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Вопросы гематологии/онкологии и иммунопатологии в педиатрии. 2016; 15: 57-65

Роль и место трансплантации гемопоэтических стволовых клеток в лечении острого миелоидного лейкоза у детей. Опыт Беларуси

Баровская Юлия Александровна, Прудников Дмитрий Владимирович, Минаковская Нина Вячеславовна, Стёганцева Мария Владимировна, Белевцев Михаил Владимирович, Алейникова Ольга Витальевна

https://doi.org/10.24287/1726-1708-2016-15-4-57-65

Аннотация

Целью данной работы явился анализ роли трансплантации гемопоэтических стволовых клеток в лечении острого миелоидного лейкоза у детей. В исследование включен 151 пациент с de novo острым миелоидным лейкозом и 39 пациентов с рецидивом. Пациенты с de novo острым миелоидным лейкозом получали программное лечение в Республиканском научно-практическом центре детской онкологии, гематологии и иммунологии г. Минска в период с мая 1999 г. по декабрь 2013 г. по оригинальным протоколам ОМЛ-ММ-2000 и ОМЛ-ММ-2006. Пациенты с рецидивом острого миелоидного лейкоза получали лечение по международному протоколу AML-Relapsed 2001/01. В ходе анализа показано, что проведение аллогенной трансплантации гемопоэтических стволовых клеток абсолютно показано пациентам с прогностически неблагоприятными молекулярно-генетическими поломками в первой ремиссии и всем пациентам во второй линии лечения. Пациенты благоприятной прогностической группы имеют хорошие результаты лечения и без проведения трансплантации гемопоэтических стволовых клеток в первой ремиссии.
Список литературы

1. Rubnitz J, Inaba Н. Childhood Acute Myeloid Leukaemia. British J of Haematology. 2012;159(3):259-76.

2. Hasle H. A critical review of which children with acute myeloid leukaemia need stem cell procedures. British J of Haematology. 2014;166(1):23-33.

3. Armenian S, Sun C, Kawashima T. Long-term health-related outcomes in survivors of childhood cancer treated with HSCT versus conventional therapy: a report from the Bone Marrow Transplant Survivor Study (BMTSS) and Childhood Cancer Survivor Study (CCSS). Blood. 2011;118(5):1413-20.

4. Pession A, Masetti R, Rizzari C, Putti M, Casale F, Fagioli F, et al. Results of the AIEOP AML 2002/01 multicenter prospective trial for the treatment of children with acute myeloid leukemia. Blood. 2013;122(2):170-8.

5. Creutzig U, Zimmermann M, Bourquin J, Dworzak M, Fleischhack G, Graf N, et al. Randomized trial comparing liposomal daunorubicin with idarubicin as induction for pediatric acute myeloid leukemia: results from Study AML-BFM 2004. Blood. 2013;122(1):37-43.

6. Canner J, Alonzo T, Franklin J, Freyer D, Gamis A, Gerbing R, et al. Differences in outcomes of newly diagnosed acute myeloid leukemia for adolescent/young adult and younger patients: a report from the Children's Oncology Group. Cancer. 2013;119(23):4162-9.

7. Tsukimoto I, Tawa A, Horibe K, Tabuchi K, Kigasawa H, Tsuchida M, et al. Risk-stratified therapy and the intensive use of cytarabine improves the outcome in childhood acute myeloid leukemia: the AML99 trial from the Japanese Childhood AML Cooperative Study Group. J Clin Oncol. 2009;27(24):4007-13.

8. Perel Y, Auvrignon A, Leblanc T, Michel G, Reguerre Y, Vannier J, et al. Treatment of childhood acute myeloblastic leukemia: dose intensification improves outcome and maintenance therapy is of no benefit--multicenter studies of the French LAME (Leucemie Aiguё Myeloblastique Enfant) Cooperative Group. Leukemia. 2005; 19(12):2082-9.

9. Gibson B, Webb D, Howmann A, De Graaf S, Harrison C, Wheatley K, et al. Results of a randomized trial in children with Acute Myeloid Leukaemia: medical research council AML12 trial. British J of Haematology. 2011;155:366-76.

