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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Main Authors: 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
Format: Article
Language:English
Published: Nature Portfolio 2024-03-01
Series:Scientific Reports
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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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