Роль неларабина в лечении Т-клеточной острой лимфобластной лейкемии: обзор литературы и результаты собственных исследований
Роль неларабина в лечении Т-клеточной острой лимфобластной лейкемии: обзор литературы и результаты собственных исследований
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Аннотация
Цель исследования. Анализ собственного опыта применения неларабина при рефрактерных формах/рецидивах Т-клеточного острого лимфобластного лейкоза (Т-ОЛЛ) в зависимости от иммунофенотипа и линии терапии.
Материалы и методы. В анализ включены больные с рецидивами или рефрактерными формами Т-ОЛЛ в возрасте от 0 до 18 лет, получавшие терапию неларабином в составе терапевтического элемента R6. У всех больных проводился детальный иммунологический анализ лейкемических клеток с выделением иммуновариантов ТI, ТII, ТIII и ТIV. При использовании неларабина как первого терапевтического элемента пациентов относили к группе 1-й линии терапии, всех остальных – ко 2-й. Неларабин применялся в виде внутривенной инфузии в дозе 650 мг/м2 1–5-й день. Для всех больных планирование аллогенной трансплантации гемопоэтических стволовых клеток (алло-ТГСК) считали обязательным.
Результаты. С 2009 по 2017 г. 54 пациента с рефрактерными формами/рецидивами Т-ОЛЛ получили лечение неларабином. Бессобытийная выживаемость (БСВ) и общая выживаемость (ОВ) в течение 5 лет для всех больных составили 28%, кумулятивный риск рецидивов (КРР) – 27%. БСВ оказалась достоверно выше при применении неларабина в 1-й линии по сравнению с таковым во 2-й линии (34±8% vs 8±8%, p=0,05). У пациентов после алло-ТГСК БСВ составила 51±10%, ОВ – 50±10% и КРР – 39,1±9,5%. Наилучшие результаты достигнуты у пациентов с иммунофенотипом ТI. При лечении неларабином случаев смерти, связанной с токсичностью, тяжелых неврологических осложнений и прерываний курса терапии не зарегистрировано.
Заключение. Применение неларабина является эффективной стратегией лечения рецидивирующих и рефрактерных форм Т-ОЛЛ. Лучшие результаты получены у больных с вариантом ТI и при применении препарата в 1-й линии. Оптимальные режимы дозирования могут быть установлены в рамках рандомизированных клинических исследований.
Ключевые слова: неларабин, 9-β-D-арабинофуранозилгуанин (ara-G), рецидивы/рефрактерные формы Т-клеточного острого лимфобластного лейкоза, острый лимфобластный лейкоз, иммунофенотипирование Т-ОЛЛ, протоколы «Москва–Берлин».
Materials and methods. All the patients with relapsed or refractory T-ALL aged from 0 to 18 years who received treatment with nelarabine as a part of the therapeutic element R6 were included in the study. For all patients a detailed immunological analysis of leukemia cells with discrimination of immunological variants TI, TII, TIII or TIV was performed. Patients administered with nelarabine as a first therapeutic element were referred to the first-line therapy group, other patients were referred to the second-line therapy group. Nelarabine was administered as intravenous infusion at a dose of 650 mg/m2, on days 1-5. Allogeneic hematopoietic stem cells transplantation (allo-HSCT) was considered for all patients.
Results. From 2009 to 2017, 54 patients with refractory/relapsed T-ALL were treated with nelarabine. Five-year event-free survival (EFS) and overall survival (OS) was 28% for all patients, cumulative risk of relapse (CIR) was 27%. EFS was significantly higher in nelarabine first-line therapy group in comparison with second-line therapy group (34±8% vs 8±8%, p=0,05). In patients after allo-HSCT EFS, OS and CIR were 51±10%, 50±10% and 39,1±9,5% accordingly. The best results were achieved in patients with TI immunophenotype. No toxicity-related mortality as well as severe neurologic complications or discontinuation of therapy associated with use of nelarabine were reported.
Conclusion. The use of nelarabine is an effective strategy for the treatment of relapsed and refractory T-ALL. The best treatment outcomes were obtained in patients with TI immunophenotype and in the first-line therapy group. Optimal dosage regimens can be established during controlled clinical trials.
Keywords: nelarabine, 9-β-D-arabinofuranosylguanine (ara-G), relapsed/refractory T cell acute lymphoblastic leukemia, acute lymphoblastic leukemia, T-ALL immunophenotyping, Moscow-Berlin protocol.
Материалы и методы. В анализ включены больные с рецидивами или рефрактерными формами Т-ОЛЛ в возрасте от 0 до 18 лет, получавшие терапию неларабином в составе терапевтического элемента R6. У всех больных проводился детальный иммунологический анализ лейкемических клеток с выделением иммуновариантов ТI, ТII, ТIII и ТIV. При использовании неларабина как первого терапевтического элемента пациентов относили к группе 1-й линии терапии, всех остальных – ко 2-й. Неларабин применялся в виде внутривенной инфузии в дозе 650 мг/м2 1–5-й день. Для всех больных планирование аллогенной трансплантации гемопоэтических стволовых клеток (алло-ТГСК) считали обязательным.
Результаты. С 2009 по 2017 г. 54 пациента с рефрактерными формами/рецидивами Т-ОЛЛ получили лечение неларабином. Бессобытийная выживаемость (БСВ) и общая выживаемость (ОВ) в течение 5 лет для всех больных составили 28%, кумулятивный риск рецидивов (КРР) – 27%. БСВ оказалась достоверно выше при применении неларабина в 1-й линии по сравнению с таковым во 2-й линии (34±8% vs 8±8%, p=0,05). У пациентов после алло-ТГСК БСВ составила 51±10%, ОВ – 50±10% и КРР – 39,1±9,5%. Наилучшие результаты достигнуты у пациентов с иммунофенотипом ТI. При лечении неларабином случаев смерти, связанной с токсичностью, тяжелых неврологических осложнений и прерываний курса терапии не зарегистрировано.