10. Hasle H, Abrahamsson J, Forestier E, Ha S, Heldrup J, Jahnukainen K, et al. Gemtuzumab ozogamicin as postconsolidation therapy does not prevent relapse in children with AML: results from NOPHO-AML 2004. Blood. 2012;120(5): 978-84.

11. Rubnitz J, Inaba H, Dahl G, Ribeiro R, Bowman W, Taub J, et al. Minimal residual disease-directed therapy for childhood acute myeloid leukaemia: results of the AML02 multicentre trial. Lancet Oncol. 2010;11(6):543-52.

12. Horan J, Alonzo T, Lyman G, Gerbing R, Lange B, Ravindranath Y, et al. Impact of Disease Risk on Efficacy of Matched Related Bone Marrow Transplantation for Pediatric Acute Myeloid Leukemia: The Children's Oncology Group. J Clin Oncol. 2008;26(35):5797-801.

13. Neiwerth D, Creutzig U, Bierings M, Kaspers G. A review on allogeneic stem cell transplantation for newly diagnosed pediatric acute myeloid leukemia. Blood. 2010;116(13):2205-14.

14. Lee D, Kwon Y, Lim J, Kim Y, Han K, Chung N, et al. Comparable outcomes of HLA-matched unrelated and HLA-identical sibling donor bone marrow transplantation for childhood acute myeloid leukemia in first remission. Pediatr Transplant. 2009 Mar;13(2):210-6.

15. Shinzato A, Tabuchi K, Atsuta Y, Inoue M, Inagaki J, Yabe H, et al. PBSCT is associated with poorer survival and increased chronic GvHD than BMT in Japanese paediatric patients with acute leukaemia and an HLA-matched sibling donor. Pediatr Blood Cancer. 2013;60(9):1513-9.

16. Ballen K, Gluckman E, Broxmeyer H. Umbilical cord blood transplantation: the first 25 years and beyond. Blood. 2013;122(4):491-8.

17. Rocha V, Gluckman E, Frassoni F, Podesta M, Gallamini A, Bonifazi F, et al. Unrelated cord blood transplantation:outcomes after single-unit intrabone injection compared with double-unitintravenous injection in patients with hematological malignancies. Transplantation. 2013;95(10):1284-91.

18. Page K, Zhang L, Kurtzberg J, Wease S, Carter S, Gentry T, et al. Total colony-forming units are a strong, independent predictor of neutrophil and platelet engraftment after unrelated umbilical cord blood transplantation: a single-center analysis of 435 cord blood transplants. Biol. Blood Marrow Transplant. 2011;17(9):1362-74.

19. Barker J, Scaradavou A, Stevens C. Combined effect of total nucleated cell dose and HLA match on transplantation outcome in 1061 cord blood recipients with hematologic malignancies. Blood. 2010;115:1843-9.

20. Sideri A, Neokleous N, Gluckman E, Guerton B, Le Bousse Kerdilles M, Uzan G, et al. An overview of the progress on double umbilical cord blood transplantation. Haematologica. 2011;96(8):1213-20.

21. Rocha V, Crotta A, Gluckman E, Purtill D, Boudjedir K, Herr A, et al. Double cord blood transplantation: extending the use of unrelated umbilical cord blood cells for patients with hematological diseases. Best Pract Res Clin Haematol. 2010;23(2):223-9.

22. Locatelli F, Vinti L, Palumbo G, Rossi F, Bertaina A, Mastronuzzi A, et al. Strategies to optimize the outcome of children given T-cell depleted HLA-haploidentical hematopoietic stem cell transplantation. Best Pract Res Clin Haematol. 2011;24(3):339-49.

23. Субботина Н, Долгополов С, Попа А, Бояршинов В, Пименов Р, Менткевич Г. Гаплоидентичная трансплантация гемопоэтических стволовых клеток у детей с острыми миелоидными лейкозами: эволюция метода и собственные данные. Клиническая онкогематология. 2014;7(2):131 -6

24. Abrahamsson J, Forestier E, Heldrup J, Jahnukainen K, Jönsson O, Lausen B, et al. Response-guided induction therapy in pediatric acute myeloid leukemia with excellent remission rate. J Clin Oncol. 2011;29(3):310-5.

