Một đánh giá về việc ghép tế bào gốc đồng bào cho trẻ em mới được chẩn đoán mắc bệnh bạch cầu myeloid cấp tính

Blood - Tập 116 - Trang 2205-2214 - 2010
Denise Niewerth1, Ursula Creutzig2, Marc B. Bierings3, Gertjan J.L. Kaspers1
1Department of Pediatric Oncology/Hematology, VU University Medical Center, Amsterdam, The Netherlands
2Department of Pediatric Hematology/Oncology, University of Muenster, Muenster, Germany; and
3Wilhelmina Children’s Hospital, University Medical Center Utrecht, Utrecht, The Netherlands

Tóm tắt

Tóm tắt Sự sống sót của trẻ em mắc bệnh bạch cầu myeloid cấp tính (AML) đã cải thiện đáng kể trong vài thập kỷ qua. Kể từ năm 1985, việc ghép tế bào gốc đồng bào (allo-SCT) được khuyến nghị rộng rãi cho các bệnh nhân có người cho tế bào gốc tương thích. Tuy nhiên, vấn đề còn gây tranh cãi là liệu allo-SCT có vượt trội hơn hóa trị liệu đối với trẻ em mới được chẩn đoán mắc AML hay không. Bài đánh giá này tổng hợp các thử nghiệm lâm sàng giai đoạn 3 so sánh allo-SCT với hóa trị liệu (bao gồm cả SCT tự thân) ở bệnh nhi mắc AML, không bao gồm các nghiên cứu không sử dụng phân tích theo ý định điều trị hoặc không điều chỉnh cho thời gian đến ghép. Mặc dù allo-SCT có thể ngăn ngừa nhiều đợt tái phát hơn hóa trị liệu, số bệnh nhân cần được ghép (với allo-SCT) để ngăn ngừa một đợt tái phát là khoảng 10 bệnh nhân. Hơn nữa, tỷ lệ sống sót toàn bộ là tương tự với cả hai phương pháp trong hầu hết các nghiên cứu gần đây, rõ ràng bởi vì khả năng phục hồi cao hơn của một đợt tái phát khi liệu pháp ban đầu chỉ liên quan đến hóa trị liệu, và vì tỷ lệ tử vọng liên quan đến điều trị cao hơn với allo-SCT. Do allo-SCT cũng gây ra nhiều tác dụng phụ nghiêm trọng hơn và thường dẫn đến các khối u thứ phát hơn so với hóa trị liệu, chúng tôi không khuyến nghị allo-SCT trong đợt remiss đầu tiên cho bệnh nhi mắc AML nói chung. Nghiên cứu thêm nên tập trung vào khả năng rằng các nhóm bệnh nhân con có thể hưởng lợi từ allo-SCT, nhằm hướng tới việc cải thiện thêm tiên lượng cho trẻ em mắc AML.

