Department of Hematology, the First Affiliated Hospital of Wenzhou Medical University, Wenzhou, People's Republic of China.
Department of Hematology, the First Affiliated Hospital of Wenzhou Medical University, Wenzhou, People's Republic of China; Department of Hematology, the First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, People's Republic of China.
Biol Blood Marrow Transplant. 2019 Aug;25(8):1592-1596. doi: 10.1016/j.bbmt.2019.03.028. Epub 2019 Apr 2.
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only available curative treatment for patients with β-thalassemia major (β-TM). However, the problem of finding a suitable sibling donor with well-matched human leukocyte antigens is still a major obstacle to curing these patients. With the progress in high-resolution HLA typing technology and supportive care, outcomes after allogeneic HSCT from an HLA well-matched unrelated donor (UD) now approach those of well-matched sibling donors. However, UD HSCT is hampered by an increased risk of graft-versus-host disease and transplant-related mortality. Here we report the outcome of transplantation in patients with β-TM using a novel WZ-14-TM transplant protocol, based on cyclophosphamide, intravenous busulfan, fludarabine, and antithymocyte globulin, in our center. Forty-eight patients between 2 and 11 years of age with β-TM received HLA well-matched UD peripheral blood stem cell transplantation following the WZ-14-TM protocol. All of the transplanted patients achieved donor engraftment. The incidences of grade II to IV acute and chronic graft-versus-host disease were 8.3% and 8.3%, respectively. The overall survival and thalassemia-free survival rates were both 100%. This encouraging result suggests that the WZ-14-TM protocol is a feasible and safe conditioning regime for patients with β-TM undergoing UD HSCT.
同种异体造血干细胞移植（HSCT）是治疗重型β地中海贫血（β-TM）患者的唯一有效方法。然而，找到合适的与人类白细胞抗原匹配的同胞供体仍然是治愈这些患者的主要障碍。随着高分辨率 HLA 分型技术和支持性护理的进步，现在来自 HLA 匹配的无关供体（UD）的同种异体 HSCT 的结果接近与匹配的同胞供体的结果。然而，UD HSCT 受到移植物抗宿主病和移植相关死亡率增加的阻碍。在这里，我们报告了我们中心使用基于环磷酰胺、静脉用白消安、氟达拉滨和抗胸腺细胞球蛋白的新型 WZ-14-TM 移植方案治疗β-TM 患者的移植结果。48 名年龄在 2 至 11 岁之间的β-TM 患者根据 WZ-14-TM 方案接受了 HLA 匹配的 UD 外周血造血干细胞移植。所有移植患者均实现了供体植入。Ⅱ至Ⅳ级急性和慢性移植物抗宿主病的发生率分别为 8.3%和 8.3%。总生存率和无地中海贫血生存率均为 100%。这一令人鼓舞的结果表明，WZ-14-TM 方案是接受 UD HSCT 的β-TM 患者可行且安全的预处理方案。