25. Walter R, Buckley S, Pagel J, Wood B, Storer B, Sandmaier B, et al. Significance of minimal residual disease before myeloablative allogeneic hematopoietic cell transplantation for AML in first and second complete remission. Blood. 2013;122(10):1813-21.

26. Leung W, Pui C, Coustan-Smith E, Yang J, Pei D, Gan K, Srinivasan A, et al. Detectable minimal residual disease before hematopoietic cell transplantation is prognostic but does not preclude cure for children with very-high-risk leukemia. Blood. 2012;120(2):468-72.

27. van Dongen J, Macintyre E, Gabert J, Delabesse E, Rossi V, Saglio G, et al. Standardized RT-PCR analysis of fusion gene transcripts from chromosome aberrations in acute leukemia for detection of minimal residual disease. Report of the BIOMED-1 Concerted Action: investigation of minimal residual disease in acute leukemia. Leukemia. 1999;13(12):1901-28.

28. Kaplan E, Meier P. Nonparametric Estimation from Incomplete Observations. J of the American Statistical Association. 1958;53(282):457-81.

29. Gooley T, Leisenring W, Crowley J, Storer B. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine. 1999;18(6):695-706.

30. Gray R. A class of K-sample tests for comparing the cumulative incidence of a competing risk. The Annals of Statistics. 1988;16(3):1140-54.

31. Creutzig U, van den Heuvel-Eibrink M, Gibson B, Dworzak M, Adachi S, de Bont E, et al. Diagnosis and management of acute myeloid leukemia in children and adolescents: recommendations from an international expert panel. Blood. 2012;120(16):3187-205.

32. Кирсанова НП, Алейникова ОВ, Кустанович АМ, Савицкая ТВ, Юцкевич РИ, Волочник ЕВ и др. Результаты лечения острого миелоидного лейкоза у детей неблагоприятного прогноза по протоколу ОМЛ-ММ-2000. Онкогематология. 2009;1:5-13.

Pediatric Hematology/Oncology and Immunopathology. 2016; 15: 57-65

The role and place of haemopoietic stem cell transplantation in treatment of acute myeloid leukemia in children. A Belarus experience

Barovskaya Yulia A., Prudnikov Dmitriy V., Minakovskaya Nina V., Stegantseva Maria V., Belevtsev Mikhail V., Aleynikova Olga V.

https://doi.org/10.24287/1726-1708-2016-15-4-57-65

Abstract

The work was aimed at analysis of the role of haemopoietic stem cell transplantation in treatment of acute myeloid leukemia in children. The study included 151 patients with de novo acute myeloid leukemia and 39 patients with relapsed. Patients with de novo acute myeloid leukemia received programme treatment in the Republican Research and Practical Centre of Paediatric Oncology, Haematology and Immunology of the city of Minsk in the period from May 1999 to December 2013 according to original protocols AML-ММ-2000 and AML-ММ-2006. Patients with relapsed acute myeloid leukemia received treatment according to the international protocol AML-Relapsed 2001/01. Our analysis has shown that allogenic haemopoietic stem cell transplantation is absolutely indicated to patients with prognostically unfavourable molecular-genetic abnormalities in first remission and to all patients receiving second-line treatment. Patients of a favourable prognostic group have good results of treatment even without haemopoietic stem cell transplantation in first remission.
References

1. Rubnitz J, Inaba N. Childhood Acute Myeloid Leukaemia. British J of Haematology. 2012;159(3):259-76.

2. Hasle H. A critical review of which children with acute myeloid leukaemia need stem cell procedures. British J of Haematology. 2014;166(1):23-33.

3. Armenian S, Sun C, Kawashima T. Long-term health-related outcomes in survivors of childhood cancer treated with HSCT versus conventional therapy: a report from the Bone Marrow Transplant Survivor Study (BMTSS) and Childhood Cancer Survivor Study (CCSS). Blood. 2011;118(5):1413-20.

