Monacord, Centre Scientifique de Monaco, Monaco; Eurocord, Hôpital Saint Louis, Universite Paris Diderot, Paris, France.
Monacord, Centre Scientifique de Monaco, Monaco; Eurocord, Hôpital Saint Louis, Universite Paris Diderot, Paris, France.
Hematol Oncol Stem Cell Ther. 2020 Dec;13(4):181-188. doi: 10.1016/j.hemonc.2019.12.011. Epub 2020 Mar 16.
Allogeneic hematopoietic stem cell transplantation (HSCT) is, to date, the only curative treatment for sickle cell disease (SCD). Because an human leukocyte antigen (HLA)-matched sibling donor is not always available, alternative stem cell sources such as unrelated or haploidentical related donors have been explored. To date, few series of SCD patients transplanted with an unrelated donor, cord blood, and haploidentical related donor have been reported, but the high rates of rejection and chronic graft versus host disease have limited their widespread application. We describe the outcomes of a retrospective, registry-based, survey on 144 alternative donor HSCT performed for SCD in 30 European Society for Blood and Marrow Transplantation centers between 1999 and 2017. Data on 70 unrelated adult donors (49%), six cord blood (4%), and 68 haploidentical donors (47%; including post-HSCT Cy, ex vivo T-cell depleted, and other haplo-HSCTs) were reported and missing information was updated by the centers. Overall, 16% patients experienced graft failure, Grade II-IV acute GVHD at 100 days was 24%, whereas Grade III-IV was 10%. Chronic GVHD was observed in 24% (limited for 13 patients and extensive for 18 patients). Overall, the 3-year overall survival (OS) was 86% ± 3% and 3-year event-free survival (EFS; considering death and graft failure as events) was 72% ± 4%. We therefore conclude that alternative donor HSCT for SCD can be feasible but efforts in decreasing relapse and GVHD should be promoted to increase its safe and successful utilization. Moreover, a better knowledge of HLA matching and the tailoring of conditioning could help improve EFS and OS.
同种异体造血干细胞移植（HSCT）是目前治疗镰状细胞病（SCD）的唯一根治方法。由于人类白细胞抗原（HLA）匹配的同胞供体并非总是可用，因此已经探索了替代干细胞来源，如无关供体或半相合相关供体。迄今为止，已有少数 SCD 患者接受无关供体、脐带血和半相合相关供体移植的系列报道，但排斥反应和慢性移植物抗宿主病的高发生率限制了其广泛应用。我们描述了 30 个欧洲血液和骨髓移植学会中心在 1999 年至 2017 年间进行的 144 例 SCD 替代供体 HSCT 的回顾性、基于登记的调查结果。报告了 70 例无关成人供体（49%）、6 例脐带血（4%）和 68 例半相合供体（47%；包括 HSCT 后 Cy、体外 T 细胞耗竭和其他半相合 HSCT）的数据，中心更新了缺失信息。总体而言，16%的患者发生移植物失败，100 天的 II-IV 级急性移植物抗宿主病为 24%，而 III-IV 级为 10%。观察到慢性移植物抗宿主病 24%（13 例局限性和 18 例广泛性）。总体而言，3 年总生存率（OS）为 86%±3%，3 年无事件生存率（EFS；考虑死亡和移植物失败为事件）为 72%±4%。因此，我们得出结论，替代供体 HSCT 治疗 SCD 是可行的，但应努力降低复发和移植物抗宿主病的风险，以提高其安全和成功应用。此外，更好地了解 HLA 匹配和调整调理方案可能有助于提高 EFS 和 OS。
