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1. Ballmaier M, Germeshausen M, Schulze H, Cherkaoui K, Lang S, Gaudig A, Krukemeier S, Eilers M, Strauss G, Welte K: c-mpl mutations are the cause of congenital amegakaryocytic thrombocytopenia. Blood; 2001 Jan 1;97(1):139-46
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  • Congenital amegakaryocytic thrombocytopenia (CAMT) is a rare disease presenting with isolated thrombocytopenia in infancy and developing into a pancytopenia in later childhood.
  • However, platelets and hematopoietic progenitor cells of patients with CAMT did not show any reactivity to TPO, as measured by testing TPO-synergism to adenosine diphosphate in platelet activation or by megakaryocyte colony assays.
  • The type of mutations correlated with the clinical course of the disease.
  • [MeSH-major] Megakaryocytes / pathology. Neoplasm Proteins. Proto-Oncogene Proteins / genetics. Receptors, Cytokine. Thrombocytopenia / etiology
  • [MeSH-minor] Adenosine Diphosphate / pharmacology. Blood Platelets / drug effects. Child. Child, Preschool. Colony-Forming Units Assay. DNA Mutational Analysis. Disease Progression. Female. Genotype. Hematopoietic Stem Cells / drug effects. Humans. Infant. Male. Mutation. Pancytopenia / etiology. Phosphorylation. Platelet Activation / drug effects. Prognosis. Receptors, Thrombopoietin. Signal Transduction. Thrombopoietin / blood. Thrombopoietin / pharmacology


2. Pui CH, Chessells JM, Camitta B, Baruchel A, Biondi A, Boyett JM, Carroll A, Eden OB, Evans WE, Gadner H, Harbott J, Harms DO, Harrison CJ, Harrison PL, Heerema N, Janka-Schaub G, Kamps W, Masera G, Pullen J, Raimondi SC, Richards S, Riehm H, Sallan S, Sather H, Shuster J, Silverman LB, Valsecchi MG, Vilmer E, Zhou Y, Gaynon PS, Schrappe M: Clinical heterogeneity in childhood acute lymphoblastic leukemia with 11q23 rearrangements. Leukemia; 2003 Apr;17(4):700-6
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  • [Title] Clinical heterogeneity in childhood acute lymphoblastic leukemia with 11q23 rearrangements.
  • Hematopoietic stem cell transplantation failed to improve outcome in either age group.
  • [MeSH-minor] Adolescent. Age Factors. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. B-Lymphocytes / pathology. Child. Child, Preschool. Chromosomes, Human, Pair 19 / ultrastructure. Chromosomes, Human, Pair 4 / ultrastructure. Chromosomes, Human, Pair 9 / ultrastructure. Cohort Studies. Combined Modality Therapy. DNA-Binding Proteins / genetics. Disease-Free Survival. Drug Resistance, Neoplasm. Europe / epidemiology. Female. Hematopoietic Stem Cell Transplantation. Histone-Lysine N-Methyltransferase. Humans. Infant. Leukocyte Count. Male. Myeloid-Lymphoid Leukemia Protein. Neoplastic Stem Cells / pathology. Oncogene Proteins, Fusion / genetics. Prednisone / administration & dosage. Prognosis. Proportional Hazards Models. Retrospective Studies. Risk Factors. T-Lymphocytes / pathology. Translocation, Genetic. Treatment Outcome. United States / epidemiology

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  • (PMID = 12682627.001).
  • [ISSN] 0887-6924
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA 31566; United States / NCI NIH HHS / CA / CA21765; United States / NCI NIH HHS / CA / CA29139; United States / NCI NIH HHS / CA / CA37379; United States / NCI NIH HHS / CA / CA51001; United States / NCI NIH HHS / CA / CA78824
  • [Publication-type] Comparative Study; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.; Research Support, U.S. Gov't, P.H.S.; Review
  • [Publication-country] England
  • [Chemical-registry-number] 0 / DNA-Binding Proteins; 0 / MLL protein, human; 0 / Oncogene Proteins, Fusion; 0 / Transcription Factors; 149025-06-9 / Myeloid-Lymphoid Leukemia Protein; EC 2.1.1.43 / Histone-Lysine N-Methyltransferase; VB0R961HZT / Prednisone
  • [Number-of-references] 41
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3. 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).
  • PATIENTS AND METHODS: Myeloblasts for drug resistance testing were obtained from the bone marrow either on diagnosis or at relapse, before the HSCT procedure and were tested by the MTT assay.
  • Despite being nondiscriminative, the combined ex vivo drug resistance profile to fludarabine, treosulfan and etoposide (FTE score) was the strongest prognostic factor by multivariate analysis.
  • 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

