亲属单倍体非体外去T细胞造血干细胞移植治疗儿童恶性血液病

Transplantation of haploidentical hematopoietic stem cells from family members without in vitro T-cell depletion for treatment of hematologic malignancies in pediatric patients

目的:评估恶性血液病儿童亲属单倍体造血干细胞移植的安全性及其疗效。方法:58例14岁及以下恶性血液病患儿诊断时均具有提示预后不良的遗传学或临床特征,移植前重新定义高危组与标危组。分析其移植并发症规律及其疗效。结果:58例患儿HLA-A、B、C和DR位点中2个位点配型不合7例(12.1%),3个位点配型不合20例(34.5%),4个位点配型不合31例(53.4%),中位随访时间1 663(752～2 664)d。全部患儿获植入。急性移植物抗宿主病(GVHD)2～4度累计发生率为(54.8±7.6)%,3～4度发生率为(11.4±4.8)%。慢性GVHD累计发生率为(45.6±7.8)%,广泛型为(19.6±6.5)%。38例患者存活,其中33例患者无病存活(LFS)。2年实际总生存高危组和标危组分别为59.0%和78.9%,3年预计LFS分别为(58.6±8.0)%和(68.4±10.7)%。结论:体外非去T细胞亲属间造血干细胞移植用于具有预后不良因素的恶性血液病患儿治疗安全有效。

Objective：To evaluate the efficacy and safety of haploidentical （from family member do- nors） bematopoietic stem cell transplantation （HSCT） for children with hematologic malignancies. Methods：Fifty-eight children under fourteen years with hematological malignancies underwent haploiden- tical HSCT. All patients had poor-risk clinical or cytogenetic features and reclassified into high-risk and standard-risk groups. Transplantation outcomes were analyzed. Results： Of fifty-eight patient/donor pairs, seven （12. 1% ） were mismatched in two HLA loci, twenty （34. 5% ） in three loci, and thirty-one （53.4%） in four loci. Follow-ups were performed for a median of 1 663 （752 -2 664） days after trans- plantation. All patients achieved stable engraftments. The cumulative incidence of acute graft-versus-host disease （GVHD） of grades 2 - 4 was （54. 8 ± 7.6） %, and that of grades 3 - 4 was （ 11.4 ± 4. 8 ） %. The cumulative incidence of chronic GVHD was （45.6 ± 7.8 ） % for the total and （ 19.6 ± 6.5 ） % for the extensive. Thirty-eight patients survived with a 2-year actual overall rate of survival 59.0% and 78.9% in the high-risk and standard-risk group, respectively. The 3-year probability of LFS was （58.6 ± 8.0）% and （68.4 ± 10. 7）%, respectively. Conclusion： The results encourage extending haploidentical HSCT without T-cell depletion treatments to children with an indication for transplantation.