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
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,...
Main Authors: | , , , , , , , , , , , , |
---|---|
Format: | Article |
Language: | English |
Published: |
Wiley
2021-02-01
|
Series: | Stem Cells Translational Medicine |
Subjects: | |
Online Access: | https://doi.org/10.1002/sctm.20-0345 |
id |
doaj-827debe97bb04ab9b1442a7e1be64b94 |
---|---|
record_format |
Article |
spelling |
doaj-827debe97bb04ab9b1442a7e1be64b942021-02-10T11:40:43ZengWileyStem Cells Translational Medicine2157-65642157-65802021-02-0110229130210.1002/sctm.20-0345A 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 patientsLi Ding0Dong‐Mei Han1Xiao‐Li Zheng2Hong‐Min Yan3Mei Xue4Jing Liu5Ling Zhu6Sheng Li7Ning Mao8Zi‐Kuan Guo9Hong‐Mei Ning10Heng‐Xiang Wang11Heng Zhu12Air Force Medical Center, PLA Beijing People's Republic of ChinaAir Force Medical Center, PLA Beijing People's Republic of ChinaAir Force Medical Center, PLA Beijing People's Republic of ChinaAir Force Medical Center, PLA Beijing People's Republic of ChinaAir Force Medical Center, PLA Beijing People's Republic of ChinaAir Force Medical Center, PLA Beijing People's Republic of ChinaAir Force Medical Center, PLA Beijing People's Republic of ChinaAir Force Medical Center, PLA Beijing People's Republic of ChinaBeijing Institute of Basic Medical Sciences Beijing People's Republic of ChinaDepartment of Experimental Hematology & Biochemistry Beijing Institute of Radiation Medicine Beijing People's Republic of ChinaBeijing Institute of Basic Medical Sciences Beijing People's Republic of ChinaAir Force Medical Center, PLA Beijing People's Republic of ChinaDepartment of Experimental Hematology & Biochemistry Beijing Institute of Radiation Medicine Beijing People's Republic of ChinaAbstract 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.https://doi.org/10.1002/sctm.20-0345bone marrow nichegraft vs host diseaseHLA‐haploidentical HSCTmesenchymal stem cellspediatric and adolescent patientssevere aplastic anemia |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Li Ding Dong‐Mei Han Xiao‐Li Zheng Hong‐Min Yan Mei Xue Jing Liu Ling Zhu Sheng Li Ning Mao Zi‐Kuan Guo Hong‐Mei Ning Heng‐Xiang Wang Heng Zhu |
spellingShingle |
Li Ding Dong‐Mei Han Xiao‐Li Zheng Hong‐Min Yan Mei Xue Jing Liu Ling Zhu Sheng Li Ning Mao Zi‐Kuan Guo Hong‐Mei Ning Heng‐Xiang Wang Heng Zhu 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 bone marrow niche graft vs host disease HLA‐haploidentical HSCT mesenchymal stem cells pediatric and adolescent patients severe aplastic anemia |
author_facet |
Li Ding Dong‐Mei Han Xiao‐Li Zheng Hong‐Min Yan Mei Xue Jing Liu Ling Zhu Sheng Li Ning Mao Zi‐Kuan Guo Hong‐Mei Ning Heng‐Xiang Wang Heng Zhu |
author_sort |
Li Ding |
title |
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 |
title_short |
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 |
title_full |
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 |
title_fullStr |
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 |
title_full_unstemmed |
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 |
title_sort |
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 |
publisher |
Wiley |
series |
Stem Cells Translational Medicine |
issn |
2157-6564 2157-6580 |
publishDate |
2021-02-01 |
description |
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. |
topic |
bone marrow niche graft vs host disease HLA‐haploidentical HSCT mesenchymal stem cells pediatric and adolescent patients severe aplastic anemia |
url |
https://doi.org/10.1002/sctm.20-0345 |
work_keys_str_mv |
AT liding astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT dongmeihan astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT xiaolizheng astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT hongminyan astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT meixue astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT jingliu astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT lingzhu astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT shengli astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT ningmao astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT zikuanguo astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT hongmeining astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT hengxiangwang astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT hengzhu astudyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT liding studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT dongmeihan studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT xiaolizheng studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT hongminyan studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT meixue studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT jingliu studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT lingzhu studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT shengli studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT ningmao studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT zikuanguo studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT hongmeining studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT hengxiangwang studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients AT hengzhu studyofhumanleukocyteantigenhaploidenticalhematopoieticstemcellstransplantationcombinedwithallogenicmesenchymalstemcellinfusionfortreatmentofsevereaplasticanemiainpediatricandadolescentpatients |
_version_ |
1724275389164945408 |