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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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4. Steinbach D, Wittig S, Cario G, Viehmann S, Mueller A, Gruhn B, Haefer R, Zintl F, Sauerbrey A: The multidrug resistance-associated protein 3 (MRP3) is associated with a poor outcome in childhood ALL and may account for the worse prognosis in male patients and T-cell immunophenotype. Blood; 2003 Dec 15;102(13):4493-8
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  • [Title] The multidrug resistance-associated protein 3 (MRP3) is associated with a poor outcome in childhood ALL and may account for the worse prognosis in male patients and T-cell immunophenotype.
  • The family of multidrug resistance-associated proteins (MRPs) belongs to the superfamily of adenosine triphosphate-binding-cassette (ABC) transporters, which have the ability to function as outward pumps for chemotherapeutic drugs and therefore might be involved in drug resistance.
  • Our results suggest that MRP3 is involved in drug resistance in childhood ALL.
  • [MeSH-major] Leukemia-Lymphoma, Adult T-Cell / blood. Multidrug Resistance-Associated Proteins / blood. Neoplasm Proteins / blood. Precursor Cell Lymphoblastic Leukemia-Lymphoma / blood
  • [MeSH-minor] Adolescent. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Case-Control Studies. Child. Child, Preschool. Drug Resistance, Multiple / genetics. Drug Resistance, Neoplasm / genetics. Female. Hematopoietic Stem Cells / metabolism. Humans. Immunophenotyping. Infant. Leukocytes, Mononuclear / metabolism. Male. Neoplastic Stem Cells / metabolism. Prognosis. Proportional Hazards Models. RNA, Messenger / blood. RNA, Neoplasm / blood. Reverse Transcriptase Polymerase Chain Reaction. Survival Analysis. Treatment Outcome

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  • (PMID = 12816874.001).
  • [ISSN] 0006-4971
  • [Journal-full-title] Blood
  • [ISO-abbreviation] Blood
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article; Multicenter Study
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Multidrug Resistance-Associated Proteins; 0 / Neoplasm Proteins; 0 / RNA, Messenger; 0 / RNA, Neoplasm; 0 / multidrug resistance-associated protein 3
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5. Pui CH: Genetic studies in acute lymphoblastic leukemia. Acta Paediatr Taiwan; 2000 Nov-Dec;41(6):303-7
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  • Studies to identify genetic polymorphisms with pharmacokinetic and pharmacodynamic significance promise to enhance the discovery of new drugs and to optimize chemotherapy.
  • Recent successes in directing therapy to specific genetic abnormalities demonstrate the feasibility of molecular medicine for the childhood leukemias, and predict the development of highly effective drug regimens with minimal toxicity against normal hematopoietic cells.
  • [MeSH-minor] Child. Chromosome Aberrations. Humans. Infant. Molecular Epidemiology. Neoplasm, Residual / diagnosis. Neoplasm, Residual / genetics. Pharmacogenetics. Philadelphia Chromosome. Prognosis

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  • (PMID = 11198935.001).
  • [ISSN] 1608-8115
  • [Journal-full-title] Acta paediatrica Taiwanica = Taiwan er ke yi xue hui za zhi
  • [ISO-abbreviation] Acta Paediatr Taiwan
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA-20180; United States / NCI NIH HHS / CA / CA21765; United States / NCI NIH HHS / CA / CA51001
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.; Review
  • [Publication-country] China (Republic : 1949- )
  • [Number-of-references] 54
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6. Landau H, Lamanna N: Clinical manifestations and treatment of newly diagnosed acute lymphoblastic leukemia in adults. Curr Hematol Malig Rep; 2006 Sep;1(3):171-9
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  • Adult acute lymphoblastic leukemia (ALL) is a heterogeneous disease with distinct biologic and prognostic subgroups.
  • The treatment of adults with ALL has evolved largely from the therapy developed for childhood ALL and, despite differences across regimens, can be broadly classified as including induction, consolidation, maintenance, and central nervous system prophylaxis.
  • [MeSH-minor] Adult. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Burkitt Lymphoma / classification. Burkitt Lymphoma / therapy. Central Nervous System / pathology. Child. Clinical Trials as Topic. Cranial Irradiation. Eye / pathology. Hematopoietic Stem Cell Transplantation. Humans. Immunophenotyping. Incidence. Leukemic Infiltration / drug therapy. Leukemic Infiltration / prevention & control. Leukemic Infiltration / radiotherapy. Lymphocyte Subsets / pathology. Male. Neoplasm, Residual. Prognosis. Remission Induction. Testis / pathology. Translocation, Genetic

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  • (PMID = 20425348.001).
  • [ISSN] 1558-822X
  • [Journal-full-title] Current hematologic malignancy reports
  • [ISO-abbreviation] Curr Hematol Malig Rep
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] United States
  • [Number-of-references] 60
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7. Hamilton VM, Norris C, Bunin N, Goldwein JW, Bunin GR, Lange B, Meadows AT: Cyclophosphamide-based, seven-drug hybrid and low-dose involved field radiation for the treatment of childhood and adolescent Hodgkin disease. J Pediatr Hematol Oncol; 2001 Feb;23(2):84-8
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  • [Title] Cyclophosphamide-based, seven-drug hybrid and low-dose involved field radiation for the treatment of childhood and adolescent Hodgkin disease.
  • In an attempt to reduce long-term toxicities while preserving excellent cure rates, we developed a combined-modality protocol using a modified seven-drug hybrid and low-dose (2,000 cGy) involved field radiation therapy (RT).
  • The hybrid used cumulative doses of alkylating agents and anthracyclines that were lower than those used in previous four-drug regimens and substituted a less leukemogenic agent, cyclophosphamide, for nitrogen mustard.
  • Treatment consisted of four cycles of therapy for stages I and IIA, six cycles for stages IIB and III, and eight cycles for stage IV.
  • CONCLUSIONS: This therapy has resulted in 5-year overall survival and disease-free survival rates similar to regimens using higher doses of alkylating agents, anthracyclines, and radiation.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Hodgkin Disease / drug therapy
  • [MeSH-minor] Adolescent. Bleomycin / administration & dosage. Bleomycin / adverse effects. Child. Combined Modality Therapy. Cyclophosphamide / administration & dosage. Cyclophosphamide / adverse effects. Disease-Free Survival. Doxorubicin / administration & dosage. Doxorubicin / adverse effects. Female. Follow-Up Studies. Hematopoietic Stem Cell Transplantation. Humans. Hypothyroidism / etiology. Male. Neoplasm Staging. Neoplasms, Second Primary / etiology. Neoplasms, Second Primary / prevention & control. Neutropenia / chemically induced. Prednisone / administration & dosage. Prednisone / adverse effects. Procarbazine / administration & dosage. Procarbazine / adverse effects. Puberty, Delayed / etiology. Radiotherapy Dosage. Radiotherapy, Adjuvant / adverse effects. Remission Induction. Survival Rate. Thrombocytopenia / chemically induced. Treatment Outcome. Vinblastine / administration & dosage. Vinblastine / adverse effects. Vincristine / administration & dosage. Vincristine / adverse effects

