造血干细胞移植治疗5例Wiskott-Aldrich综合征疗效分析

Analysis of curative effect by hematopoietic stem cell transplantation in the treatment of 5 WiskottAldrich syndrome cases

目的分析造血干细胞移植治疗Wiskott-Aldrich综合征（WAS）患儿的治疗效果。方法回顾分析接受异基因造血干细胞移植治疗的5例WAS患儿的长期随访资料。结果 5例患儿均获得造血功能重建,2例未发生急性移植物抗宿主病（a GVHD）,2例出现Ⅰ~Ⅱ度a GVHD,1例移植后合并肝静脉阻塞性疾病（VOD）死亡,4例平均随访时间30个月,目前健康状况良好。结论造血干细胞移植可有效治疗WAS,积极预防及治疗a GVHD,提高生存质量,可使患儿获得长期生存。

Objective To analyze curative effect by hematopoietic stem cell transplantation in the treatment of Wiskott-Aldrich syndrome（WAS）. Methods Long-term follow-up data of 5 WAS children patients who received allogene hematopoietic stem cell transplantation were retrospectively analyzed. Results All 5 cases had hemopoietic reconstitution. There were 2 cases without acute graft versus host disease（a GVHD）, 2 cases with Ⅰ~Ⅱa GVHD, and 1 death case due to complicated hepatic venous obstructive disease（VOD） after transplantation. Mean follow-up time was 30 months in 4 cases, who were all in good health. Conclusion Hematopoietic stem cell transplantation can effectively treat WAS, prevent a GVHD and improve quality of life, along with long-term survival in patients.