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A study of human leukocyte antigen‐haploidentical hematopoietic stem cells transplantation combined with allogenic mesenchymal stem cell infusion for treatment of severe aplastic anemia in pediatric and adolescent patients
STEM CELLS Translational Medicine ( IF 6 ) Pub Date : 2020-09-25 , DOI: 10.1002/sctm.20-0345
Li Ding 1, 2 , Dong-Mei Han 1 , Xiao-Li Zheng 1 , Hong-Min Yan 1 , Mei Xue 1 , Jing Liu 1 , Ling Zhu 1 , Sheng Li 1 , Ning Mao 3 , Zi-Kuan Guo 2, 3, 4 , Hong-Mei Ning 3, 5 , Heng-Xiang Wang 1 , Heng Zhu 2, 3, 4, 6
Affiliation  

The clinical applications of human leukocyte antigen (HLA) haploidentical hematopoietic stem cells transplantation (haplo‐HSCT) have offered most of the young severe aplastic anemia (SAA) patients an opportunity to accept curative therapy at the early stage of bone marrow lesions. However, the outcome of juvenile SAA patients received haplo‐HSCT remain to be improved due to high incidence of graft failure and graft vs host disease (GVHD). Mesenchymal stem cells (MSCs) have been characterized by their hematopoiesis‐supporting and immunomodulatory properties. In the current study, we designed a combination of haplo‐HSCT with allogenic MSC for treatment of SAA in pediatric and adolescent patients and evaluated its effects. Juvenile patients (<18 years) with SAA (n = 103) were given HLA‐haploidentical HSC combined with allogenic MSC after a conditioning regimen consisting of busulfan, cyclophosphamide, fludarabine, and antithymocyte globulin and an intensive GVHD prophylaxis, including cyclosporine, short‐term methotrexate, mycophenolate mofetil, and basiliximab. Neutrophil engraftment was achieved in 102 of 103 patients in a median time of 14.3 days (range 9‐25 days). The median time of platelet engraftment was 25.42 days (range 8‐93 days). The cumulative incidence of II‐IV acute GVHD at day +100 was 26.32% ± 0.19% and III‐IV acute GVHD was 6.79% ± 0.06% at day +100, respectively. The cumulative incidence of chronic GVHD was 25.56% ± 0.26%. The overall survival was 87.15% ± 3.3% at a median follow‐up of 40 (1.3‐98) months. Our data suggest that cotransplantation of HLA‐haploidentical HSC and allogenic mesenchymal stem cell may provide an effective and safe treatment for children and adolescents with SAA who lack matched donors.

中文翻译:

人白细胞抗原-半相合造血干细胞移植联合异体间充质干细胞输注治疗儿童和青少年重度再生障碍性贫血的研究

人类白细胞抗原(HLA)单倍体造血干细胞移植(haplo-HSCT)的临床应用为大多数年轻的重度再生障碍性贫血(SAA)患者提供了在骨髓病变早期接受根治性治疗的机会。然而,由于移植物失败和移植物抗宿主病 (GVHD) 的高发生率,青少年 SAA 患者接受 haplo-HSCT 的结果仍有待改善。间充质干细胞 (MSCs) 具有造血支持和免疫调节特性。在目前的研究中,我们设计了一种 haplo-HSCT 与同种异体 MSC 的组合,用于治疗儿科和青少年患者的 SAA,并评估了其效果。青少年患者(< 18 岁)患有 SAA(n = 103)的患者在接受白消安、环磷酰胺、氟达拉滨和抗胸腺细胞球蛋白的预处理方案和包括环孢素、短期甲氨蝶呤、霉酚酸酯在内的强化 GVHD 预防后,给予 HLA 半相合 HSC 联合同种异体 MSC莫非替尼和巴利昔单抗。103 名患者中有 102 名在中位时间 14.3 天(范围 9-25 天)内实现了中性粒细胞植入。血小板植入的中位时间为 25.42 天(范围 8-93 天)。在 +100 天 II-IV 急性 GVHD 的累积发生率分别为 26.32% ± 0.19% 和 III-IV 急性 GVHD 在 +100 天的累积发病率分别为 6.79% ± 0.06%。慢性 GVHD 的累积发生率为 25.56% ± 0.26%。在中位随访 40 (1.3-98) 个月时,总生存率为 87.15% ± 3.3%。
更新日期:2020-09-25
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