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  • [CommentIn] J Pediatr Hematol Oncol. 2001 Aug-Sep;23(6):368-9 [11563772.001]
  • (PMID = 11216711.001).
  • [ISSN] 1077-4114
  • [Journal-full-title] Journal of pediatric hematology/oncology
  • [ISO-abbreviation] J. Pediatr. Hematol. Oncol.
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 11056-06-7 / Bleomycin; 35S93Y190K / Procarbazine; 5J49Q6B70F / Vincristine; 5V9KLZ54CY / Vinblastine; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide; VB0R961HZT / Prednisone; CVPPABO protocol
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8. Pui CH, Schrappe M, Ribeiro RC, Niemeyer CM: Childhood and adolescent lymphoid and myeloid leukemia. Hematology Am Soc Hematol Educ Program; 2004;:118-45
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  • [Title] Childhood and adolescent lymphoid and myeloid leukemia.
  • Remarkable progress has been made in the past decade in the treatment and in the understanding of the biology of childhood lymphoid and myeloid leukemias.
  • With contemporary improved risk assessment, chemotherapy, hematopoietic stem cell transplantation and supportive care, approximately 80% of children with newly diagnosed acute lymphoblastic leukemia and 50% of those with myeloid neoplasm can be cured to date.
  • Ching-Hon Pui describes certain clinical and biologic features that still have prognostic and therapeutic relevance in the context of contemporary treatment programs.
  • He emphasizes that treatment failure in some patients is not due to intrinsic drug resistance of leukemic cells but is rather caused by suboptimal drug dosing due to host compliance, pharmacodynamics, and pharmacogenetics.
  • Martin Schrappe performs detailed analyses of the prognostic impact of presenting age, leukocyte count, sex, immunophenotype, genetic abnormality, early treatment response, and in vitro drug sensitivity/resistance in childhood acute lymphoblastic leukemia, based on the large database of the Berlin-Frankfurt-Münster consortium.
  • The finding of essentially identical gene expression profiling by DNA microarray in certain specific genetic subtypes of childhood and adult acute myeloid leukemia suggests a shared leukemogenesis.
  • In Section IV, Dr.
  • Charlotte Niemeyer describes a new classification of myelodysplastic and myeloproliferative diseases in childhood, which has greatly facilitated the diagnosis of myelodysplastic syndromes and juvenile myelomonocytic leukemia.
  • She then describes the various treatment approaches for both juvenile myelomonocytic leukemia and myelodysplastic syndromes in the US and Europe, emphasizing the differences between childhood and adult cases for the latter group of diseases.

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  • (PMID = 15561680.001).
  • [ISSN] 1520-4391
  • [Journal-full-title] Hematology. American Society of Hematology. Education Program
  • [ISO-abbreviation] Hematology Am Soc Hematol Educ Program
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / CA-21765; United States / NCI NIH HHS / CA / CA-36401; United States / NCI NIH HHS / CA / CA-51001; United States / NCI NIH HHS / CA / CA-60419; United States / NCI NIH HHS / CA / CA-71907; United States / NCI NIH HHS / CA / CA-71970; United States / NCI NIH HHS / CA / CA-78224; United States / NCI NIH HHS / CA / CA20180; United States / NIGMS NIH HHS / GM / GM-61374; United States / NIGMS NIH HHS / GM / GM-61393
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.; Research Support, U.S. Gov't, P.H.S.; Review
  • [Publication-country] United States
  • [Number-of-references] 157
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9. Stoneham S, Ashley S, Pinkerton CR, Wallace WH, Shankar AG, United Kingdom Children's Cancer Study Group: Outcome after autologous hemopoietic stem cell transplantation in relapsed or refractory childhood Hodgkin disease. J Pediatr Hematol Oncol; 2004 Nov;26(11):740-5
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  • [Title] Outcome after autologous hemopoietic stem cell transplantation in relapsed or refractory childhood Hodgkin disease.
  • [MeSH-major] Hematopoietic Stem Cell Transplantation / methods. Hodgkin Disease / therapy. Salvage Therapy / methods
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Drug Resistance, Neoplasm. Female. Humans. Male. Prognosis. Recurrence. Retrospective Studies. Survival Analysis. Transplantation, Autologous. Treatment Outcome

