脐血移植治疗WAS的疗效观察

Observation of the effect of umbilical cord blood transplantation in the treatment of WAS

目的:分析湿疹血小板减少伴免疫缺陷综合征(WAS)患儿脐血移植后的疗效和影响因素,为供体选择和受体移植的时机提供经验。方法:5例WAS患儿接受非血缘脐血干细胞移植,分析移植前营养状态、移植时年龄、HLA相合度、移植后移植物抗宿主病(GVHD)及感染因素对移植效果的影响,随访时间截止到2016年1月。结果:5例WAS患儿成功植入。粒细胞植入时间分别为15d、12d、11d、14d、15d,血小板植入时间分别为39d、16d、16d、34d、36d,HLA高分辨检测显示供受体相合度分别为8/10、10/10、5/10、10/10、10/10;其中3例患儿移植前有巨细胞病毒血症,移植后血小板植入缓慢,1例中度营养不良的患儿移植后有皮肤及肠道重度GVHD和反复重症感染。结论:移植前患儿巨细胞病毒血症和移植前营养状态是影响血小板植入和预后的主要因素,HLA高分辨位点相合程度对粒细胞和血小板植入及GVHD没有显著影响。

Objective： To explore the outcome and impacting factors of unrelated donor cord blood transplantation （UCBT） on children with Wiskott-Aldrich syndrome （WAS）. Method： Five patients diagnosed as WAS were treated with UCBT in our center. The factors as the nutritional status prior to transplantation, age of transplantation, HLA matched degree, post-transplantation graft versus host disease （GVHD）, and infection status were analyzed. Follow-up ended by January 2016. Result： Five children with WAS were cured by UCBT. Granulocyte engraftment was 15 days,12 days, 11 days, 14 days and 15 days, respectively. Platelet engraftment was 39 days, 16 days, 16 days, 34 days and 36 days, respectively. HLA high resolution matched between receipt and donor was 8/ 10,10/10,5/10,10/10,10/10, respectively. Three of the patients suffering CMV viremia before transplantation showed a platelet engraftment. One case with severe malnutrition showed severe GVHD of skin and intestine and recurrent severe infections after transplantation. Conclusion：CMV viremia and nutritional status before transplantation influence the platelet engraftment and complications of UCBT. The degree of HLA high resolution has no relations with the engraftment of neutrophil and platelet and complications of UCBT.