Oevermann L, Sodani Pietro
Department of Pediatric Oncology & Hematology, Charité University Medicine, Berlin, Germany.
Department of Pediatric Oncology & Hematology, Charité University Medicine, Berlin, Germany.
Hematol Oncol Stem Cell Ther. 2020 Jun;13(2):116-119. doi: 10.1016/j.hemonc.2019.12.004. Epub 2020 Mar 12.
This mini review is based on an oral presentation reflecting the current status quo of allogeneic hematopoietic stem cell transplantation (HSCT) for patients with sickle cell disease (SCD) using matched unrelated donors (MUDs) presented at the EBMT Sickle Disease Meeting held in Regensburg, Germany, in May 2019. Although the clinical trial landscape for MUD HSCT in patients with SCD is limited to date, some attempts to improve patient outcome in terms of overall survival and event-free survival have been made recently, including optimization of conditioning regimens and prevention of engraftment failure as well as graft-versus-host disease. The results achieved by these approaches are summarized in this review and are still unsatisfactory. Whether new haploidentical transplantation protocols will achieve superior results and are able to replace MUD HSCT for patients with SCD remains to be elucidated.
本综述基于一次口头报告，该报告反映了2019年5月在德国雷根斯堡举行的欧洲血液与骨髓移植协会（EBMT）镰状细胞病会议上所呈现的使用匹配无关供者（MUD）对镰状细胞病（SCD）患者进行异基因造血干细胞移植（HSCT）的现状。尽管迄今为止，SCD患者MUD HSCT的临床试验情况有限，但最近已在尝试从总生存和无事件生存方面改善患者结局，包括优化预处理方案、预防植入失败以及移植物抗宿主病。本综述总结了这些方法所取得的结果，而这些结果仍不尽人意。新的单倍型相合移植方案能否取得更好的结果并能够替代SCD患者的MUD HSCT仍有待阐明。