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  • [CommentIn] J Pediatr Hematol Oncol. 2006 Nov;28(11):772 [17114969.001]
  • (PMID = 15543009.001).
  • [ISSN] 1077-4114
  • [Journal-full-title] Journal of pediatric hematology/oncology
  • [ISO-abbreviation] J. Pediatr. Hematol. Oncol.
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article
  • [Publication-country] United States
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10. Rubnitz JE: Molecular diagnostics in the treatment of childhood acute lymphoblastic leukemia. J Biol Regul Homeost Agents; 2000 Jul-Sep;14(3):182-6
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  • [Title] Molecular diagnostics in the treatment of childhood acute lymphoblastic leukemia.
  • For example, the prognosis for patients with acute lymphoblastic leukemia whose leukemic cells express the TEL-AML1 fusion is favorable when they are treated on modem chemotherapy protocols, whereas patients whose leukemic lymphoblasts contain the MLL-AF4 or the BCR-ABL fusion sometimes require allogeneic hematopoietic stem cell transplantation for cure.
  • Molecular techniques are also used to detect minimal residual disease and genetic polymorphisms that are important in optimizing drug therapy.
  • [MeSH-minor] Child. Core Binding Factor Alpha 2 Subunit. Fusion Proteins, bcr-abl / genetics. Humans. Myeloid-Lymphoid Leukemia Protein. Neoplasm, Residual. Oncogene Proteins, Fusion / genetics. Pharmacogenetics

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  • (PMID = 11037050.001).
  • [ISSN] 0393-974X
  • [Journal-full-title] Journal of biological regulators and homeostatic agents
  • [ISO-abbreviation] J. Biol. Regul. Homeost. Agents
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA-20180; United States / NCI NIH HHS / CA / CA-21765
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.; Review
  • [Publication-country] ITALY
  • [Chemical-registry-number] 0 / Core Binding Factor Alpha 2 Subunit; 0 / MLL-AF4 fusion protein, human; 0 / Oncogene Proteins, Fusion; 0 / TEL-AML1 fusion protein; 149025-06-9 / Myeloid-Lymphoid Leukemia Protein; EC 2.7.10.2 / Fusion Proteins, bcr-abl
  • [Number-of-references] 55
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11. Ko RH, Ji L, Barnette P, Bostrom B, Hutchinson R, Raetz E, Seibel NL, Twist CJ, Eckroth E, Sposto R, Gaynon PS, Loh ML: Outcome of patients treated for relapsed or refractory acute lymphoblastic leukemia: a Therapeutic Advances in Childhood Leukemia Consortium study. J Clin Oncol; 2010 Feb 1;28(4):648-54
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  • [Title] Outcome of patients treated for relapsed or refractory acute lymphoblastic leukemia: a Therapeutic Advances in Childhood Leukemia Consortium study.
  • The Therapeutic Advances in Childhood Leukemia (TACL) Consortium was assembled to assess novel drugs for children with resistant leukemia.
  • We hypothesize that novel agents and combinations that fail to improve baseline complete remission rates in comparable populations are unlikely to contribute to better outcomes and should be abandoned.
  • [MeSH-major] Drug Resistance, Neoplasm. Neoplasm Recurrence, Local / mortality. Neoplasm Recurrence, Local / therapy. Precursor Cell Lymphoblastic Leukemia-Lymphoma / mortality. Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy. Salvage Therapy
  • [MeSH-minor] Adolescent. Adult. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Child. Child, Preschool. Cohort Studies. Combined Modality Therapy. Female. Follow-Up Studies. Hematopoietic Stem Cell Transplantation. Humans. Infant. Infant, Newborn. Male. Prognosis. Remission Induction. Retrospective Studies. Survival Rate. Treatment Outcome. Young Adult

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  • (PMID = 19841326.001).
  • [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 / 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
  • [Other-IDs] NLM/ PMC2815999
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12. Balwierz W, Pawińska K, Skoczeń S, Klekawka T, Strojny W, Niezgoda A: [Perspectives in the use of imatinib in the treatment of childhood cancers]. Przegl Lek; 2004;61 Suppl 2:95-9
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  • [Title] [Perspectives in the use of imatinib in the treatment of childhood cancers].
  • Introduction of novel diagnostic methods and multimodal therapy has resulted in about 70% probability of cure of childhood neoplasms.
  • The only chance of cure remains allogeneic hematopoietic stem cell transplantation, however availability of transplantation is still low as a limited number of donors is available.
  • With introduction of new drugs that selectively inhibit mechanisms of maturation and proliferation of cancer cells, new hope has arisen.
  • Currently, there are several ongoing studies assessing the efficacy of this novel drug in the therapy of brain tumors, neuroblastoma, lung and prostate cancer.
  • [MeSH-major] Antineoplastic Agents / therapeutic use. Hematopoietic Stem Cell Transplantation. Leukemia, Myelogenous, Chronic, BCR-ABL Positive / drug therapy. Piperazines / therapeutic use. Protein Kinase Inhibitors / therapeutic use. Pyrimidines / therapeutic use
  • [MeSH-minor] Benzamides. Brain Neoplasms / drug therapy. Child. Clinical Trials as Topic. Drug Resistance, Neoplasm. Female. Gastrointestinal Neoplasms / drug therapy. Humans. Imatinib Mesylate. Lung Neoplasms / drug therapy. Male. Neuroblastoma / drug therapy. Prostatic Neoplasms / drug therapy. Protein-Tyrosine Kinases / antagonists & inhibitors