Заключение. Применение неларабина является эффективной стратегией лечения рецидивирующих и рефрактерных форм Т-ОЛЛ. Лучшие результаты получены у больных с вариантом ТI и при применении препарата в 1-й линии. Оптимальные режимы дозирования могут быть установлены в рамках рандомизированных клинических исследований.
Ключевые слова: неларабин, 9-β-D-арабинофуранозилгуанин (ara-G), рецидивы/рефрактерные формы Т-клеточного острого лимфобластного лейкоза, острый лимфобластный лейкоз, иммунофенотипирование Т-ОЛЛ, протоколы «Москва–Берлин».
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Materials and methods. All the patients with relapsed or refractory T-ALL aged from 0 to 18 years who received treatment with nelarabine as a part of the therapeutic element R6 were included in the study. For all patients a detailed immunological analysis of leukemia cells with discrimination of immunological variants TI, TII, TIII or TIV was performed. Patients administered with nelarabine as a first therapeutic element were referred to the first-line therapy group, other patients were referred to the second-line therapy group. Nelarabine was administered as intravenous infusion at a dose of 650 mg/m2, on days 1-5. Allogeneic hematopoietic stem cells transplantation (allo-HSCT) was considered for all patients.
Results. From 2009 to 2017, 54 patients with refractory/relapsed T-ALL were treated with nelarabine. Five-year event-free survival (EFS) and overall survival (OS) was 28% for all patients, cumulative risk of relapse (CIR) was 27%. EFS was significantly higher in nelarabine first-line therapy group in comparison with second-line therapy group (34±8% vs 8±8%, p=0,05). In patients after allo-HSCT EFS, OS and CIR were 51±10%, 50±10% and 39,1±9,5% accordingly. The best results were achieved in patients with TI immunophenotype. No toxicity-related mortality as well as severe neurologic complications or discontinuation of therapy associated with use of nelarabine were reported.
Conclusion. The use of nelarabine is an effective strategy for the treatment of relapsed and refractory T-ALL. The best treatment outcomes were obtained in patients with TI immunophenotype and in the first-line therapy group. Optimal dosage regimens can be established during controlled clinical trials.
Keywords: nelarabine, 9-β-D-arabinofuranosylguanine (ara-G), relapsed/refractory T cell acute lymphoblastic leukemia, acute lymphoblastic leukemia, T-ALL immunophenotyping, Moscow-Berlin protocol.
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2. Garand R, Vannier JP, Bene MC, Faure G, Favre M, Bernard A. Comparison of outcome, clinical, laboratory, and immunological features in 164 children and adults with T-ALL. The Groupe d’Etude Immunologique des Leucemies. Leukemia. 1990; 4:739-44.
3. Bash RO, Crist WM, Shuster JJ, Link MP, Amylon M, Pullen J, Carroll AJ, Buchanan GR, Smith RG, Baer R. Clinical features and outcome of T-cell acute lymphoblastic leukemia in childhood with respect to alterations at the TAL1 locus: a Pediatric Oncology Group study. Blood. 1993; 81:2110-7.
4. Uckun FM, Sensel MG, Sun L, Steinherz PG, Trigg ME, Heerema NA, Sather HN, Reaman GH, Gaynon PS. Biology and treatment of childhood T-lineage acute lymphoblastic leukemia. Blood.1998; 91:735-46.
5. Pui CH, Relling MV, Evans WE. Role of pharmacogenomics and pharmacodynamics in the treatment of acute lymphoblastic leukaemia. Best Pract Res Clin Haematol. 2002; 15:741-56.
6. Reiter A, Schrappe M, Ludwig WD, Hiddemann W, Sauter S, Henze G, Zimmermann M, Lampert F, Havers W, Niethammer D, Odenwald E, Ritter J, Mann G, Welte K, Gadner H, Riehm H. Chemotherapy in 998 unselected childhood acute lymphoblastic leukemia patients. Results and conclusions of the multicenter trial ALL-BFM 86. Blood. 1994; 84:3122-33.
7. Schorin MA, Blattner S, Gelber RD, Tarbell NJ, Donnelly M, Dalton V, Cohen HJ, Sallan SE. Treatment of childhood acute lymphoblastic leukemia: results of Dana-Farber Cancer Institute/Children’s Hospital Acute Lymphoblastic Leukemia Consortium Protocol 85-01. J Clin Oncol. 1994; 12:740-7.
8. Cohen MH, Johnson JR, Massie T, Sridhara R, McGuinn WD Jr, Abraham S, Booth BP, Goheer MA, Morse D, Chen XH, Chidambaram N, Kenna L, Gobburu JV, Justice R, Pazdur R. Approval summary: nelarabine for the treatment of T-cell lymphoblastic leukemia/lymphoma. Clin Cancer Res. 2006; 12:5329-35.
9. Kurtzberg J. The long and winding road of the clinical development of Nelarabine. Leukemia&Lymphoma. 2007; 48(1): 1-2.
10. Giblett E, Ammann A, Wara D, Sandman R, Diamond L. Nucleoside-phosphorylase deficiency in a child with severely defective T-cell immunity and normal B-cell immunity. Lancet. 1975; 1:1010-1013.
11. Cohen A, Gudas LJ, Ammann AJ, Staal GE, Martin DW Jr. Deoxyguanosine triphosphate, a possible toxic metabolite in immunodeficiency associated with purine nucleotide phosphorylase deficiency. J Clin Invest. 1978; 61:1405 1410.
12. Gravatt LC, Chaffee S, Hebert ME, Halperin EC, Friedman HS, Kurtzberg J. Efficacy and toxicity of 9-Beta-Darabinofuranosylguanine (araG) as an agent to purge malignant T cells from murine bone marrow: application to an in vivo T-leukemia model. Leukemia. 1993; 7:1261-1267.