4. Pession A, Masetti R, Rizzari C, Putti M, Casale F, Fagioli F, et al. Results of the AIEOP AML 2002/01 multicenter prospective trial for the treatment of children with acute myeloid leukemia. Blood. 2013;122(2):170-8.

5. Creutzig U, Zimmermann M, Bourquin J, Dworzak M, Fleischhack G, Graf N, et al. Randomized trial comparing liposomal daunorubicin with idarubicin as induction for pediatric acute myeloid leukemia: results from Study AML-BFM 2004. Blood. 2013;122(1):37-43.

6. Canner J, Alonzo T, Franklin J, Freyer D, Gamis A, Gerbing R, et al. Differences in outcomes of newly diagnosed acute myeloid leukemia for adolescent/young adult and younger patients: a report from the Children's Oncology Group. Cancer. 2013;119(23):4162-9.

7. Tsukimoto I, Tawa A, Horibe K, Tabuchi K, Kigasawa H, Tsuchida M, et al. Risk-stratified therapy and the intensive use of cytarabine improves the outcome in childhood acute myeloid leukemia: the AML99 trial from the Japanese Childhood AML Cooperative Study Group. J Clin Oncol. 2009;27(24):4007-13.

8. Perel Y, Auvrignon A, Leblanc T, Michel G, Reguerre Y, Vannier J, et al. Treatment of childhood acute myeloblastic leukemia: dose intensification improves outcome and maintenance therapy is of no benefit--multicenter studies of the French LAME (Leucemie Aigue Myeloblastique Enfant) Cooperative Group. Leukemia. 2005; 19(12):2082-9.

9. Gibson B, Webb D, Howmann A, De Graaf S, Harrison C, Wheatley K, et al. Results of a randomized trial in children with Acute Myeloid Leukaemia: medical research council AML12 trial. British J of Haematology. 2011;155:366-76.

10. Hasle H, Abrahamsson J, Forestier E, Ha S, Heldrup J, Jahnukainen K, et al. Gemtuzumab ozogamicin as postconsolidation therapy does not prevent relapse in children with AML: results from NOPHO-AML 2004. Blood. 2012;120(5): 978-84.

11. Rubnitz J, Inaba H, Dahl G, Ribeiro R, Bowman W, Taub J, et al. Minimal residual disease-directed therapy for childhood acute myeloid leukaemia: results of the AML02 multicentre trial. Lancet Oncol. 2010;11(6):543-52.

12. Horan J, Alonzo T, Lyman G, Gerbing R, Lange B, Ravindranath Y, et al. Impact of Disease Risk on Efficacy of Matched Related Bone Marrow Transplantation for Pediatric Acute Myeloid Leukemia: The Children's Oncology Group. J Clin Oncol. 2008;26(35):5797-801.

13. Neiwerth D, Creutzig U, Bierings M, Kaspers G. A review on allogeneic stem cell transplantation for newly diagnosed pediatric acute myeloid leukemia. Blood. 2010;116(13):2205-14.

14. Lee D, Kwon Y, Lim J, Kim Y, Han K, Chung N, et al. Comparable outcomes of HLA-matched unrelated and HLA-identical sibling donor bone marrow transplantation for childhood acute myeloid leukemia in first remission. Pediatr Transplant. 2009 Mar;13(2):210-6.

15. Shinzato A, Tabuchi K, Atsuta Y, Inoue M, Inagaki J, Yabe H, et al. PBSCT is associated with poorer survival and increased chronic GvHD than BMT in Japanese paediatric patients with acute leukaemia and an HLA-matched sibling donor. Pediatr Blood Cancer. 2013;60(9):1513-9.

16. Ballen K, Gluckman E, Broxmeyer H. Umbilical cord blood transplantation: the first 25 years and beyond. Blood. 2013;122(4):491-8.

17. Rocha V, Gluckman E, Frassoni F, Podesta M, Gallamini A, Bonifazi F, et al. Unrelated cord blood transplantation:outcomes after single-unit intrabone injection compared with double-unitintravenous injection in patients with hematological malignancies. Transplantation. 2013;95(10):1284-91.

