Peripheral-blood stem cells versus bone marrow from unrelated donors.
Peripheral-blood stem cells versus bone marrow from unrelated donors.
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DOI：
10.1056/nejmoa1203517
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发表时间：
2012-10-18
期刊：
影响因子：
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通讯作者：
Blood and Marrow Transplant Clinical Trials Network
中科院分区：
文献类型：
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作者：
Anasetti C;Logan BR;Lee SJ;Waller EK;Weisdorf DJ;Wingard JR;Cutler CS;Westervelt P;Woolfrey A;Couban S;Ehninger G;Johnston L;Maziarz RT;Pulsipher MA;Porter DL;Mineishi S;McCarty JM;Khan SP;Anderlini P;Bensinger WI;Leitman SF;Rowley SD;Bredeson C;Carter SL;Horowitz MM;Confer DL;Blood and Marrow Transplant Clinical Trials Network
随机试验表明，与骨髓移植相比，从HLA相同的兄弟姐妹中移植非格司亭动员的外周血干细胞加速了植入，但增加了急性和慢性移植物抗宿主病（GVHD）的风险。一些研究还表明，外周血干细胞与高危白血病患者复发率降低和生存率提高有关。我们进行了一项3期、多中心、随机试验，比较了外周血干细胞移植与非亲缘供者骨髓移植的2年生存率，并采用意向治疗分析。在2004年3月至2009年9月期间，我们在48个中心招募了551名患者。患者按1：1的比例随机分配接受外周血干细胞或骨髓移植，并根据移植中心和疾病风险分层。存活患者的中位随访时间为36个月（四分位距，30 - 37）。外周血组的2年总生存率为51%（95%置信区间[CI]，45 - 57），而骨髓组为46%（95% CI，40 - 52）（P = 0.29），绝对差异为5个百分点（95% CI，-3至14）。外周血组移植物衰竭的总发生率为3%（95%CI，1 - 5），而骨髓组为9%（95%CI，6 - 13）（P = 0.002）。外周血组2年慢性GVHD的发生率为53%（95%CI，45 ~ 61），而骨髓组为41%（95%CI，34 ~ 48）（P = 0.01）。急性GVHD或复发的发生率没有显著的组间差异。我们没有发现无关供体的外周血干细胞和骨髓移植之间存在显着的生存差异。对次要终点的探索性分析表明，外周血干细胞可降低移植物衰竭的风险，而骨髓可降低慢性GVHD的风险。（由国家心脏、肺和血液研究所-国家癌症研究所和其他机构资助; ClinicalTrials.gov编号，NCT 00075816。
Randomized trials have shown that the transplantation of filgrastim-mobilized peripheral-blood stem cells from HLA-identical siblings accelerates engraftment but increases the risks of acute and chronic graft-versus-host disease (GVHD), as compared with the transplantation of bone marrow. Some studies have also shown that peripheral-blood stem cells are associated with a decreased rate of relapse and improved survival among recipients with high-risk leukemia. We conducted a phase 3, multicenter, randomized trial of transplantation of peripheral-blood stem cells versus bone marrow from unrelated donors to compare 2-year survival probabilities with the use of an intention-to-treat analysis. Between March 2004 and September 2009, we enrolled 551 patients at 48 centers. Patients were randomly assigned in a 1:1 ratio to peripheral-blood stem-cell or bone marrow transplantation, stratified according to transplantation center and disease risk. The median follow-up of surviving patients was 36 months (interquartile range, 30 to 37). The overall survival rate at 2 years in the peripheral-blood group was 51% (95% confidence interval [CI], 45 to 57), as compared with 46% (95% CI, 40 to 52) in the bone marrow group (P = 0.29), with an absolute difference of 5 percentage points (95% CI, −3 to 14). The overall incidence of graft failure in the peripheral-blood group was 3% (95% CI, 1 to 5), versus 9% (95% CI, 6 to 13) in the bone marrow group (P = 0.002). The incidence of chronic GVHD at 2 years in the peripheral-blood group was 53% (95% CI, 45 to 61), as compared with 41% (95% CI, 34 to 48) in the bone marrow group (P = 0.01). There were no significant between-group differences in the incidence of acute GVHD or relapse. We did not detect significant survival differences between peripheral-blood stem-cell and bone marrow transplantation from unrelated donors. Exploratory analyses of secondary end points indicated that peripheral-blood stem cells may reduce the risk of graft failure, whereas bone marrow may reduce the risk of chronic GVHD. (Funded by the National Heart, Lung, and Blood Institute–National Cancer Institute and others; ClinicalTrials.gov number, NCT00075816.)