13. Hebert ME, Greenberg ML, Chaffee S, Gravatt L, Hershfield MS, Elion GB, Kurtzberg J. Pharmacologic purging of malignant T cells from human bone marrow using 9-Beta-Darabinofuranosylguanine. Transplantation. 1991; 52:634-640.
14. Momparler RL, Fisher GA. Mammalian deoxynucleoside kinase. I. Deoxycytidine kinase: purification, properties, and kinetic studies with cytosine arabinoside. J Biol Chem. 1968; 243:4298-304.
15. Rodriquez CO, Mitchell BS, Ayres M, Eriksson S, Gandhi V. Arabinosylguanine is phosphorylated by both cytoplasmic deoxycytidine kinase and mitochondrial deoxyguanosine kinase. Cancer Res. 2002; 62:3100-5.
16. Giblett ER, Ammann AJ, Wara DW, Sandman R, Diamond LK. Nucleoside-phosphorylase deficiency in a child with severely defective T-cell immunity and normal B-cell immunity. Lancet. 1975; 1:1010-3.
17. Mitchell BS, Mejias E, Daddona PE, Kelley WN. Purinogenic immunodeficiency diseases: selective toxicity of deoxyribonucleosides for T-cells. Proc Natl Acad Sci USA. 1978; 75:5011-4.
18. Osborne WR, Scott CR. The metabolism of deoxyguanosine and guanosine in human B and T-lymphoblasts. A role for deoxyguanosine kinase activity in the selective T-cell defect associated with purine nucleoside phosphorylase deficiency. Biochem J. 1983; 214:711-8.
19. Fairbanks LD, Taddeo A, Duley JA, Simmonds HA. Mechanisms of deoxyguanosine lymphotoxicity. Human thymocytes, but not peripheral blood lymphocytes accumulate deoxy-GTP in conditions simulating purine nucleoside phosphorylase deficiency. J Immunol. 1990; 144:485-91.
20. Cohen A, Lee JW, Gelfand EW. Selective toxicity of deoxyguanosine and arabinosylguanine for T leukemic cells. Blood. 1983; 61:660-6.
21. Lambe CU, Averett DR, Paff MT, Reardon JE, Wilson JG, Krenitsky TA. 2 Amino 6 methoxypurine arabinoside: an agent for T-cell malignancies. Cancer Res. 1995; 55:3352-6.
22. Prus KL, Averett DR, Zimmerman TP. Transport and metabolism of 9 β D arabinofuranosylguanine in a human T-lymphoblastoid cell line: nitrobenzylthioinosine-sensitive and -insensitive influx. Cancer Res. 1990; 50:1817-21.
23. Gandhi V, Mineishi S, Huang P, Chapman AJ, Yang Y, Chen F, Nowak B, Chubb S, Hertel LW, Plunkett W. Cytotoxicity, metabolism, and mechanisms of action of 2',2' difluorodeoxyguanosine in Chinese hamster ovary cells. Cancer Res. 1995; 55:1517-24.
24. Rodriguez CO, Gandhi V. Arabinosylguanine-induced apoptosis of T lymphoblastic cells: incorporation into DNA is a necessary step. Cancer Res. 1999; 59:4937-43.
25. Rodriquez CO, Stellrecht CM, Gandhi V. Mechanisms for T-cell selective cytotoxicity of arabinosylguanine. Blood. 2003; 102:1842-8.
26. Kisor DF. Nelarabine: a nucleoside analog with efficacy in T cell and other leukemias. Ann Pharmacother. 2005 Jun; 39 (6): 1056-63.
27. Gandhi V, Plunkett W, Rodriguez Jr CO. Compound GW506U78 in refractory hematologic malignancies: relation between cellular pharmacokinetics and clinical response. J Clin Oncol. 1998 Nov; 16 (11): 3607-15.
28. Gandhi V, Plunkett W, Weller S, Du M, Ayres M, Rodriguez CO Jr, Ramakrishna P, Rosner GL, Hodge JP, O'Brien S, Keating MJ. Evaluation of the combination of nelarabine and fludarabine in leukemias: clinical response, pharmacokinetics, and pharmacodynamics in leukemia cells. J Clin Oncol. 2001 Apr 15; 19 (8): 2142 52.
29. Ravandi F, Gandhi V. Novel purine nucleoside analogues for T cell-lineage acute lymphoblastic leukaemia and lymphoma. Expert Opin Investig Drugs. 2006 Dec; 15 (12): 1601-13.
30. European Medicines Agency. Atriance:scientific discussion [online]. Available from URL. http://www.emea.europa.eu/humandocs/Humans/EPAR/atriance/atriance.htm
31. Gandhi V, Plunkett W. Clofarabine and nelarabine: two new purine nucleoside analogs. Curr Opin Oncol. 2006 Nov; 18 (6): 584-90.
32. European Medicines Agency. Atriance (nelarabine): summary of product information [online]. Available from URL. http://www.emea.europa.eu/humandocs/PDFs/EPAR/atriance/H-752-PI-en.pdf [Accessed 2007 Dec 6]
33. Kurtzberg J, Ernst TJ, Keating MJ, Gandhi V, Hodge JP, Kisor DF, Lager JJ, Stephens C, Levin J, Krenitsky T, Elion G, Mitchell BS. Phase I study of 506U78 administered on a consecutive 5-day schedule in children and adults with refractory hematologic malignancies. J Clin Oncol. 2005; 23:3396-403.
34. DeAngelo DJ, Yu D, Johnson JL, Coutre SE, Stone RM, Stopeck AT, Gockerman JP, Mitchell BS, Appelbaum FR, Larson RA. Nelarabine induces complete remissions in adults with relapsed or refractory T-lineage acute lymphoblastic leukemia or lymphoblastic lymphoma: Cancer and Leukemia Group B study 19801. Blood. 2007 Jun 15; 109 (12): 5136-42.
