Gillio A P, Boulad F, Small T N, Kernan N A, Reyes B, Childs B H, Brochstein J A, Laver J, Castro-Malaspina H, O'Reilly R J
Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY, USA.
Biol Blood Marrow Transplant. 1997 Apr;3(1):18-24.
Children with severe aplastic anemia (SAA) are treated with bone marrow transplantation (BMT) if a human leukocyte antigen (HLA) compatible sibling donor is available, or alternatively with immunosuppressive therapy (IST). Three retrospective trials examining BMT vs IST in pediatric patients treated from 1970-1988 found BMT resulted in a superior survival rate. Advances have been made in general supportive care and in the approach to each of these treatment modalities in the last decade. To compare survival following BMT and IST in a more recent era, we retrospectively analyzed the results of 48 consecutively treated children with SAA presenting to Memorial Sloan-Kettering Cancer Center (MSKCC) between 1983 and 1992. In contrast to the previous studies, the estimated survival of the BMT and IST groups at 120 months are equivalent, 75.6% and 73.8%, respectively. The IST results in our series are superior to the 42-48% (2-10 year) survival previously published, but similar to survival data observed in more recent IST trials employing more intensive immunosuppression (antithymocyte globulin and cyclosporine). The overall BMT survival rates are similar to those previously published, although BMT results improved dramatically during the latter five years of this analysis, with all 11 patients transplanted surviving with a minimum follow-up of 3 years. No surviving BMT patient has extensive chronic graft-versus-host disease (GvHD), a major cause of long-term mortality post-BMT. Therefore, it is likely the BMT survival curve will remain stable. In contrast, the survival curve of the IST patients is likely unstable, since patients are still at risk for relapse or development of clonal disease. Thus, despite overall similar survival rates, we continue to recommend BMT as first-line therapy in pediatric SAA patients with matched sibling donors.
患有严重再生障碍性贫血（SAA）的儿童，如果有人类白细胞抗原（HLA）相匹配的同胞供者，可接受骨髓移植（BMT）治疗，否则接受免疫抑制治疗（IST）。三项回顾性试验比较了1970年至1988年接受治疗的儿科患者中BMT与IST的疗效，发现BMT的生存率更高。在过去十年中，一般支持治疗以及这两种治疗方式的方法都取得了进展。为了比较更近时期BMT和IST后的生存率，我们回顾性分析了1983年至1992年在纪念斯隆凯特琳癌症中心（MSKCC）连续治疗的48例SAA儿童的结果。与之前的研究不同，BMT组和IST组在120个月时的估计生存率相当，分别为75.6%和73.8%。我们系列研究中IST的结果优于之前发表的42%-48%（2至10年）的生存率，但与近期采用更强免疫抑制（抗胸腺细胞球蛋白和环孢素）的IST试验中观察到的生存数据相似。总体BMT生存率与之前发表的相似，尽管在该分析的后五年中BMT的结果有显著改善，所有11例接受移植的患者均存活，最短随访3年。没有存活的BMT患者发生广泛的慢性移植物抗宿主病（GvHD），这是BMT后长期死亡的主要原因。因此，BMT的生存曲线可能会保持稳定。相比之下，IST患者的生存曲线可能不稳定，因为患者仍有复发或发生克隆性疾病的风险。因此，尽管总体生存率相似，我们仍然建议在有匹配同胞供者的儿科SAA患者中，将BMT作为一线治疗方法。
