National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.
Expert Rev Hematol. 2021 May;14(5):449-455. doi: 10.1080/17474086.2021.1923475. Epub 2021 May 10.
: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has resulted in increased levels of disease-free survival in severe aplastic anemia (SAA). Haploidentical transplantation (haplo-SCT) was previously not recommended due to unacceptable incidences of graft-versus-host disease (GvHD) and graft failures. With the advent of intensive GvHD prophylaxis strategies, the outcomes obtained with haplo-SCT for SAA have gradually improved.: A comprehensive search considered PubMed reported articles before 1 February 2021, presented abstracts, and clinical trials pertaining to haplo-HSCT for SAA. This manuscript covers modern approaches with intensive GvHD prophylaxis in haplo-SCT for SAA. The representative methods consist of granulocyte colony stimulating factor (G-CSF) plus ATG-based and posttransplantation cyclophosphamide (PT-Cy)-based protocols.: Currently, haplo-SCT has become a feasible option for treating SAA. The G-CSF/ATG-based protocol included the largest sample size and reported comparable survival rates with identical siblings. The PT-Cy protocol resulted in a relatively lower incidence of GvHD and seemingly poorer but continuously improved engraftment with augmented conditioning. The optimized outcomes are constantly updated with the modification of the conditioning regimen, donor selection, graft source and GvHD prophylaxis. In the future, we should pay more attention to quality of life in addition to survival, and personalized haplo-SCT may improve outcomes.
: 异基因造血干细胞移植(allo-HSCT)可提高重型再生障碍性贫血(SAA)患者的无病生存率。由于移植物抗宿主病(GvHD)和移植物失败的发生率较高,此前不推荐使用单倍体移植(haplo-SCT)。随着强化 GvHD 预防策略的出现,haplo-SCT 治疗 SAA 的结果逐渐改善。: 全面检索考虑了截至 2021 年 2 月 1 日之前在 PubMed 上报告的文章、摘要和与 SAA 的 haplo-HSCT 相关的临床试验。本文涵盖了 SAA 的 haplo-SCT 中强化 GvHD 预防的现代方法。代表性方法包括粒细胞集落刺激因子(G-CSF)联合 ATG 方案和移植后环磷酰胺(PT-Cy)方案。: 目前,haplo-SCT 已成为治疗 SAA 的可行选择。G-CSF/ATG 方案纳入的样本量最大,报告的生存率与同胞供者相同。PT-Cy 方案导致 GvHD 的发生率相对较低,尽管植入情况似乎较差,但随着预处理强度的增加而不断改善。随着预处理方案、供者选择、移植物来源和 GvHD 预防策略的修改,优化结果不断更新。未来,除了生存外,我们还应更加关注生活质量,个体化 haplo-SCT 可能会改善结果。