35. Winter SS, Dunsmore KP, Devidas M, Eisenberg N, Asselin BL, Wood BL, Leonard Rn MS, Murphy J, Gastier-Foster JM, Carroll AJ, Heerema NA, Loh ML, Raetz EA, Winick NJ, Carroll WL, Hunger SP. Safe integration of nelarabine into intensive chemotherapy in newly diagnosed T-cell acute lymphoblastic leukemia: Children’s Oncology Group Study AALL0434. Pediatr Blood Cancer. 2015; 62(7):1176-1183.
36. Zwaan CM, Kowalczyk J, Schmitt C, Bielorai B, Russo MW, Woessner M, Ranganathan S, Leverger G. Safety and efficacy of nelarabine in children and young adults with relapsed or refractory T-lineage acute lymphoblastic leukaemia or T-lineage lymphoblastic lymphoma: results of a phase 4 study. British Journal of Haematology. 2017; 179: 284-293.
37. Seibel NL, Steinherz PG, Sather HN, Nachman JB, Delaat C, Ettinger LJ, Freyer DR, Mattano LA Jr, Hastings CA, Rubin CM, Bertolone K, Franklin JL, Heerema NA, Mitchell TL, Pyesmany AF, La MK, Edens C, Gaynon PS. Early postinduction intensification therapy improves survival for children and adolescents with high-risk acute lymphoblastic leukemia: a report from the Children’s Oncology Group. Blood. 2008; 111(5):2548-2555.
38. Kamps WA, Bokkerink JP, Hahlen K, Hermans J, Riehm H, Gadner H, Schrappe M, Slater R, van den Berg-de Ruiter E, Smets LA, de Vaan GA, Weening RS, van Weerden JF, van Wering ER, den der Does-van den Berg A. Intensive treatment of children with acute lymphoblastic leukemia according to ALL-BFM-86 without cranial radiotherapy: results of Dutch Childhood Leukemia Study Group Protocol ALL-7 (1988–1991). Blood. 1999; 94(4):1226-1236.
39. Salzer WL, Devidas M, Carroll WL Winick N, Pullen J, Hunger SP, Camitta BA. Long-term results of the pediatric oncology group studies for childhood acute lymphoblastic leukemia 1984 – 2001: a report from the children’s oncology group. Leukemia. 2010; 24(2):355-370.
40. Goldberg JM, Silverman LB, Levy DE, Dalton VK, Gelber RD, Lehmann L, Cohen HJ, Sallan SE, Asselin BL. Childhood T-cell acute lymphoblastic leukemia: the Dana-Farber Cancer Institute acute lymphoblastic leukemia consortium experience. J Clin Oncol. 2003; 21(19):3616-3622.
41. Nachman JB, Sather HN, Sensel MG, Trigg ME, Cherlow JM, Lukens JN, Wolff L, Uckun FM, Gaynon PS. Augmented post-induction therapy for children with high-risk acute lymphoblastic leukemia and a slow response to initial therapy. N Engl J Med. 1998; 338(23):1663-71.
42. Dunsmore KP, Devidas M, Linda SB, Borowitz MJ, Winick N, Hunger SP, Carroll WL, Camitta BM. Pilot study of nelarabine in combination with intensive chemotherapy in high-risk T-cell acute lymphoblastic leukemia: a report from the Children’s Oncology Group. J Clin Oncol. 2012; 30(22):2753-2759.
43. Loh M. Efficacy of nelarabine in newly diagnosed T-cell acute lymphoblastic leukemia: Children’s Oncology Group Study AALL0434. 29th Annual Meeting of the international BFM Study Group. 2018.
44. [Novikova IA, Verzhbitskaya TYu, Movchan LV, Tsaur GA, Belevtsev MV, Popov AM. Russian-belarusian multicenter group standard guidelines for childhood acute lymphoblastic leukemia flow cytometric diagnostics. Oncohematology. 2018;13(1):73-82. (In Russ.)].
45. Bene MC, Castoldi G, Knapp W, Ludwig WD, Matutes E, Orfao A, van't Veer MB. Proposals for the immunological classification of acute leukemias. European Group for the Immunological Characterization of Leukemias (EGIL). Leukemia. 1995; 9: 1783-1786.
46. Dworzak MN, Gaipa G, Ratei R, Veltroni M, Schumich A, Maglia O, Karawajew L, Benetello A, Pötschger U, Husak Z, Gadner H, Biondi A, Ludwig WD, Basso G. Standardization of flow cytometric minimal residual disease evaluation in acute lymphoblastic leukemia: multicentric assessment is feasible. Cytometry B Clin Cytom. 2008; 74: 331-340.
47. [Popov АM, Verzhbitskaya ТYu, Fechina LG, Shestopalov AV, Plyasunova SA. Acute Leukemias: Immunophenotypic Differences between Blast Cells and Their Nonneoplastic Analogues in Bone Marrow. Clinical oncohematology. 2016; 9(3):302-13. (In Russ.)].
48. Berg SL, Blaney SM, Devidas M, Lampkin TA, Murgo A, Bernstein M, Billett A, Kurtzberg J, Reaman G, Gaynon P, Whitlock J, Krailo M, Harris MB. Phase II study of nelarabine (compound 506U78) in children and young adults with refractory T-cell malignancies: a report from the Children’s Oncology Group. J Clin Oncol. 2005; 23:3376 82.
49. Kadia TM, Gandhi V. Nelarabine in the treatment of pediatric and adult patients with T cell acute lymphoblastic leukemia and lymphoma. Expert Rev Hematol. 2017 January; 10(1): 1-8.
