Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantation
Abstract This study aimed to assess haematopoietic stem cell transplantation (HSCT) safety and efficacy while exploring strategies for optimising outcomes in patients with hepatitis-associated aplastic anaemia (HAAA). We retrospectively reviewed 35 HAAA patients who underwent HSCT at a large Chinese...
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Nature Portfolio
2024-03-01
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Online Access: | https://doi.org/10.1038/s41598-024-55843-7 |
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author | Jia Li Yilin Liu Jieru Wang Yan Wang Aiming Pang Donglin Yang Xin Chen Rongli Zhang Jialin Wei Qiaoling Ma Weihua Zhai Yi He Erlie Jiang Mingzhe Han Sizhou Feng |
author_facet | Jia Li Yilin Liu Jieru Wang Yan Wang Aiming Pang Donglin Yang Xin Chen Rongli Zhang Jialin Wei Qiaoling Ma Weihua Zhai Yi He Erlie Jiang Mingzhe Han Sizhou Feng |
author_sort | Jia Li |
collection | DOAJ |
description | Abstract This study aimed to assess haematopoietic stem cell transplantation (HSCT) safety and efficacy while exploring strategies for optimising outcomes in patients with hepatitis-associated aplastic anaemia (HAAA). We retrospectively reviewed 35 HAAA patients who underwent HSCT at a large Chinese blood disease hospital between 2008 and 2022. HAAA patients receiving HSCT typically presented with severe (28.6%) and very severe (65.7%) AA. Male patients predominated (68.6%), with a median onset age of 23 years (range, 9–44). Haploidentical donor-HSCT and matched sibling donor-HSCT were in comparable proportions. The 5-year overall survival (OS) rate was 74.0%, with cumulative incidences of grade II–IV acute and chronic graft-versus-host disease (GVHD) at 37.1% and 22.4%, respectively. A diagnosis-to-HSCT interval ≥ 75 days, acute GVHD, and post-HSCT liver events (e.g., hepatic GVHD and a three-fold increase in aminotransferase or bilirubin) significantly worsened 5-year OS. In the multivariate models, recipients with sex-matched grafts had better OS, and those with younger male donors had a lower incidence of II–IV aGVHD. Higher HLA matching degree (HLA > = 7/10) was an independent prognostic factor associated with better OS and GFFS. A diagnosis-to-HSCT interval ≥ 75 days was predictive of post-transplant liver events in HAAA patients. In conclusion, HSCT was a safe and effective treatment for HAAA. Early transplantation, careful donor selection and improving post-transplant liver events were crucial to optimise outcomes. |
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spelling | doaj.art-246d6a0ace0540208cdd4815b9db3e482024-03-05T18:39:46ZengNature PortfolioScientific Reports2045-23222024-03-0114111310.1038/s41598-024-55843-7Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantationJia Li0Yilin Liu1Jieru Wang2Yan Wang3Aiming Pang4Donglin Yang5Xin Chen6Rongli Zhang7Jialin Wei8Qiaoling Ma9Weihua Zhai10Yi He11Erlie Jiang12Mingzhe Han13Sizhou Feng14Haematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeHaematopoietic Stem Cell Transplantation Center, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Haematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical CollegeAbstract This study aimed to assess haematopoietic stem cell transplantation (HSCT) safety and efficacy while exploring strategies for optimising outcomes in patients with hepatitis-associated aplastic anaemia (HAAA). We retrospectively reviewed 35 HAAA patients who underwent HSCT at a large Chinese blood disease hospital between 2008 and 2022. HAAA patients receiving HSCT typically presented with severe (28.6%) and very severe (65.7%) AA. Male patients predominated (68.6%), with a median onset age of 23 years (range, 9–44). Haploidentical donor-HSCT and matched sibling donor-HSCT were in comparable proportions. The 5-year overall survival (OS) rate was 74.0%, with cumulative incidences of grade II–IV acute and chronic graft-versus-host disease (GVHD) at 37.1% and 22.4%, respectively. A diagnosis-to-HSCT interval ≥ 75 days, acute GVHD, and post-HSCT liver events (e.g., hepatic GVHD and a three-fold increase in aminotransferase or bilirubin) significantly worsened 5-year OS. In the multivariate models, recipients with sex-matched grafts had better OS, and those with younger male donors had a lower incidence of II–IV aGVHD. Higher HLA matching degree (HLA > = 7/10) was an independent prognostic factor associated with better OS and GFFS. A diagnosis-to-HSCT interval ≥ 75 days was predictive of post-transplant liver events in HAAA patients. In conclusion, HSCT was a safe and effective treatment for HAAA. Early transplantation, careful donor selection and improving post-transplant liver events were crucial to optimise outcomes.https://doi.org/10.1038/s41598-024-55843-7Haematopoietic stem cell transplantHepatitis-associated aplastic anaemiaSurvivalGraft vs. host diseaseRisk factors |
spellingShingle | Jia Li Yilin Liu Jieru Wang Yan Wang Aiming Pang Donglin Yang Xin Chen Rongli Zhang Jialin Wei Qiaoling Ma Weihua Zhai Yi He Erlie Jiang Mingzhe Han Sizhou Feng Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantation Scientific Reports Haematopoietic stem cell transplant Hepatitis-associated aplastic anaemia Survival Graft vs. host disease Risk factors |
title | Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantation |
title_full | Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantation |
title_fullStr | Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantation |
title_full_unstemmed | Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantation |
title_short | Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantation |
title_sort | exploring strategies to optimise outcomes in hepatitis associated aplastic anaemia patients following haematopoietic stem cell transplantation |
topic | Haematopoietic stem cell transplant Hepatitis-associated aplastic anaemia Survival Graft vs. host disease Risk factors |
url | https://doi.org/10.1038/s41598-024-55843-7 |
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