Từ khóa


Tài liệu tham khảo

Kaspers, 2005, Pediatric acute myeloid leukemia: international progress and future directions., Leukemia, 19, 2025, 10.1038/sj.leu.2403958 Michel, 2003, Unrelated cord blood transplantation for childhood acute myeloid leukemia: a Eurocord Group analysis., Blood, 102, 4290, 10.1182/blood-2003-04-1288 Bleakley, 2002, Bone marrow transplantation for paediatric AML in first remission: a systematic review and meta-analysis., Bone Marrow Transplant, 29, 843, 10.1038/sj.bmt.1703528 Gibson, 2005, Treatment strategy and long-term results in paediatric patients treated in consecutive UK AML trials., Leukemia, 19, 2130, 10.1038/sj.leu.2403924 Ravindranath, 2005, Pediatric Oncology Group (POG) studies of acute myeloid leukemia (AML): a review of four consecutive childhood AML trials conducted between 1981 and 2000., Leukemia, 19, 2101, 10.1038/sj.leu.2403927 Smith, 2005, Long-term results of children with acute myeloid leukemia: a report of three consecutive Phase III trials by the Children's Cancer Group: CCG 251, CCG 213 and CCG 2891., Leukemia, 19, 2054, 10.1038/sj.leu.2403925 Woods, 2001, A comparison of allogeneic bone marrow transplantation, autologous bone marrow transplantation, and aggressive chemotherapy in children with acute myeloid leukemia in remission., Blood, 97, 56, 10.1182/blood.V97.1.56 Heritier, 2003, Inclusion of patients in clinical trial analysis: the intention-to-treat principle., Med J Aust, 179, 438, 10.5694/j.1326-5377.2003.tb05627.x Horan, 2008, 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, 26, 5797, 10.1200/JCO.2007.13.5244 Kaspers, 2007, Pediatric acute myeloid leukemia: towards high-quality cure of all patients., Haematologica, 92, 1519, 10.3324/haematol.11203 Pession, 2005, Treatment and long-term results in children with acute myeloid leukaemia treated according to the AIEOP AML protocols., Leukemia, 19, 2043, 10.1038/sj.leu.2403869 Creutzig, 2005, Treatment strategies and long-term results in paediatric patients treated in four consecutive AML-BFM trials., Leukemia, 19, 2030, 10.1038/sj.leu.2403920 Creutzig, 2006, Less toxicity by optimizing chemotherapy, but not by addition of granulocyte colony-stimulating factor in children and adolescents with acute myeloid leukemia: results of AML-BFM 98., J Clin Oncol, 24, 4499, 10.1200/JCO.2006.06.5037 Reinhardt, 2006, No improvement of overall-survival in children with high-risk acute myeloid leukemia by stem cell transplantation in 1st complete remission [abstract]., Blood, 108, 10.1182/blood.V108.11.320.320 Therapy-Optimization Trial for the Treatment of Acute Myeloid Leukemias (AML) in Children and Adolescents. Accessed March 10, 2009 http://www.clinicaltrials.gov/ct2/show/NCT00111345 Alonzo, 2005, Postremission therapy for children with acute myeloid leukemia: the children's cancer group experience in the transplant era., Leukemia, 19, 965, 10.1038/sj.leu.2403763 Lange, 2008, Outcomes in CCG-2961, a children's oncology group phase 3 trial for untreated pediatric acute myeloid leukemia: a report from the children's oncology group., Blood, 111, 1044, 10.1182/blood-2007-04-084293 Phase III Randomized Study of Conventional Combination Chemotherapy With Versus Without Gemtuzumab Ozogamicin in Young Patients With Newly Diagnosed Acute Myeloid Leukemia. Accessed March 31, 2009 http://www.cancer.gov/clinicaltrials/COG-AAML0531 Kardos, 2005, Treatment strategy and results in children treated on three Dutch Childhood Oncology Group acute myeloid leukemia trials., Leukemia, 19, 2063, 10.1038/sj.leu.2403873 Entz-Werle, 2005, Results of 58872 and 58921 trials in acute myeloblastic leukemia and relative value of chemotherapy vs allogeneic bone marrow transplantation in first complete remission: the EORTC Children Leukemia Group report., Leukemia, 19, 2072, 10.1038/sj.leu.2403932 Perel, 2005, 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, 19, 2082, 10.1038/sj.leu.2403867 Michel, 1996, Allogeneic bone marrow transplantation vs aggressive post-remission chemotherapy for children with acute myeloid leukemia in first complete remission. A prospective study from the French Society of Pediatric Hematology and Immunology (SHIP)., Bone Marrow Transplant, 17, 191 Treating Patients With Childhood Acute Myeloid Leukemia With Interleukin-2 (Elam02). Accessed February 11, 2009 http://clinicaltrials.gov/ct2/show/NCT00149162 Lie, 2005, Long-term results in children with AML: NOPHO-AML Study Group–report of three consecutive trials., Leukemia, 19, 2090, 10.1038/sj.leu.2403962 NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia. Accessed February 12, 2009 http://www.clinicaltrials.gov/ct2/show/NCT00476541 Ravindranath, 1996, Autologous bone marrow transplantation versus intensive consolidation chemotherapy for acute myeloid leukemia in childhood. Pediatric Oncology Group., N Engl J Med, 