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. (25th September 2020)

• Record Type: Journal Article
• Authors: Ding, Li; Han, Dong‐Mei; Zheng, Xiao‐Li; Yan, Hong‐Min; Xue, Mei; Liu, Jing; Zhu, Ling; Li, Sheng; Mao, Ning; Guo, Zi‐Kuan; Ning, Hong‐Mei; Wang, Heng‐Xiang; Zhu, Heng
• Is Part Of: Stem cells translational medicine. Volume 10:Number 2(2021)
• Page Start: 291
• Page End: 302
• Publication Date: 2020-09-25
• Subjects: bone marrow niche -- graft vs host disease -- HLA‐haploidentical HSCT -- mesenchymal stem cells -- pediatric and adolescent patients -- severe aplastic anemia; Stem cells -- Periodicals; Regenerative medicine -- Periodicals; Periodicals; 616.0277405
• DOI: 10.1002/sctm.20-0345
• Languages: English
• ISSNs: 2157-6564

Abstract

Abstract: 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. Abstract : The cotransplantation of allogenic mesenchymal stem cells (MSCs) and haploidentical hematopoietic stem cells transplantation (haplo‐HSCs) for juvenile severe aplastic anemia (SAA). In the current study, the transplantation‐related mortality rate was 14.72% ± 3.51%. A total of 103 patients have survived with an overall survival (OS) was 87.15% ± 3.3% at the follow‐up of 40 (1.3‐98) months. The OS for the volume of MSC ≥ 1.09 × 10 6 /kg and MSC < 1.09 × 10 6 /kg were 93.12% ± 3.83% and 81.03% ± 5.16% ( P = .08), respectively.