50. Lonetti A, Cappellini A, Bertaina A, Locatelli F, Pession A, Buontempo F, Evangelisti Ca, Evangelisti Ce, Orsini E, Zambonin L, Neri LM, Martelli AM, Chiarini F. Improving nelarabine efficacy in T cell acute lymphoblastic leukemia by targeting aberrant PI3K/AKT/mTOR signaling pathway. J Hematol Oncol. 2016; 9: 114.
51. Herold N, Rudd SG, Sanjiv K, Kutzner J, Bladh J, Paulin CBJ, Helleday T, Henter JI, Schaller T. SAMHD1 protects cancer cells from various nucleoside-based antimetabolites. Cell Cycle. 2017; 16: 1029-1038.
52. Burley K, Wolf J, Raffoux E, Marks DI. Long-term survival following post-allograft relapse of T-cell acute lymphoblastic leukaemia: a novel approach using nelarabine and donor lymphocyte infusions. Bone Marrow Transplant. 2017; published online 31 Dec. https://doi.org/10. 1038/s41409-017-0038-8
2. Garand R, Vannier JP, Bene MC, Faure G, Favre M, Bernard A. Comparison of outcome, clinical, laboratory, and immunological features in 164 children and adults with T-ALL. The Groupe d’Etude Immunologique des Leucemies. Leukemia. 1990; 4:739-44.
3. Bash RO, Crist WM, Shuster JJ, Link MP, Amylon M, Pullen J, Carroll AJ, Buchanan GR, Smith RG, Baer R. Clinical features and outcome of T-cell acute lymphoblastic leukemia in childhood with respect to alterations at the TAL1 locus: a Pediatric Oncology Group study. Blood. 1993; 81:2110-7.
4. Uckun FM, Sensel MG, Sun L, Steinherz PG, Trigg ME, Heerema NA, Sather HN, Reaman GH, Gaynon PS. Biology and treatment of childhood T-lineage acute lymphoblastic leukemia. Blood.1998; 91:735-46.
5. Pui CH, Relling MV, Evans WE. Role of pharmacogenomics and pharmacodynamics in the treatment of acute lymphoblastic leukaemia. Best Pract Res Clin Haematol. 2002; 15:741-56.
6. Reiter A, Schrappe M, Ludwig WD, Hiddemann W, Sauter S, Henze G, Zimmermann M, Lampert F, Havers W, Niethammer D, Odenwald E, Ritter J, Mann G, Welte K, Gadner H, Riehm H. Chemotherapy in 998 unselected childhood acute lymphoblastic leukemia patients. Results and conclusions of the multicenter trial ALL-BFM 86. Blood. 1994; 84:3122-33.
7. Schorin MA, Blattner S, Gelber RD, Tarbell NJ, Donnelly M, Dalton V, Cohen HJ, Sallan SE. Treatment of childhood acute lymphoblastic leukemia: results of Dana-Farber Cancer Institute/Children’s Hospital Acute Lymphoblastic Leukemia Consortium Protocol 85-01. J Clin Oncol. 1994; 12:740-7.
8. Cohen MH, Johnson JR, Massie T, Sridhara R, McGuinn WD Jr, Abraham S, Booth BP, Goheer MA, Morse D, Chen XH, Chidambaram N, Kenna L, Gobburu JV, Justice R, Pazdur R. Approval summary: nelarabine for the treatment of T-cell lymphoblastic leukemia/lymphoma. Clin Cancer Res. 2006; 12:5329-35.
9. Kurtzberg J. The long and winding road of the clinical development of Nelarabine. Leukemia&Lymphoma. 2007; 48(1): 1-2.
10. Giblett E, Ammann A, Wara D, Sandman R, Diamond L. Nucleoside-phosphorylase deficiency in a child with severely defective T-cell immunity and normal B-cell immunity. Lancet. 1975; 1:1010-1013.
11. Cohen A, Gudas LJ, Ammann AJ, Staal GE, Martin DW Jr. Deoxyguanosine triphosphate, a possible toxic metabolite in immunodeficiency associated with purine nucleotide phosphorylase deficiency. J Clin Invest. 1978; 61:1405 1410.
12. Gravatt LC, Chaffee S, Hebert ME, Halperin EC, Friedman HS, Kurtzberg J. Efficacy and toxicity of 9-Beta-Darabinofuranosylguanine (araG) as an agent to purge malignant T cells from murine bone marrow: application to an in vivo T-leukemia model. Leukemia. 1993; 7:1261-1267.
13. Hebert ME, Greenberg ML, Chaffee S, Gravatt L, Hershfield MS, Elion GB, Kurtzberg J. Pharmacologic purging of malignant T cells from human bone marrow using 9-Beta-Darabinofuranosylguanine. Transplantation. 1991; 52:634-640.
14. Momparler RL, Fisher GA. Mammalian deoxynucleoside kinase. I. Deoxycytidine kinase: purification, properties, and kinetic studies with cytosine arabinoside. J Biol Chem. 1968; 243:4298-304.
15. Rodriquez CO, Mitchell BS, Ayres M, Eriksson S, Gandhi V. Arabinosylguanine is phosphorylated by both cytoplasmic deoxycytidine kinase and mitochondrial deoxyguanosine kinase. Cancer Res. 2002; 62:3100-5.
16. Giblett ER, Ammann AJ, Wara DW, Sandman R, Diamond LK. Nucleoside-phosphorylase deficiency in a child with severely defective T-cell immunity and normal B-cell immunity. Lancet. 1975; 1:1010-3.
17. Mitchell BS, Mejias E, Daddona PE, Kelley WN. Purinogenic immunodeficiency diseases: selective toxicity of deoxyribonucleosides for T-cells. Proc Natl Acad Sci USA. 1978; 75:5011-4.
18. Osborne WR, Scott CR. The metabolism of deoxyguanosine and guanosine in human B and T-lymphoblasts. A role for deoxyguanosine kinase activity in the selective T-cell defect associated with purine nucleoside phosphorylase deficiency. Biochem J. 1983; 214:711-8.
