亲缘单倍体造血干细胞移植治疗22例儿童高危血液肿瘤的安全性及疗效分析

The safety and efficacy of hematopoietic stem cell transplantation for 22 children with high-risk hematologic malignancies

目的评估亲缘单倍体造血干细胞移植在儿童高危血液肿瘤治疗中的安全性及疗效。方法回顾性分析22例14岁及以下的高危恶性血液病患儿在接受亲缘单倍体造血干细胞移植后的并发症及疗效。结果全部患儿移植后造血干细胞植入成功。I～Ⅱ度急性移植物抗宿主病（GVHD）发生率为64％（14／22），Ⅲ-Ⅳ度为14％（3／22）；慢性广泛型GVHD发生率为23％（5／22）；6例无GVHD发生。至随访期末，移植早期（〈100d）相关死亡率为O，总生存率为86％（19／22），多因素分析提示移植后原发病的复发为影响总生存率的高危因素（P〈0．05），移植后未出现复发或者复发倾向、出现复发或者复发倾向的两组患儿总生存率分别为94％和60％（P=0．017）。结论亲缘单倍体因造血干细胞移植在高危儿童血液肿瘤的治疗中是安全有效的，亲缘单倍体供者为合适的供者选择。

Objective To evaluate the efficacy and safety of haploidentical （ from family member donors） hematopoietic stem cell transplantation （ HSCT ） for children with high-risk hematologic malignancies. Methods Twenty-two children under fourteen years old withhigh-risk hematological malignancies underwent haploidentical HSCT. Results Follow-ups were performed for a median of 27.4 months （12.2-76.6） after transplantation. All patients achieved stable engraftment. The overall survival rate was 86.4% （19/22） at the end of the follow-up period. There was no early transplant-related mortality occurred. And 4 cases suffered from hGVHD. The cumulative incidence of acute graft-versus-host disease （GVHD） of grades 1-2 and grades 3-4 were 63.6% （ 14/22 ） and 13. 6% （ 3/22 ） respectively. The cumulative incidence of chronic GVHD was 72% （16/22） for the total and 22.7% （ 5/22 ） for the extensive. Neither acute nor chronic GVHD occurred in 6 patients. The analysis by SPSS 19.0 showed that the disease relapse of post-transplantation is the high-risk factor affecting the outcome. Conclusions Haploidentical allogeneie hematopoietic stem cell transplantation for children with high-risk hematologic malignancies is safe and effective, and family member is a suitablehaploid donor selection.