Shanghai Institute of Hematology, State Key Laboratory for Medical Genomics, National Research Center for Translational Medicine, Blood and Marrow Transplantation Center, Department of Hematology, Collaborative Innovation Center of Hematology, Ruijin Hospital affiliated to School of Medicine, Shanghai Jiao Tong University, Shanghai, China.
Shanghai Clinical Research Center (SCRC), Fenglin International Centre, Shanghai, China.
Bone Marrow Transplant. 2024 Jul;59(7):1006-1011. doi: 10.1038/s41409-024-02277-4. Epub 2024 Apr 10.
While post-transplant cyclophosphamide (PTCy) is commonly used as graft-versus-host disease (GvHD) prophylaxis in haploidentical stem cell transplantation (haplo-HSCT), its dose remains a matter of debate due to side effect concerns. Standard dose of 100 mg/kg associated with tacrolimus and post-engraftment anti-thymocyte globulin (ATG) was used as the reference GvHD prophylaxis in our center and had demonstrated encouraging results. Though PTCy 80 mg/kg was shown to be feasible in patients in reduced-intensity conditioning, whether it exerts equivalent GvHD prophylactic efficacy in myeloablative conditioning (MAC) setting has not been confirmed. Here, we retrospectively analyzed the efficacy and safety of PTCy 80 mg/kg combined with tacrolimus and post-engraftment ATG as GvHD prophylaxis in patients aged more than 55 years or with cardiac antecedents or HCT-CI score >2 undergoing haplo-HSCT with MAC. The cumulative incidence of grade III-IV aGvHD at day 100 and moderate-to-severe cGvHD at 1 year was 4.8% ± 3.4% and 19.9% ± 7.0%, respectively. When compared with patients receiving the reference regimen, patients from the PTCy 80 mg/kg group had similar incidence of GvHDs and survival as their younger counterparts. Thus, PTCy 80 mg/kg seems to be feasible for patients treated with MAC conditioning regimens in haplo-HSCT, inviting further investigation notably in frail patients.
在单倍体造血干细胞移植(haplo-HSCT)中,环磷酰胺(PTCy)常用于移植物抗宿主病(GvHD)的预防,但由于副作用的担忧,其剂量仍存在争议。本中心采用标准剂量 100mg/kg 联合他克莫司和移植后抗胸腺细胞球蛋白(ATG)作为 GvHD 预防方案,取得了令人鼓舞的结果。虽然在接受低强度预处理的患者中使用 80mg/kg PTCy 是可行的,但在骨髓清除性预处理(MAC)方案中,其是否具有等效的 GvHD 预防效果尚未得到证实。在此,我们回顾性分析了 PTCy 80mg/kg 联合他克莫司和移植后 ATG 作为 GvHD 预防方案,用于接受 MAC 预处理、年龄>55 岁或有心脏病史或 HCT-CI 评分>2 的患者的疗效和安全性。第 100 天 III-IV 级急性 GvHD 和 1 年时中重度慢性 GvHD 的累积发生率分别为 4.8%±3.4%和 19.9%±7.0%。与接受参考方案的患者相比,PTCy 80mg/kg 组患者与年轻患者相比,GvHD 发生率和生存情况相似。因此,PTCy 80mg/kg 似乎适用于接受 MAC 预处理方案的 haplo-HSCT 患者,特别是在体弱患者中值得进一步研究。