异基因造血干细胞移植治疗儿童重型再生障碍性贫血疗效分析

Efficacy Analysis of Allogeneic Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia

目的:本研究旨在探讨异基因造血干细胞移植在儿童重型再生障碍性贫血(SAA)治疗中的作用。方法:本院11例SAA患儿中7例行HLA相合同胞供者异基因造血干细胞移植,2例行脐血移植,2例行亲缘单倍体相合造血干细胞移植。结果:10例患儿原发性植入成功,3例患儿继发性植入失败,进行供者淋巴细胞输注,供体都稳定植入,造血恢复。1例单份脐血移植患儿原发性植入失败,但自体造血恢复。9例接受亲缘相关供体移植的患儿,中性粒细胞>0.5×109/L时间为10-19 d,中位时间14d,血小板>20×109/L时间为8-42 d,中位时间17 d;双份脐血移植的患儿,中性粒细胞>0.5×109/L时间为16 d,血小板>20×109/L时间为41 d。3例患儿出现了不同程度的急性GVHD,2例患儿发生了可控的局限性慢性GVHD(c GVHD)。4例患儿发生了CMV病毒血症,2例发展为CMV肺炎而死亡。3例患儿发生了EB病毒血症,1例发展为EB病毒肺炎。结论:如有HLA相合的同胞供者,异基因造血干细胞移植可作为儿童SAA的首选治疗;联合免疫抑制治疗无效或无HLA相合同胞供者,可考虑行选择性供体移植,如脐血移植或亲缘单倍体造血干细胞移植;供者淋巴细胞输注可促进移植物植入。

Objective: To study the efficacy of allogeneic hematopoietic stem cell transplantation( allo-HSCT) in children with severe aplastic anemia( SAA). Methods: A total of 11 children with SAA were treated with HLA matched siblings( n = 7),umbilical cord blood( n = 2) and haploidentical HSCT( n = 2). Results: Among 11 children patients,10 patients achieved engraftment,but 3 children patients experienced secondary graft failure,after donor lymphocyte infusions( DLI),they achieved engraftment again. One patient received cord blood transplantation and experienced primary graft rejection,but acquired autologous recovery. The median time for neutrophils to reach over 0. 5 × 109/ L was 14days( 10-19 days) in the 9 children received bone marrowor bone marrowand peripheral blood allo-HSCT,while the median time for platelets to reach over 20 × 109/ L was 17 days( 8-42 days). For the patient received double cord blood transplantation,the time of neutrophile and platelet level recovery was 16 days and 41 days,respectively. Conclusion: If HLA-matched sibling donor is available,allo-HSCT can be recommended as the first line of treatment for children with SAA. It is feasible for children with SAA to receive allo-HSCT from selective donor,including cord blood and haploidentical HSCT. Donor lymphocyte infusions can improve engraftment.