19. Fairbanks LD, Taddeo A, Duley JA, Simmonds HA. Mechanisms of deoxyguanosine lymphotoxicity. Human thymocytes, but not peripheral blood lymphocytes accumulate deoxy-GTP in conditions simulating purine nucleoside phosphorylase deficiency. J Immunol. 1990; 144:485-91.
20. Cohen A, Lee JW, Gelfand EW. Selective toxicity of deoxyguanosine and arabinosylguanine for T leukemic cells. Blood. 1983; 61:660-6.
21. Lambe CU, Averett DR, Paff MT, Reardon JE, Wilson JG, Krenitsky TA. 2 Amino 6 methoxypurine arabinoside: an agent for T-cell malignancies. Cancer Res. 1995; 55:3352-6.
22. Prus KL, Averett DR, Zimmerman TP. Transport and metabolism of 9 β D arabinofuranosylguanine in a human T-lymphoblastoid cell line: nitrobenzylthioinosine-sensitive and -insensitive influx. Cancer Res. 1990; 50:1817-21.
23. Gandhi V, Mineishi S, Huang P, Chapman AJ, Yang Y, Chen F, Nowak B, Chubb S, Hertel LW, Plunkett W. Cytotoxicity, metabolism, and mechanisms of action of 2',2' difluorodeoxyguanosine in Chinese hamster ovary cells. Cancer Res. 1995; 55:1517-24.
24. Rodriguez CO, Gandhi V. Arabinosylguanine-induced apoptosis of T lymphoblastic cells: incorporation into DNA is a necessary step. Cancer Res. 1999; 59:4937-43.
25. Rodriquez CO, Stellrecht CM, Gandhi V. Mechanisms for T-cell selective cytotoxicity of arabinosylguanine. Blood. 2003; 102:1842-8.
26. Kisor DF. Nelarabine: a nucleoside analog with efficacy in T cell and other leukemias. Ann Pharmacother. 2005 Jun; 39 (6): 1056-63.
27. Gandhi V, Plunkett W, Rodriguez Jr CO. Compound GW506U78 in refractory hematologic malignancies: relation between cellular pharmacokinetics and clinical response. J Clin Oncol. 1998 Nov; 16 (11): 3607-15.
28. Gandhi V, Plunkett W, Weller S, Du M, Ayres M, Rodriguez CO Jr, Ramakrishna P, Rosner GL, Hodge JP, O'Brien S, Keating MJ. Evaluation of the combination of nelarabine and fludarabine in leukemias: clinical response, pharmacokinetics, and pharmacodynamics in leukemia cells. J Clin Oncol. 2001 Apr 15; 19 (8): 2142 52.
29. Ravandi F, Gandhi V. Novel purine nucleoside analogues for T cell-lineage acute lymphoblastic leukaemia and lymphoma. Expert Opin Investig Drugs. 2006 Dec; 15 (12): 1601-13.
30. European Medicines Agency. Atriance:scientific discussion [online]. Available from URL. http://www.emea.europa.eu/humandocs/Humans/EPAR/atriance/atriance.htm
31. Gandhi V, Plunkett W. Clofarabine and nelarabine: two new purine nucleoside analogs. Curr Opin Oncol. 2006 Nov; 18 (6): 584-90.
32. European Medicines Agency. Atriance (nelarabine): summary of product information [online]. Available from URL. http://www.emea.europa.eu/humandocs/PDFs/EPAR/atriance/H-752-PI-en.pdf [Accessed 2007 Dec 6]
33. Kurtzberg J, Ernst TJ, Keating MJ, Gandhi V, Hodge JP, Kisor DF, Lager JJ, Stephens C, Levin J, Krenitsky T, Elion G, Mitchell BS. Phase I study of 506U78 administered on a consecutive 5-day schedule in children and adults with refractory hematologic malignancies. J Clin Oncol. 2005; 23:3396-403.
34. DeAngelo DJ, Yu D, Johnson JL, Coutre SE, Stone RM, Stopeck AT, Gockerman JP, Mitchell BS, Appelbaum FR, Larson RA. Nelarabine induces complete remissions in adults with relapsed or refractory T-lineage acute lymphoblastic leukemia or lymphoblastic lymphoma: Cancer and Leukemia Group B study 19801. Blood. 2007 Jun 15; 109 (12): 5136-42.
35. Winter SS, Dunsmore KP, Devidas M, Eisenberg N, Asselin BL, Wood BL, Leonard Rn MS, Murphy J, Gastier-Foster JM, Carroll AJ, Heerema NA, Loh ML, Raetz EA, Winick NJ, Carroll WL, Hunger SP. Safe integration of nelarabine into intensive chemotherapy in newly diagnosed T-cell acute lymphoblastic leukemia: Children’s Oncology Group Study AALL0434. Pediatr Blood Cancer. 2015; 62(7):1176-1183.
36. Zwaan CM, Kowalczyk J, Schmitt C, Bielorai B, Russo MW, Woessner M, Ranganathan S, Leverger G. Safety and efficacy of nelarabine in children and young adults with relapsed or refractory T-lineage acute lymphoblastic leukaemia or T-lineage lymphoblastic lymphoma: results of a phase 4 study. British Journal of Haematology. 2017; 179: 284-293.
37. Seibel NL, Steinherz PG, Sather HN, Nachman JB, Delaat C, Ettinger LJ, Freyer DR, Mattano LA Jr, Hastings CA, Rubin CM, Bertolone K, Franklin JL, Heerema NA, Mitchell TL, Pyesmany AF, La MK, Edens C, Gaynon PS. Early postinduction intensification therapy improves survival for children and adolescents with high-risk acute lymphoblastic leukemia: a report from the Children’s Oncology Group. Blood. 2008; 111(5):2548-2555.
