Tannumsaeung Supavich, Anurathapan Usanarat, Pakakasama Samart, Pongpitcha Pongpak, Songdej Duantida, Sirachainan Nongnuch, Andersson Borje S, Hongeng Suradej
Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Krung Thep Maha Nakhon (Bangkok), Thailand.
Department of Stem Cell Transplantation, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Eur J Haematol. 2023 Mar;110(3):305-312. doi: 10.1111/ejh.13906. Epub 2022 Dec 7.
Patients with high-risk hematologic diseases require intensive modalities, including high-dose chemotherapy and allogeneic hematopoietic stem cell transplantation (allo-HSCT). Haploidentical T-cell-replete transplantation is a logical choice because of the limited availability of matched sibling donors and the prolonged time needed to identify matched unrelated donors in Thailand.
The clinical outcomes data of 43 patients undergoing allo-HSCT were reviewed. All patients had high-risk hematologic malignancies, were younger than 20 years, and were in complete cytological remission at the time of allo-HSCT. We used two different conditioning regimens: total body irradiation (TBI) combined with cyclophosphamide, fludarabine, and melphalan (n = 23) and thiotepa combined with fludarabine and busulfan (n = 20). All patients received a graft-versus-host disease prophylaxis regimen consisting of cyclophosphamide, mycophenolate mofetil, and a calcineurin inhibitor or sirolimus.
There was no difference in engraftment between patients receiving either of the regimens. After a median follow-up of 35.8 (range, 0.6-106.2) months, the overall survival (OS) and event-free survival (EFS) rates were 62.4% and 54.7%, respectively. OS and EFS were comparable between the respective regimens.
We conclude that thiotepa-based conditioning has similar efficacy and tolerability as TBI-based conditioning for haploidentical HSCT with post-transplant cyclophosphamide.
高危血液病患者需要强化治疗方式，包括大剂量化疗和异基因造血干细胞移植（allo-HSCT）。由于泰国同胞全相合供体数量有限以及寻找非血缘全相合供体所需时间较长，单倍体相合且富含T细胞的移植是一种合理选择。
回顾了43例行allo-HSCT患者的临床结局数据。所有患者均患有高危血液系统恶性肿瘤，年龄小于20岁，且在allo-HSCT时处于完全细胞学缓解状态。我们采用了两种不同的预处理方案：全身照射（TBI）联合环磷酰胺、氟达拉滨和美法仑（n = 23）以及塞替派联合氟达拉滨和白消安（n = 20）。所有患者均接受了由环磷酰胺、霉酚酸酯和钙调神经磷酸酶抑制剂或西罗莫司组成的移植物抗宿主病预防方案。
接受两种方案之一的患者在植入方面无差异。中位随访35.8（范围0.6 - 106.2）个月后，总生存（OS）率和无事件生存（EFS）率分别为62.4%和54.7%。各方案之间的OS和EFS相当。
我们得出结论，对于单倍体相合HSCT联合移植后环磷酰胺治疗，基于塞替派的预处理与基于TBI的预处理具有相似的疗效和耐受性。