334, 1428, 10.1056/NEJM199605303342203 Becton, 2006, Randomized use of cyclosporin A (CsA) to modulate P-glycoprotein in children with AML in remission: Pediatric Oncology Group Study 9421., Blood, 107, 1315, 10.1182/blood-2004-08-3218 Ribeiro, 2005, Successive clinical trials for childhood acute myeloid leukemia at St Jude Children's Research Hospital, from 1980 to 2000., Leukemia, 19, 2125, 10.1038/sj.leu.2403872 Treatment of Children and Adolescents With Refractory or Relapsed Acute Myeloid Leukemia. Accessed February 2, 2008 http://www.clinicaltrials.gov/ct2/show/NCT00186966 Tomizawa, 2007, Repetitive cycles of high-dose cytarabine are effective for childhood acute myeloid leukemia: long-term outcome of the children with AML treated on two consecutive trials of Tokyo Children's Cancer Study Group., Pediatr Blood Cancer, 49, 127, 10.1002/pbc.20944 Tsukimoto, 2009, 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, 27, 4007, 10.1200/JCO.2008.18.7948 Stevens, 1998, Marked improvements in outcome with chemotherapy alone in paediatric acute myeloid leukemia: results of the United Kingdom Medical Research Council's 10th AML trial. MRC Childhood Leukaemia Working Party., Br J Haematol, 101, 130, 10.1046/j.1365-2141.1998.00677.x Burnett, 2002, The value of allogeneic bone marrow transplant in patients with acute myeloid leukaemia at differing risk of relapse: results of the UK MRC AML 10 trial., Br J Haematol, 118, 385, 10.1046/j.1365-2141.2002.03724.x Slats, 2005, Causes of death–other than progressive leukemia–in childhood acute lymphoblastic (ALL) and myeloid leukemia (AML): the Dutch Childhood Oncology Group experience., Leukemia, 19, 537, 10.1038/sj.leu.2403665 Watson, 1999, Severe adverse impact on sexual functioning and fertility of bone marrow transplantation, either allogeneic or autologous, compared with consolidation chemotherapy alone: analysis of the MRC AML 10 trial., Cancer, 86, 1231, 10.1002/(SICI)1097-0142(19991001)86:7<1231::AID-CNCR18>3.0.CO;2-Y Zittoun, 1997, Quality of life in patients with acute myelogenous leukemia in prolonged first complete remission after bone marrow transplantation (allogeneic or autologous) or chemotherapy: a cross-sectional study of the EORTC-GIMEMA AML 8A trial., Bone Marrow Transplant, 20, 307, 10.1038/sj.bmt.1700888 Leahey, 1999, Late effects of chemotherapy compared to bone marrow transplantation in the treatment of pediatric acute myeloid leukemia and myelodysplasia., Med Pediatr Oncol, 32, 163, 10.1002/(SICI)1096-911X(199903)32:3<163::AID-MPO1>3.0.CO;2-# Leung, 2000, Late effects of treatment in survivors of childhood acute myeloid leukemia., J Clin Oncol, 18, 3273, 10.1200/JCO.2000.18.18.3273 Parsons, 1999, Quality-adjusted survival after treatment for acute myeloid leukemia in childhood: A Q-TWiST analysis of the Pediatric Oncology Group Study 8821., J Clin Oncol, 17, 2144, 10.1200/JCO.1999.17.7.2144 Goemans, 2008, Outcome for children with relapsed acute myeloid leukemia in the Netherlands following initial treatment between 1980 and 1998: survival after chemotherapy only?, Haematologica, 93, 1418, 10.3324/haematol.12807. Redaelli, 2004, The clinical and epidemiological burden of chronic lymphocytic leukaemia., Eur J Cancer Care (Engl), 13, 279, 10.1111/j.1365-2354.2004.00489.x Barr, 1996, Economic evaluation of allogeneic bone marrow transplantation: a rudimentary model to generate estimates for the timely formulation of clinical policy., J Clin Oncol, 14, 1413, 10.1200/JCO.1996.14.5.1413 Dufoir, 1992, Comparative cost of allogeneic or autologous bone marrow transplantation and chemotherapy in patients with acute myeloid leukaemia in first remission., Bone Marrow Transplant, 10, 323 Welch, 1989, Cost effectiveness of bone marrow transplantation in acute nonlymphocytic leukemia., N Engl J Med, 321, 807, 10.1056/NEJM198909213211207 Uyl-de Groot, 2001, Costs of diagnosis, treatment, and follow up of patients with acute myeloid leukemia in the Netherlands., J Hematother Stem Cell Res, 10, 187, 10.1089/152581601750098499 Remberger, 2007, Similar outcome after unrelated allogeneic peripheral blood stem cell transplantation compared with bone marrow in children and adolescents., Transplantation, 84, 551, 10.1097/01.tp.0000275184.41831.6d Creutzig, 2002, Current controversies: which patients with acute myeloid leukaemia should receive a bone marrow transplantation?–a European view., Br J Haematol, 118, 365, 10.1046/j.1365-2141.2002.03697.x Wheatley, 2002, Current controversies: which patients with acute myeloid leukaemia should receive a bone marrow transplantation?–a statistician's view., Br J Haematol, 118, 351, 10.1046/j.1365-2141.2002.03696.x Gravel, 2007, The intention-to-treat approach in randomized controlled trials: are authors saying what they do and doing what they say?, Clin Trials, 4, 350, 10.1177/1740774507081223