38. Kamps WA, Bokkerink JP, Hahlen K, Hermans J, Riehm H, Gadner H, Schrappe M, Slater R, van den Berg-de Ruiter E, Smets LA, de Vaan GA, Weening RS, van Weerden JF, van Wering ER, den der Does-van den Berg A. Intensive treatment of children with acute lymphoblastic leukemia according to ALL-BFM-86 without cranial radiotherapy: results of Dutch Childhood Leukemia Study Group Protocol ALL-7 (1988–1991). Blood. 1999; 94(4):1226-1236.
39. Salzer WL, Devidas M, Carroll WL Winick N, Pullen J, Hunger SP, Camitta BA. Long-term results of the pediatric oncology group studies for childhood acute lymphoblastic leukemia 1984 – 2001: a report from the children’s oncology group. Leukemia. 2010; 24(2):355-370.
40. Goldberg JM, Silverman LB, Levy DE, Dalton VK, Gelber RD, Lehmann L, Cohen HJ, Sallan SE, Asselin BL. Childhood T-cell acute lymphoblastic leukemia: the Dana-Farber Cancer Institute acute lymphoblastic leukemia consortium experience. J Clin Oncol. 2003; 21(19):3616-3622.
41. Nachman JB, Sather HN, Sensel MG, Trigg ME, Cherlow JM, Lukens JN, Wolff L, Uckun FM, Gaynon PS. Augmented post-induction therapy for children with high-risk acute lymphoblastic leukemia and a slow response to initial therapy. N Engl J Med. 1998; 338(23):1663-71.
42. Dunsmore KP, Devidas M, Linda SB, Borowitz MJ, Winick N, Hunger SP, Carroll WL, Camitta BM. Pilot study of nelarabine in combination with intensive chemotherapy in high-risk T-cell acute lymphoblastic leukemia: a report from the Children’s Oncology Group. J Clin Oncol. 2012; 30(22):2753-2759.
43. Loh M. Efficacy of nelarabine in newly diagnosed T-cell acute lymphoblastic leukemia: Children’s Oncology Group Study AALL0434. 29th Annual Meeting of the international BFM Study Group. 2018.
44. Новикова И.А., Вержбицкая Т.Ю., Мовчан Л.В., Цаур Г.А., Белевцев М.В., Попов А.М. Стандарт Российско-белорусской кооперативной группы по иммунофенотипированию острого лимфобластного лейкоза у детей. Онкогематология. 2018;13(1): 73 82. [Novikova IA, Verzhbitskaya TYu, Movchan LV, Tsaur GA, Belevtsev MV, Popov AM. Russian-belarusian multicenter group standard guidelines for childhood acute lymphoblastic leukemia flow cytometric diagnostics. Oncohematology. 2018;13(1):73-82. (In Russ.)].
45. Bene MC, Castoldi G, Knapp W, Ludwig WD, Matutes E, Orfao A, van't Veer MB. Proposals for the immunological classification of acute leukemias. European Group for the Immunological Characterization of Leukemias (EGIL). Leukemia. 1995; 9: 1783-1786.
46. Dworzak MN, Gaipa G, Ratei R, Veltroni M, Schumich A, Maglia O, Karawajew L, Benetello A, Pötschger U, Husak Z, Gadner H, Biondi A, Ludwig WD, Basso G. Standardization of flow cytometric minimal residual disease evaluation in acute lymphoblastic leukemia: multicentric assessment is feasible. Cytometry B Clin Cytom. 2008; 74: 331-340.
47. Попов А.М., Вержбицкая Т.Ю., Фечина Л.Г., Шестопалов А.В., Плясунова С.А. Острые лейкозы: различия иммунофенотипа бластных клеток и их неопухолевых аналогов в костном мозге. Клин. онкогематол. 2016; 9(3): 302-13. [Popov АM, Verzhbitskaya ТYu, Fechina LG, Shestopalov AV, Plyasunova SA. Acute Leukemias: Immunophenotypic Differences between Blast Cells and Their Nonneoplastic Analogues in Bone Marrow. Clinical oncohematology. 2016; 9(3):302-13. (In Russ.)].
48. Berg SL, Blaney SM, Devidas M, Lampkin TA, Murgo A, Bernstein M, Billett A, Kurtzberg J, Reaman G, Gaynon P, Whitlock J, Krailo M, Harris MB. Phase II study of nelarabine (compound 506U78) in children and young adults with refractory T-cell malignancies: a report from the Children’s Oncology Group. J Clin Oncol. 2005; 23:3376 82.
49. Kadia TM, Gandhi V. Nelarabine in the treatment of pediatric and adult patients with T cell acute lymphoblastic leukemia and lymphoma. Expert Rev Hematol. 2017 January; 10(1): 1-8.
50. Lonetti A, Cappellini A, Bertaina A, Locatelli F, Pession A, Buontempo F, Evangelisti Ca, Evangelisti Ce, Orsini E, Zambonin L, Neri LM, Martelli AM, Chiarini F. Improving nelarabine efficacy in T cell acute lymphoblastic leukemia by targeting aberrant PI3K/AKT/mTOR signaling pathway. J Hematol Oncol. 2016; 9: 114.
51. Herold N, Rudd SG, Sanjiv K, Kutzner J, Bladh J, Paulin CBJ, Helleday T, Henter JI, Schaller T. SAMHD1 protects cancer cells from various nucleoside-based antimetabolites. Cell Cycle. 2017; 16: 1029-1038.
52. Burley K, Wolf J, Raffoux E, Marks DI. Long-term survival following post-allograft relapse of T-cell acute lymphoblastic leukaemia: a novel approach using nelarabine and donor lymphocyte infusions. Bone Marrow Transplant. 2017; published online 31 Dec. https://doi.org/10. 1038/s41409-017-0038-8
________________________________________________
2. Garand R, Vannier JP, Bene MC, Faure G, Favre M, Bernard A. Comparison of outcome, clinical, laboratory, and immunological features in 164 children and adults with T-ALL. The Groupe d’Etude Immunologique des Leucemies. Leukemia. 1990; 4:739-44.