18. Page K, Zhang L, Kurtzberg J, Wease S, Carter S, Gentry T, et al. Total colony-forming units are a strong, independent predictor of neutrophil and platelet engraftment after unrelated umbilical cord blood transplantation: a single-center analysis of 435 cord blood transplants. Biol. Blood Marrow Transplant. 2011;17(9):1362-74.

19. Barker J, Scaradavou A, Stevens C. Combined effect of total nucleated cell dose and HLA match on transplantation outcome in 1061 cord blood recipients with hematologic malignancies. Blood. 2010;115:1843-9.

20. Sideri A, Neokleous N, Gluckman E, Guerton B, Le Bousse Kerdilles M, Uzan G, et al. An overview of the progress on double umbilical cord blood transplantation. Haematologica. 2011;96(8):1213-20.

21. Rocha V, Crotta A, Gluckman E, Purtill D, Boudjedir K, Herr A, et al. Double cord blood transplantation: extending the use of unrelated umbilical cord blood cells for patients with hematological diseases. Best Pract Res Clin Haematol. 2010;23(2):223-9.

22. Locatelli F, Vinti L, Palumbo G, Rossi F, Bertaina A, Mastronuzzi A, et al. Strategies to optimize the outcome of children given T-cell depleted HLA-haploidentical hematopoietic stem cell transplantation. Best Pract Res Clin Haematol. 2011;24(3):339-49.

23. Subbotina N, Dolgopolov S, Popa A, Boyarshinov V, Pimenov R, Mentkevich G. Gaploidentichnaya transplantatsiya gemopoeticheskikh stvolovykh kletok u detei s ostrymi mieloidnymi leikozami: evolyutsiya metoda i sobstvennye dannye. Klinicheskaya onkogematologiya. 2014;7(2):131 -6

24. Abrahamsson J, Forestier E, Heldrup J, Jahnukainen K, Jönsson O, Lausen B, et al. Response-guided induction therapy in pediatric acute myeloid leukemia with excellent remission rate. J Clin Oncol. 2011;29(3):310-5.

25. Walter R, Buckley S, Pagel J, Wood B, Storer B, Sandmaier B, et al. Significance of minimal residual disease before myeloablative allogeneic hematopoietic cell transplantation for AML in first and second complete remission. Blood. 2013;122(10):1813-21.

26. Leung W, Pui C, Coustan-Smith E, Yang J, Pei D, Gan K, Srinivasan A, et al. Detectable minimal residual disease before hematopoietic cell transplantation is prognostic but does not preclude cure for children with very-high-risk leukemia. Blood. 2012;120(2):468-72.

27. van Dongen J, Macintyre E, Gabert J, Delabesse E, Rossi V, Saglio G, et al. Standardized RT-PCR analysis of fusion gene transcripts from chromosome aberrations in acute leukemia for detection of minimal residual disease. Report of the BIOMED-1 Concerted Action: investigation of minimal residual disease in acute leukemia. Leukemia. 1999;13(12):1901-28.

28. Kaplan E, Meier P. Nonparametric Estimation from Incomplete Observations. J of the American Statistical Association. 1958;53(282):457-81.

29. Gooley T, Leisenring W, Crowley J, Storer B. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine. 1999;18(6):695-706.

30. Gray R. A class of K-sample tests for comparing the cumulative incidence of a competing risk. The Annals of Statistics. 1988;16(3):1140-54.

31. Creutzig U, van den Heuvel-Eibrink M, Gibson B, Dworzak M, Adachi S, de Bont E, et al. Diagnosis and management of acute myeloid leukemia in children and adolescents: recommendations from an international expert panel. Blood. 2012;120(16):3187-205.

32. Kirsanova NP, Aleinikova OV, Kustanovich AM, Savitskaya TV, Yutskevich RI, Volochnik EV i dr. Rezul'taty lecheniya ostrogo mieloidnogo leikoza u detei neblagopriyatnogo prognoza po protokolu OML-MM-2000. Onkogematologiya. 2009;1:5-13.