Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade:: a report from the European Group for Blood and Marrow Transplantation
Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade:: a report from the European Group for Blood and Marrow Transplantation
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DOI：
10.3324/haematol.10075
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发表时间：
2007-01-01
期刊：
影响因子：
--
通讯作者：
Fuhrer, Monika
中科院分区：
文献类型：
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作者：
Locasciulli, Anna;Oneto, Rosi;Fuhrer, Monika
背景与目的获得性再生障碍性贫血（acquired aplastic anemia，AA）的治疗主要依靠异基因骨髓移植（alloy bone marrow transplantation，BMT）和免疫抑制剂治疗。本研究的目的是评估过去十年中接受AA治疗的儿童和成人的结果，并确定结果是否在两个连续时间段内有所改善：设计和方法我们研究了2479例连续的AA患者，根据一线治疗，BMT（n=1567）或免疫抑制治疗（n=912）分类，并根据两个连续的时间段分层。分析包括变量相关的患者，疾病和transplant.Results精算10年生存率分别为73%和68%的BMT或免疫抑制治疗的患者，分别（p=0.002）。骨髓移植的结果随着时间的推移而显著改善（69%和77%，p= 0.001），对于匹配的同胞供体（MSD）（74%和80%; p=0.003）和替代供体（38%和65%，p=0.0001）移植，儿童移植更好（79%比68%，p < 0.0001）。在多变量分析中，有利的预测因素（p < 0.001）是年龄较小、1996年以后移植、MDS、诊断-移植间隔时间短和未接受过放疗。接受免疫抑制治疗的患者随时间推移无显著改善（69%和73% p=0.29）。儿童的生存率明显更高（81% vs 70%，p=0.001），尤其是重度AA患者（83% vs 62%，p=0.0002）。联合免疫抑制上级单一药物治疗（77%比62%，p=0.002）。在多变量分析中，免疫抑制治疗后生存率的显著预测因素为年龄≥ 16岁（p=0.0009），诊断-治疗间隔时间较长（p=0.04），单药与联合免疫抑制（p=0.02）.解释和结论AA患者亚组的结果有所改善：接受一线骨髓移植的患者和接受免疫抑制治疗的极重度AA儿童。年龄仍然是两种治疗后结局的主要预测因素。早期干预与显著更好的结果相关，强烈推荐，无论一线治疗如何。
Background and Objectives The treatment of acquired aplastic anemia (AA) is based on allogeneic bone marrow transplantation (BMT) and immunosuppressive therapy. The aim of this study was to assess the outcome of children and adults with AA treated in the last decade, and to determine whether results have improved in two sequential time periods: 1991-1996 and 1997-2002.Design and Methods We studied 2479 consecutive patients with AA, classified according to first-line treatment, BMT (n=1567) or immunosuppressive therapy (n=912), and stratified according to two sequential time periods. Analyses included variables related to the patients, disease and transplant.Results The actuarial 10-year survival was 73% and 68% for patients treated with BMT or immunosuppression, respectively (p=0.002). BMT outcome improved significantly with time (69% and 77%, p=001) for both matched sibling donor (MSD) (74% and 80%; p=0.003) and alternative donor (38% and 65% p=0.0001) transplants, and was better in children (79% versus 68%, p < 0.0001). In multivariate analysis favorable predictors (p < 0.001) were younger age, transplant after 1996, a MDS, ashort diagnosis-transplant interval, and no irradiation. There was no significant improvement over time for patients receiving immunosuppressive therapy (69% and 73% p=0.29). Survival was significantly better in children (81% versus 70%, p=0.001), especially in those with severe AA (83% versus 62%, p=0.0002). Combined immunosuppression was superior to single drug treatment (77% versus 62%, p=0.002). In multivariate analysis significant predictors of survival following immunosuppressive treatment were age >= 16 years (p=0.0009), longer interval between diagnosis -treatment (p=0.04), and single drug versus combined immunosuppression (p=0.02).Interpretation and Conclusions Outcome has improved in subsets of AA patients: those receiving first-line BMT and children with very severe AA treated with immunosuppression. Age remains a major predictor of outcome following both treatments. Early intervention is associated with a significantly better outcome and is strongly recommended, whatever the firstline therapy.