广西医学

目的探讨基于移植后环磷酰胺（PTCy）的移植物抗宿主病（GVHD）预防方案在地中海贫血患儿单倍体造血干细胞移植中的应用效果。方法回顾性分析28例接受单倍体造血干细胞移植的地中海贫血患儿的临床资料。所有患儿均接受白消安+环磷酰胺+氟达拉滨+抗人胸腺细胞免疫球蛋白（ATG）联合PTCy+吗替麦考酚酯+环孢素方案以预防GVHD。评估患儿的植入效果、 GVHD发生情况、感染及死亡情况、总体生存率及无事件生存率。结果（1）28例患儿的移植物CD34+细胞数、总有核细胞数、单个核细胞数中位值分别为11.2×106/kg、17.6×108/kg、5.1×108/kg，植入成功率为100%，粒系植入和巨核系植入的中位时间分别为14 d和24 d，植入功能不良发生率为7.1%。（2）GVHD的发生率为28.5%，其中Ⅰ~Ⅱ型、Ⅲ~ Ⅳ型急性GVHD的发生率分别为7.1%、14.3%，慢性GVHD的发生率为7.1%。（3）巨细胞病毒感染、Epstein⁃Barr 病毒感染、真菌感染的发生率分别为64.3%、10.7%、21.4%，共死亡2例（7.1%），患儿的3年总生存率和3年无事件生存率均为92.9%。结论在地中海贫血患儿行单倍体造血干细胞移植时，白消安+环磷酰胺+氟达拉滨+ATG联合PTCy+吗替麦考酚酯+环孢素方案是一个安全性较高，能有效降低移植后GVHD发生率且不影响植入效果的选择。但移植后感染等并发症依然是临床上面临的严峻问题，需要继续探索更为优化的方案。

ion cyclophosphamide (PTCy) in haploid hematopoietic stem cell transplantation in children with thalassemia. Methods The clinical data of 28 children with thalassemia who received haploid hematopoietic stem cell transplantation were retrospectively analyzed. All children received regimen of busulfan+cyclophosphamide+fludarabine+anti⁃human thymocyte globulin (ATG) combined with PTCy+mycophenolate mofetil+cyclosporin to prevent GVHD. The transplantation effect, GVHD occurrence, infection and death, overall survival rate, and event⁃free survival rate were evaluated in children. Results (1) The median values of CD34+ cell number, total nucleated cell number, and mononuclear cell number were 11.2×106/kg, 17.6×108/kg, and 5.1×108/kg in graft of 28 children, with the engraftment success rate of 100%, and the median time of neutrophil engraftment was 14 days and 24 days for platelet engraftment, respectively. The incidence rate of engraftment dysfunction was 7.1%. (2) The incidence rate of GVHD was 28.5%, therein the incidence rates of acute GVHD in types Ⅰ-Ⅱ and Ⅲ-Ⅳ were 7.1% and 14.3%, respectively, and the incidence rate of chronic GVHD was 7.1%. (3) The incidence rate of cytomegalovirus infection, Epstein⁃Barr virus infection, and fungal infection were 64.3%, 10.7%, and 21.4%, respectively, and 2 children (7.1%) were dead in total; moreover, the 3⁃year overall survival rate and 3⁃year event⁃free survival rate were all 92.9% in children. Conclusion For children with thalassemia undergoing haploid hematopoietic stem cell transplantation, busulfan+cyclophosphamide+fludarabine+ATG combined with PTCy+mycophenolate mofetil+cyclosporin regimen is a choice of relatively high safety that can effectively decrease the post⁃transplantation GVHD incidence rate, and dose not affect engraftment effect. However, complications such as infection after transplantation are still serious clinical problems, and more optimized regimens need to be explored.