3. Bash RO, Crist WM, Shuster JJ, Link MP, Amylon M, Pullen J, Carroll AJ, Buchanan GR, Smith RG, Baer R. Clinical features and outcome of T-cell acute lymphoblastic leukemia in childhood with respect to alterations at the TAL1 locus: a Pediatric Oncology Group study. Blood. 1993; 81:2110-7.
4. Uckun FM, Sensel MG, Sun L, Steinherz PG, Trigg ME, Heerema NA, Sather HN, Reaman GH, Gaynon PS. Biology and treatment of childhood T-lineage acute lymphoblastic leukemia. Blood.1998; 91:735-46.
5. Pui CH, Relling MV, Evans WE. Role of pharmacogenomics and pharmacodynamics in the treatment of acute lymphoblastic leukaemia. Best Pract Res Clin Haematol. 2002; 15:741-56.
6. Reiter A, Schrappe M, Ludwig WD, Hiddemann W, Sauter S, Henze G, Zimmermann M, Lampert F, Havers W, Niethammer D, Odenwald E, Ritter J, Mann G, Welte K, Gadner H, Riehm H. Chemotherapy in 998 unselected childhood acute lymphoblastic leukemia patients. Results and conclusions of the multicenter trial ALL-BFM 86. Blood. 1994; 84:3122-33.
7. Schorin MA, Blattner S, Gelber RD, Tarbell NJ, Donnelly M, Dalton V, Cohen HJ, Sallan SE. Treatment of childhood acute lymphoblastic leukemia: results of Dana-Farber Cancer Institute/Children’s Hospital Acute Lymphoblastic Leukemia Consortium Protocol 85-01. J Clin Oncol. 1994; 12:740-7.
8. Cohen MH, Johnson JR, Massie T, Sridhara R, McGuinn WD Jr, Abraham S, Booth BP, Goheer MA, Morse D, Chen XH, Chidambaram N, Kenna L, Gobburu JV, Justice R, Pazdur R. Approval summary: nelarabine for the treatment of T-cell lymphoblastic leukemia/lymphoma. Clin Cancer Res. 2006; 12:5329-35.
9. Kurtzberg J. The long and winding road of the clinical development of Nelarabine. Leukemia&Lymphoma. 2007; 48(1): 1-2.
10. Giblett E, Ammann A, Wara D, Sandman R, Diamond L. Nucleoside-phosphorylase deficiency in a child with severely defective T-cell immunity and normal B-cell immunity. Lancet. 1975; 1:1010-1013.
11. Cohen A, Gudas LJ, Ammann AJ, Staal GE, Martin DW Jr. Deoxyguanosine triphosphate, a possible toxic metabolite in immunodeficiency associated with purine nucleotide phosphorylase deficiency. J Clin Invest. 1978; 61:1405 1410.
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Авторы
Ю.Ю. ДЬЯКОНОВА 1, О.И. БЫДАНОВ 1,3, А.М. ПОПОВ 1, Ю.В. ОЛЬШАНСКАЯ 1, Э.Г. БОЙЧЕНКО 2, О.В. АЛЕЙНИКОВА 3, М.А. МАСЧАН 1, Л.Н. ШЕЛИХОВА 1, Д.В. ЛИТВИНОВ 1, Л.А. ХАЧАТРЯН 1, Н.И. ПОНОМАРЕВА 4, Л.Г. ФЕЧИНА 5, Г.А. НОВИЧКОВА 1, Е.Д. ПАШАНОВ 1, А.И. КАРАЧУНСКИЙ 1
1 ФГБУ «Национальный медицинский исследовательский центр детской гематологии, онкологии и иммунологии
им. Дмитрия Рогачева» Минздрава России, Москва, Россия;
2 СПбГУЗ «Детская городская больница №1», Санкт-Петербург, Россия;
3 Республиканский научно-практический центр детской онкологии, гематологии и иммунологии, Минск, Республика Беларусь;
4 Российская детская клиническая больница Минздрава РФ, Москва, Россия;
5 Областная детская клиническая больница № 1, Екатеринбург, Россия
1 Dmitry Rogachev National Research Center for Pediatric Hematology, Oncology, and Immunology, Moscow, Russia;
2 «Children City Hospital №1, Saint-Petersburg, Russia;
3 Republican Scientific and Practical Center for Pediatric Oncology, Hematology, and Immunology, Minsk, Republic of Belarus;
4 Russian Children Clinical Hospital, Moscow, Russia;
5Regional Children Clinical Hospital №1, Ekaterinburg, Russia
1 ФГБУ «Национальный медицинский исследовательский центр детской гематологии, онкологии и иммунологии
им. Дмитрия Рогачева» Минздрава России, Москва, Россия;
2 СПбГУЗ «Детская городская больница №1», Санкт-Петербург, Россия;
3 Республиканский научно-практический центр детской онкологии, гематологии и иммунологии, Минск, Республика Беларусь;
4 Российская детская клиническая больница Минздрава РФ, Москва, Россия;
5 Областная детская клиническая больница № 1, Екатеринбург, Россия
________________________________________________
1 Dmitry Rogachev National Research Center for Pediatric Hematology, Oncology, and Immunology, Moscow, Russia;
2 «Children City Hospital №1, Saint-Petersburg, Russia;
3 Republican Scientific and Practical Center for Pediatric Oncology, Hematology, and Immunology, Minsk, Republic of Belarus;
4 Russian Children Clinical Hospital, Moscow, Russia;
5Regional Children Clinical Hospital №1, Ekaterinburg, Russia
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