Allogeneic Hematopoietic Cell Transplantation for Fanconi Anemia in Patients With Pretransplantation Cytogenetic Abnormalities, Myelodysplastic Syndrome, or Acute Leukemia
Allogeneic Hematopoietic Cell Transplantation for Fanconi Anemia in Patients With Pretransplantation Cytogenetic Abnormalities, Myelodysplastic Syndrome, or Acute Leukemia
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DOI：
10.1200/jco.2012.45.9719
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发表时间：
2013-05-01
影响因子：
45.3
通讯作者：
Gale, Robert Peter
中科院分区：
文献类型：
--
作者：
Ayas, Mouhab;Saber, Wael;Gale, Robert Peter
目的异基因造血干细胞移植（HCT）可治疗范可尼贫血（FA）患者的骨髓衰竭.患者和方法我们分析了113例FA患者的数据，这些患者有细胞遗传学异常（n = 54），MDS（n = 45），结果1985 ~ 2007年国际血液和骨髓移植研究中心报告的14例急性白血病患者中，中性粒细胞恢复率分别为78%和85分别在第28天和第100天。第100天急性移植物抗宿主病B至D级和C至D级的累积发生率分别为26%（95% CI，19%至35%）和12%（95% CI，7%至19%）。1年、3年和5年的生存概率分别为64%（95% CI，55%-73%）、58%（95% CI，48%-67%）和55%（95% CI，45%-64%）。在单变量分析中，年龄越小，5年生存率越上级（14年：69% [95%CI，57%至80%] vs 39% [95%CI，26%至53%]; P = 0.001）。在来自HLA匹配的亲属供体的移植中（n = 82），（14岁：分别为78% [95% CI，64%至90%]和34% [95% CI，20%至50%]; P <0.001）和仅细胞遗传学异常患者与MDS/急性白血病患者相比（67% [95%CI，52%至81%] v 43% [95%CI，27%至59%]; P = 0.03）具有优越的上级5年生存率。年轻的患者和HLA匹配的亲属供者移植的受者只有细胞遗传学异常才有最好的生存率。J Clin Oncol 31：1669-1676. (C)2013年美国临床肿瘤学会
PurposeAllogeneic hematopoietic cell transplantation (HCT) can cure bone marrow failure in patients with Fanconi anemia (FA). Data on outcomes in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome (MDS), or acute leukemia have not been separately analyzed.Patients and MethodsWe analyzed data on 113 patients with FA with cytogenetic abnormalities (n = 54), MDS (n = 45), or acute leukemia (n = 14) who were reported to the Center for International Blood and Marrow Transplant Research from 1985 to 2007.ResultsNeutrophil recovery occurred in 78% and 85% of patients at days 28 and 100, respectively. Day 100 cumulative incidences of acute graft-versus-host disease grades B to D and C to D were 26% (95% CI, 19% to 35%) and 12% (95% CI, 7% to 19%), respectively. Survival probabilities at 1, 3, and 5 years were 64% (95% CI, 55% to 73%), 58% (95% CI, 48% to 67%), and 55% (95% CI, 45% to 64%), respectively. In univariate analysis, younger age was associated with superior 5-year survival ( 14 years: 69% [95% CI, 57% to 80%] v 39% [95% CI, 26% to 53%], respectively; P = .001). In transplantations from HLA-matched related donors (n = 82), younger patients ( 14 years: 78% [95% CI, 64% to 90%] v 34% [95% CI, 20% to 50%], respectively; P < .001) and patients with cytogenetic abnormalities only versus MDS/acute leukemia (67% [95% CI, 52% to 81%] v 43% [95% CI, 27% to 59%], respectively; P = .03) had superior 5-year survival.ConclusionOur analysis indicates that long-term survival for patients with FA with cytogenetic abnormalities, MDS, or acute leukemia is achievable. Younger patients and recipients of HLA-matched related donor transplantations who have cytogenetic abnormalities only have the best survival. J Clin Oncol 31: 1669-1676. (C) 2013 by American Society of Clinical Oncology