Syndrome Protein Family / genetics

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  • (PMID = 20426950.001).
  • [ISSN] 0578-1310
  • [Journal-full-title] Zhonghua er ke za zhi = Chinese journal of pediatrics
  • [ISO-abbreviation] Zhonghua Er Ke Za Zhi
  • [Language] chi
  • [Publication-type] English Abstract; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] China
  • [Chemical-registry-number] 0 / RNA, Small Interfering; 0 / Wiskott-Aldrich Syndrome Protein Family
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57. Locatelli F, Pende D, Maccario R, Mingari MC, Moretta A, Moretta L: Haploidentical hemopoietic stem cell transplantation for the treatment of high-risk leukemias: how NK cells make the difference. Clin Immunol; 2009 Nov;133(2):171-8
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  • T-cell-depleted hematopoietic stem cell (HSC) transplantation from an HLA-haploidentical relative (Haplo HSCT) may represent a suitable and effective transplant option, as it is capable of rescuing not only adult patients with high-risk acute myeloid leukemias (AML) but also children with relapsed acute lymphoblastic leukemia (ALL), as shown by the two representative cases presented in this study.
  • In Haplo HSCT, the anti-leukemia effect is mediated by "alloreactive" (i.e.
  • In view of the favorable clinical outcome of children with chemo-resistant ALL, Haplo HSCT from an NK-alloreactive relative could become a first option in these high-risk leukemia patients.
  • [MeSH-major] Haplotypes / immunology. Hematopoietic Stem Cell Transplantation / methods. Killer Cells, Natural / immunology. Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy

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  • (PMID = 19481979.001).
  • [ISSN] 1521-7035
  • [Journal-full-title] Clinical immunology (Orlando, Fla.)
  • [ISO-abbreviation] Clin. Immunol.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Histocompatibility Antigens Class I; 0 / Isoantigens; 0 / Receptors, KIR
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58. Svirnovski AI, Shman TV, Serhiyenka TF, Savitski VP, Smolnikova VV, Fedasenka UU: ABCB1 and ABCG2 proteins, their functional activity and gene expression in concert with drug sensitivity of leukemia cells. Hematology; 2009 Aug;14(4):204-12
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  • [Title] ABCB1 and ABCG2 proteins, their functional activity and gene expression in concert with drug sensitivity of leukemia cells.
  • Childhood acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML) cells and chronic lymphocytic leukemia (CLL) cells of adults were studied.
  • No differences between expression of P-gp and BCRP and genes in primary and relapsed acute leukemia (AL) cells as well as in de novo and treated CLL samples were established.
  • Doxorubicine, rubomycinum and L-asparaginase resistance correlates with P-gp overexpression and increased function in pediatric AL whereas vincristine resistance might be associated with P-gp protein expression in AL samples and impared P-gp function in CLL lymphocytes only.
  • A tendency for the decreased doxorubicin cytotoxic activity was shown in BCRP-overexpressing cells both in children and adults leukemia.
  • Multifactorial ANOVA showed that P-gp/MDR1 and BCRP as well as their function could not be used as unconditional and universal predictors of leukemia cell drug resistance in vitro.
  • These results suggest that studied MDR transporter-proteins have a limited role per se in vitro and admittedly in vivo drug resistance estimated in leukemia patients or it is not yet fully understood unless would not be studied in aggregate.
  • In any event, the expression and function studies of the proteins under investigation when singularly considered do not have a crucial significance for impact on drug resistance evaluation in all leukemia patients.
  • [MeSH-major] ATP-Binding Cassette Transporters / biosynthesis. Leukemia / drug therapy. Leukemia / metabolism. Neoplasm Proteins / biosynthesis. P-Glycoprotein / biosynthesis
  • [MeSH-minor] Acute Disease. Adolescent. Bone Marrow Cells / metabolism. Bone Marrow Cells / pathology. Child. Child, Preschool. Drug Resistance, Neoplasm. Gene Expression. Humans. Leukemia, Lymphocytic, Chronic, B-Cell / drug therapy. Leukemia, Lymphocytic, Chronic, B-Cell / genetics. Leukemia, Lymphocytic, Chronic, B-Cell / metabolism. Leukemia, Myeloid / drug therapy. Leukemia, Myeloid / genetics. Leukemia, Myeloid / metabolism. P-Glycoproteins. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy. Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics. Precursor Cell Lymphoblastic Leukemia-Lymphoma / metabolism. Tumor Cells, Cultured

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  • (PMID = 19635183.001).
  • [ISSN] 1607-8454
  • [Journal-full-title] Hematology (Amsterdam, Netherlands)
  • [ISO-abbreviation] Hematology
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / ABCB1 protein, human; 0 / ABCG2 protein, human; 0 / Neoplasm Proteins; 0 / P-Glycoprotein; 0 / P-Glycoproteins
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