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  • (PMID = 15686056.001).
  • [ISSN] 0033-2240
  • [Journal-full-title] Przegla̧d lekarski
  • [ISO-abbreviation] Prz. Lek.
  • [Language] pol
  • [Publication-type] English Abstract; Journal Article; Review
  • [Publication-country] Poland
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Benzamides; 0 / Piperazines; 0 / Protein Kinase Inhibitors; 0 / Pyrimidines; 8A1O1M485B / Imatinib Mesylate; EC 2.7.10.1 / Protein-Tyrosine Kinases
  • [Number-of-references] 36
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13. Tang JY, Pan C, Chen J, Xu M, Chen J, Xue HL, Gu LJ, Dong R, Ye H, Zhou M, Wang YP: [Comprehensive protocol for diagnosis and treatment of childhood neuroblastoma--results of 45 cases]. Zhonghua Er Ke Za Zhi; 2006 Oct;44(10):770-3
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  • [Title] [Comprehensive protocol for diagnosis and treatment of childhood neuroblastoma--results of 45 cases].
  • OBJECTIVE: The aim of the paper was to improve the prognosis of neuroblastoma (NB) stage III and IV in children through the comprehensive therapy including chemotherapy, delayed tumor resection, autologous stem cell transplantation (ASCT) and inducing differentiation and to analyze the factors affecting the prognosis.
  • Comprehensive protocol included accurate staging, delayed and/or second tumor resection for stage III and IV patients, chemotherapy of different intensity mainly composed of cell cycle nonspecific drugs and 13-cis-retinoid for inducing cell differentiation.
  • Nine cases had stage I, 1 case had stage II, 8 cases had III, 26 cases had stage IV and 1 case had stage IVs of the tumor.
  • Of the thirty-nine patients, 31 achieved complete remission (CR) and 8 partial remission (PR) after surgery and/or chemotherapy.
  • Statistical analysis showed that the age older than 18 months, and stage III and IV of the tumor were the factors predicting poor prognosis (P = 0.04 and 0.003, respectively).
  • Age older than 18 months, and stage III and IV were the factors suggesting poor prognosis.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Hematopoietic Stem Cell Transplantation. Neuroblastoma / diagnosis. Neuroblastoma / therapy. Transplantation, Autologous
  • [MeSH-minor] Age Factors. Chemotherapy, Adjuvant. Child. Child, Preschool. Female. Follow-Up Studies. Humans. Infant. Male. Neoplasm Recurrence, Local / prevention & control. Neoplasm Recurrence, Local / surgery. Neoplasm Recurrence, Local / therapy. Neoplasm Staging. Prognosis. Remission Induction / methods. Retrospective Studies. Severity of Illness Index. Survival Rate. Time Factors. Treatment Outcome


14. Tonini GP, Pistoia V: Molecularly guided therapy of neuroblastoma: a review of different approaches. Curr Pharm Des; 2006;12(18):2303-17
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  • Stage 4 NB represents approximately 50% of the cases and shows metastatic dissemination at onset; its prognosis is grim, with 20% of the patients surviving at 5 years from diagnosis in spite of aggressive chemotherapy with autologous hematopoietic stem cell support.
  • Although several efforts are still needed to reach a significant cure of patients with neuroblastoma, molecularly guided approaches have opened new ways to neuroblastoma treatment and can represent useful models for other cancers of either childhood or adulthood.
  • [MeSH-major] Angiogenesis Inhibitors / therapeutic use. Antineoplastic Agents / therapeutic use. Genetic Therapy. Immunotherapy. Neuroblastoma / therapy. Retinoids / therapeutic use
  • [MeSH-minor] Animals. Antibodies, Monoclonal / therapeutic use. Apoptosis / drug effects. Cell Differentiation / drug effects. Chromosome Deletion. Cytokines / therapeutic use. Gene Expression Profiling / methods. Gene Expression Regulation, Neoplastic / drug effects. Humans. Neoplasm, Residual. Neovascularization, Pathologic / drug therapy. Nuclear Proteins / genetics. Nuclear Proteins / metabolism. Oligonucleotides, Antisense / genetics. Oligonucleotides, Antisense / metabolism. Oncogene Proteins / genetics. Oncogene Proteins / metabolism

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  • (PMID = 16787256.001).
  • [ISSN] 1381-6128
  • [Journal-full-title] Current pharmaceutical design
  • [ISO-abbreviation] Curr. Pharm. Des.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Review
  • [Publication-country] Netherlands
  • [Chemical-registry-number] 0 / Angiogenesis Inhibitors; 0 / Antibodies, Monoclonal; 0 / Antineoplastic Agents; 0 / Cytokines; 0 / MYCN protein, human; 0 / Nuclear Proteins; 0 / Oligonucleotides, Antisense; 0 / Oncogene Proteins; 0 / Retinoids
  • [Number-of-references] 145
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15. Lock RB, Liem NL, Papa RA: Preclinical testing of antileukemic drugs using an in vivo model of systemic disease. Methods Mol Med; 2005;111:323-34
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  • [Title] Preclinical testing of antileukemic drugs using an in vivo model of systemic disease.
  • Experimental models that mimic the dissemination of ALL have been difficult to establish, principally due to the poor engraftment efficiency of normal and malignant human hematopoietic cells in various strains of immune-deficient mice.
  • In this chapter we outline the methodology to (1) establish continuous xenografts from primary childhood ALL biopsies in nonobese diabetic/SCID (NOD/SCID) mice and (2) utilize these xenograft models of systemic disease to test established and experimental drugs while monitoring leukemia progression in "real time" by serial monitoring of murine peripheral blood.
  • [MeSH-major] Antineoplastic Agents / pharmacology. Drug Screening Assays, Antitumor. Precursor Cell Lymphoblastic Leukemia-Lymphoma / drug therapy
  • [MeSH-minor] Animals. Biopsy. Disease Progression. Humans. In Vitro Techniques. Mice. Mice, Nude. Mice, SCID. Neoplasm Transplantation

