Unrelated stem cell transplantation for severe acquired aplastic anemia: improved outcome in the era of high-resolution HLA matching between donor and recipient
Unrelated stem cell transplantation for severe acquired aplastic anemia: improved outcome in the era of high-resolution HLA matching between donor and recipient
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DOI：
10.3324/haematol.10899
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发表时间：
2007-05-01
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影响因子：
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通讯作者：
Socie, Gerard
中科院分区：
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作者：
Maury, Sebastien;Balere-Appert, Marie-Lorraine;Socie, Gerard
研究背景与目的重型获得性再生障碍性贫血（severe acquired aplastic anemia，SAA）是一种潜在的致命性骨髓衰竭综合征，主要发生于儿童和青壮年。免疫抑制方案和造血干细胞移植（HSCT）是仅有的两种可用的治愈性治疗。缺乏HILA相同同胞供体的患者可能会接受来自无关供体的HSCT，这是一种历史上与高死亡率相关的策略。因此，对于免疫抑制方案难治的患者，从无关供体移植干细胞的决定与支持性治疗相权衡，通常代表医生的两难境地。我们的目的是确定无关HSCT后的结果是否有所改善，在最近几年，如果是这样的话，以确定负责的improvement.Design和MethodsWe的89例患者（中位年龄17岁，范围0-52）与收购SAA从无关供体HSCT从1989年和2004年之间的结果进行了分析。连续的情况下，法国登记处（SFGM-TC）由25 centers.ResultsPatients移植在两个连续的时间段（1989-1998年和19992004年）有不同的5年生存概率（95%置信区间）：29 +/- 7%和507%，分别（P < 0.01）。两个队列之间的主要差异涉及供体和受体之间在10个HLA-A、-B、-C、-DRB 1和-DQB 1抗原的等位基因水平上的HILA匹配，这在1999-2004年比前一时期更频繁（p=0.0004）。在多变量分析中，影响生存率的仅有两个因素是HLA等位基因匹配（p < 0.01）和受体年龄较小（+/-17岁，p < 0.0001）。生存率达到78 +/- 11%，在5年的年轻，完全HLA匹配patients.Interpretation和ConclusionsSurvival无关HSCT SAA后显着改善，在过去的15年中，主要是由于更好的HLA匹配。与其供体在等位基因水平上完全HILA匹配的年轻患者的结果与从相关供体进行HSCT后观察到的结果相当。
Background and ObjectivesSevere acquired aplastic anemia (SAA) is a potentially fatal bone marrow failure syndrome occurring mainly in children and young adults. Immunosuppressive regimens and hematopoietic stem cell transplantation (HSCT) are the only two available curative treatments. Patients who lack an HILA-identical sibling donor may receive HSCT from an unrelated donor, a strategy historically associated with high mortality rates. Thus, for patients refractory to immunosuppressive regimens, the decision to transplant stem cells from unrelated donors is weighed against supportive care and often represents a dilemma for physicians. We aimed to determine whether outcome after unrelated HSCT has improved in recent years and, if so, to determine the factors responsible for the improvement.Design and MethodsWe analyzed the outcome of 89 patients (median age 17 years, range 0-52) with acquired SAA undergoing HSCT from an unrelated donor between 1989 and 2004. Cases were consecutively reported to the French Registry (SFGM-TC) by 25 centers.ResultsPatients transplanted during two successive time-periods (1989-1998 and 19992004) had different 5-year survival probabilities (95% confidence interval): 29 +/- 7% and 50 7%, respectively (p < 0.01). The main difference between the two cohorts concerned HILA matching between donors and recipients at the allelic level for the ten HLA-A, -B, -C, -DRB1 and -DQB1 antigens, which was more frequent in 1999-2004 than in the former period (p=0.0004). In multivariate analysis, the only two factors affecting survival were HLA allelic matching (p < 0.01) and younger age of recipient (+/- 17 years, p < 0.0001). Survival reached 78 +/- 11% at 5 years for the younger, fully HLA-matched patients.Interpretation and ConclusionsSurvival after unrelated HSCT for SAA has improved significantly over the past 15 years, mainly due to better HLA matching. Results for young patients who are fully HILA-matched at the allelic level with their donor are comparable to those observed after HSCT from a related donor.