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  • (PMID = 15911988.001).
  • [ISSN] 1543-1894
  • [Journal-full-title] Methods in molecular medicine
  • [ISO-abbreviation] Methods Mol. Med.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents
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16. Suttorp M, Millot F: Treatment of pediatric chronic myeloid leukemia in the year 2010: use of tyrosine kinase inhibitors and stem-cell transplantation. Hematology Am Soc Hematol Educ Program; 2010;2010:368-76
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  • Allogeneic hematopoietic stem cell transplantation (allo-SCT) remains the only proven cure for chronic myeloid leukemia (CML), a rare malignancy in childhood.
  • 2) pharmacokinetics in childhood;.
  • Because the characteristics of CML in children seem to overlap extensively with what is described in adult internal medicine, most answers and pediatric algorithms are adapted from the treatment of CML in adults.
  • The approach for young patients with suboptimal responses is unclear because data on the efficacy and safety of second-generation TKIs in childhood are almost entirely missing.


17. Gassas A, Doyle JJ, Weitzman S, Freedman MH, Hitzler JK, Sharathkumar A, Dror Y: A basic classification and a comprehensive examination of pediatric myeloproliferative syndromes. J Pediatr Hematol Oncol; 2005 Apr;27(4):192-6
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  • Based on the predominant proliferating cell lineage, the authors established a classification system for childhood MPS.
  • Secondary MPS was classified as non-clonal proliferation (eg, infections, drugs, toxins, autoimmune, non-hematologic neoplasm, and trauma), and these were excluded from the study.
  • Significant proportions of cases of childhood MPS (60%) were unique to the pediatric population and not seen in adults.
  • In contrast to adults, MPS in children is more frequently treated with hematopoietic stem cell transplantation (HSCT), the only available curative option for most of these diseases.
  • MPS in children is different from adult-type MPS in terms of biology, categories, classification, and prognosis.
  • [MeSH-minor] Adolescent. Bone Marrow / pathology. Cell Lineage. Cell Proliferation. Child. Child, Preschool. Cytogenetic Analysis. Female. Hematopoietic Stem Cell Transplantation. Humans. Infant. Male. Prognosis. Survival Rate

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  • [CommentIn] J Pediatr Hematol Oncol. 2006 Oct;28(10):700-1 [17023836.001]
  • (PMID = 15838389.001).
  • [ISSN] 1077-4114
  • [Journal-full-title] Journal of pediatric hematology/oncology
  • [ISO-abbreviation] J. Pediatr. Hematol. Oncol.
  • [Language] eng
  • [Publication-type] Meta-Analysis
  • [Publication-country] United States
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18. Pui CH, Pei D, Sandlund JT, Ribeiro RC, Rubnitz JE, Raimondi SC, Onciu M, Campana D, Kun LE, Jeha S, Cheng C, Howard SC, Metzger ML, Bhojwani D, Downing JR, Evans WE, Relling MV: Long-term results of St Jude Total Therapy Studies 11, 12, 13A, 13B, and 14 for childhood acute lymphoblastic leukemia. Leukemia; 2010 Feb;24(2):371-82
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  • [Title] Long-term results of St Jude Total Therapy Studies 11, 12, 13A, 13B, and 14 for childhood acute lymphoblastic leukemia.
  • Owing to concerns about therapy-related secondary myeloid leukemia and brain tumors, in our current trials we reserve the use of etoposide for patients with refractory or relapsed leukemia undergoing hematopoietic stem cell transplantation, and cranial irradiation for those with CNS relapse.

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  • (PMID = 20010620.001).
  • [ISSN] 1476-5551
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / R01 CA060419-13; United States / NIGMS NIH HHS / GM / U01 GM061393-06; United States / NIGMS NIH HHS / GM / U01 GM061393; United States / NCI NIH HHS / CA / R01 CA051001-15; United States / NCI NIH HHS / CA / R01 CA078224; United States / NCI NIH HHS / CA / R37 CA036401-19; United States / NCI NIH HHS / CA / R37 CA036401; United States / NCI NIH HHS / CA / CA36401; United States / NCI NIH HHS / CA / CA21765; United States / NCI NIH HHS / CA / CA78224; United States / NCI NIH HHS / CA / R01 CA060419; United States / NCI NIH HHS / CA / CA60419; United States / NIGMS NIH HHS / GM / GM61393; United States / NCI NIH HHS / CA / P30 CA021765; United States / NCI NIH HHS / CA / P30 CA021765-31; United States / NCI NIH HHS / CA / R01 CA078224-09; United States / NCI NIH HHS / CA / R01 CA051001; United States / NCI NIH HHS / CA / R01 CA036401; United States / NCI NIH HHS / CA / CA51001; United States / NCI NIH HHS / CA / U01 CA060419
  • [Publication-type] Clinical Trial; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Other-IDs] NLM/ NIHMS137362; NLM/ PMC2820159
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19. Gaynon PS: Childhood acute lymphoblastic leukaemia and relapse. Br J Haematol; 2005 Dec;131(5):579-87
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  • [Title] Childhood acute lymphoblastic leukaemia and relapse.
  • Acute lymphoblastic leukaemia (ALL) is the most common childhood cancer.
  • Insights into pharmacology may guide more effective use of existing agents.
  • Novel agents with activity against resistant lymphoblasts offer an appealing strategy.
  • However, most candidate agents fail, despite enthusiastic investigators, intriguing mechanisms of action and 'compelling' preclinical data.
  • Novel agents must, most likely, be integrated into multiagent combinations that provide a higher CR rate or better quality CR's than our conventional combinations in order to contribute substantially to cure.
  • [MeSH-major] Neoplasm, Residual / therapy. Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy. Salvage Therapy / methods
  • [MeSH-minor] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Child. Hematopoietic Stem Cell Transplantation. Humans

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  • (PMID = 16351633.001).
  • [ISSN] 0007-1048
  • [Journal-full-title] British journal of haematology
  • [ISO-abbreviation] Br. J. Haematol.
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] England
  • [Number-of-references] 90
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20. Schmitz NM, Hirt A, Aebi M, Leibundgut K: Limited redundancy in phosphorylation of retinoblastoma tumor suppressor protein by cyclin-dependent kinases in acute lymphoblastic leukemia. Am J Pathol; 2006 Sep;169(3):1074-9
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • Serine 612 phosphorylation by CDK4 also occurred in extracts of childhood acute lymphoblastic leukemia cells but not in extracts of mobilized CD34+ hemopoietic progenitor cells.
  • This phenomenon could contribute to the commitment of childhood acute lymphocytic leukemia cells to proliferate and explain their refractoriness to differentiation-inducing agents.
  • [MeSH-minor] Antigens, CD34. Cell Differentiation / drug effects. Cell Line, Tumor. Cell-Free System / metabolism. Drug Resistance, Neoplasm / drug effects. G1 Phase / drug effects. Hematopoietic Stem Cells / metabolism. Humans. Indoles / pharmacology. Multiprotein Complexes / metabolism. Phosphorylation / drug effects

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  • (PMID = 16936279.001).
  • [ISSN] 0002-9440
  • [Journal-full-title] The American journal of pathology
  • [ISO-abbreviation] Am. J. Pathol.
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antigens, CD34; 0 / Indoles; 0 / Multiprotein Complexes; 0 / Retinoblastoma-Like Protein p107; 114719-57-2 / fascaplysine; EC 2.7.11.22 / CDK2 protein, human; EC 2.7.11.22 / CDK4 protein, human; EC 2.7.11.22 / Cyclin-Dependent Kinase 2; EC 2.7.11.22 / Cyclin-Dependent Kinase 4
  • [Other-IDs] NLM/ PMC1698824
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21. McLeod HL, Krynetski EY, Relling MV, Evans WE: Genetic polymorphism of thiopurine methyltransferase and its clinical relevance for childhood acute lymphoblastic leukemia. Leukemia; 2000 Apr;14(4):567-72
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  • [Title] Genetic polymorphism of thiopurine methyltransferase and its clinical relevance for childhood acute lymphoblastic leukemia.
  • If treated with standard doses of thiopurines, TPMTdeficient patients accumulate excessive thioguanine nucleotides in hematopoietic tissues, leading to severe hematological toxicity that can be fatal.
  • However, TPMT-deficient patients can be successfully treated with a 10- to 15-fold lower dosage of these medications.
  • The impact of 6-mercaptopurine dose intensity is also being clarified as an important determinate of event-free survival in childhood leukemia.
  • [MeSH-major] 6-Mercaptopurine / pharmacokinetics. Antimetabolites, Antineoplastic / pharmacokinetics. Inactivation, Metabolic / genetics. Methyltransferases / genetics. Neoplasm Proteins / genetics. Polymorphism, Genetic. Precursor Cell Lymphoblastic Leukemia-Lymphoma / genetics
  • [MeSH-minor] African Continental Ancestry Group / genetics. Asia. Child. Child, Preschool. Codon / genetics. Disease-Free Survival. Drug Resistance, Neoplasm / genetics. England. Ethnic Groups / genetics. European Continental Ancestry Group / genetics. France. Gene Frequency. Genotype. Humans. India. Infant. Neoplasms, Second Primary / chemically induced. Point Mutation. Recombinant Fusion Proteins / genetics. Risk. Safety. Treatment Outcome


22. van Lochem EG, Wiegers YM, van den Beemd R, Hählen K, van Dongen JJ, Hooijkaas H: Regeneration pattern of precursor-B-cells in bone marrow of acute lymphoblastic leukemia patients depends on the type of preceding chemotherapy. Leukemia; 2000 Apr;14(4):688-95
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  • [Title] Regeneration pattern of precursor-B-cells in bone marrow of acute lymphoblastic leukemia patients depends on the type of preceding chemotherapy.
  • Over a period of 15 years, we studied the regeneration of CD10+, TdT+, and CD10+/TdT+ cells in BM of children with (CD10+) precursor-B-ALL during and after treatment according to three different treatment protocols of the Dutch Childhood Leukemia Study Group (DCLSG) which differed both in medication and time schedule.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / pharmacology. B-Lymphocytes / drug effects. Hematopoiesis / drug effects. Hematopoietic Stem Cells / drug effects. Precursor B-Cell Lymphoblastic Leukemia-Lymphoma / pathology
  • [MeSH-minor] Asparaginase / administration & dosage. Asparaginase / pharmacology. Biomarkers, Tumor / analysis. Cell Differentiation. Cell Division / drug effects. Child. DNA Nucleotidylexotransferase / analysis. Dexamethasone / administration & dosage. Dexamethasone / pharmacology. Drug Evaluation. Female. Flow Cytometry. Humans. Immunophenotyping. Male. Microscopy. Neoplasm Proteins / analysis. Neoplasm, Residual. Neoplastic Stem Cells / drug effects. Neoplastic Stem Cells / pathology. Neprilysin / analysis. Vincristine / administration & dosage. Vincristine / pharmacology

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  • (PMID = 10764156.001).
  • [ISSN] 0887-6924
  • [Journal-full-title] Leukemia
  • [ISO-abbreviation] Leukemia
  • [Language] eng
  • [Publication-type] Clinical Trial; Comparative Study; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
  • [Publication-country] ENGLAND
  • [Chemical-registry-number] 0 / Biomarkers, Tumor; 0 / Neoplasm Proteins; 5J49Q6B70F / Vincristine; 7S5I7G3JQL / Dexamethasone; EC 2.7.7.31 / DNA Nucleotidylexotransferase; EC 3.4.24.11 / Neprilysin; EC 3.5.1.1 / Asparaginase; ALL-VI protocol
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23. Sun Z, Wang Z, Zhu W, Liu H, Liu X, Liu Z, Wang N, Pan L, Wu S, Wu J: [Treatment of two case childhood acute lymphoblastic leukemia by HLA-mismatched unrelated umbilical cord blood transplantation]. Zhonghua Xue Ye Xue Za Zhi; 2002 Apr;23(4):198-201
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  • [Title] [Treatment of two case childhood acute lymphoblastic leukemia by HLA-mismatched unrelated umbilical cord blood transplantation].
  • OBJECTIVE: To explore the hematopoietic and immunologic reconstitution and transplantation-related complications of HLA one locus mismatched unrelated umbilical cord blood transplantation for the treatment of hematological malignancies.
  • RESULTS: The two recipients, ANC > 0.5 x 10(9)/L occurred at day 27 and day 17, BPC > 50 x 10(9)/L at day 53 and day 46, the peripheral blood counts normalization at day 60 and day 52, the immune function normalization at day 134 and day 122 and the DNA fingerprinting showing engraftment at day 19 and day 17, respectively.
  • The donor-recipient pair of case 1 was male to female, and the chromosome karyotype of recipients bone marrow and peripheral blood cells showed 100%, 46, XY cells at day 49.
  • The two recipients have survived for 353 days and 256 days.
  • CONCLUSION: The hematopoietic and immunologic reconstitution in umbilical cord blood transplantation were earlier and more durable.
  • [MeSH-major] Fetal Blood / immunology. HLA Antigens / immunology. Hematopoietic Stem Cell Transplantation. Mycophenolic Acid / analogs & derivatives. Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy
  • [MeSH-minor] Child, Preschool. Cyclosporine / therapeutic use. DNA Fingerprinting. DNA, Neoplasm / genetics. Female. Graft Survival / drug effects. Graft Survival / immunology. Graft vs Host Disease / immunology. Graft vs Host Disease / prevention & control. Histocompatibility Testing. Humans. Immunosuppressive Agents / therapeutic use. Infant. Male. Transplantation Conditioning


24. Tokimasa S, Ohta H, Sawada A, Matsuda Y, Kim JY, Nishiguchi S, Hara J, Takihara Y: Lack of the Polycomb-group gene rae28 causes maturation arrest at the early B-cell developmental stage. Exp Hematol; 2001 Jan;29(1):93-103
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  • Furthermore, the human RAE28 locus may provide a candidate gene causing the molecular pathogenesis of childhood B-cell precursor ALL.
  • [MeSH-major] B-Lymphocytes / cytology. Carrier Proteins. Hematopoiesis / genetics. Homeodomain Proteins / genetics. Immunologic Deficiency Syndromes / genetics. Neoplasm Proteins / genetics. Precursor B-Cell Lymphoblastic Leukemia-Lymphoma / genetics
  • [MeSH-minor] Animals. Burkitt Lymphoma / pathology. Cell Differentiation / genetics. Cell Transplantation. Child. Child, Preschool. Chimera. Chromosomes, Human, Pair 12 / genetics. Coculture Techniques. Crosses, Genetic. Female. Gene Deletion. Gene Expression Regulation, Leukemic. Genes, Reporter. Genotype. Green Fluorescent Proteins. Hematopoietic Stem Cells / drug effects. Humans. Interleukin-7 / pharmacology. Liver / cytology. Liver / embryology. Luminescent Proteins / genetics. Male. Mice. Mice, Inbred C57BL. Mice, Knockout. Mice, Transgenic. Polycomb Repressive Complex 1. RNA, Messenger / biosynthesis. RNA, Neoplasm / biosynthesis. Recombinant Fusion Proteins / genetics. Reverse Transcriptase Polymerase Chain Reaction. T-Lymphocytes / cytology. Thymus Gland / abnormalities. Thymus Gland / embryology. Tumor Cells, Cultured / drug effects

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  • (PMID = 11164110.001).
  • [ISSN] 0301-472X
  • [Journal-full-title] Experimental hematology
  • [ISO-abbreviation] Exp. Hematol.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] Netherlands
  • [Chemical-registry-number] 0 / Carrier Proteins; 0 / Homeodomain Proteins; 0 / Interleukin-7; 0 / Luminescent Proteins; 0 / Neoplasm Proteins; 0 / PHC1 protein, human; 0 / Phc1 protein, mouse; 0 / RNA, Messenger; 0 / RNA, Neoplasm; 0 / Recombinant Fusion Proteins; 147336-22-9 / Green Fluorescent Proteins; EC 6.3.2.19 / Polycomb Repressive Complex 1
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