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探索优化造血干细胞移植后肝炎相关性再生障碍性贫血患者治疗效果的策略。

Exploring strategies to optimise outcomes in hepatitis-associated aplastic anaemia patients following haematopoietic stem cell transplantation.

作者信息

Li Jia, Liu Yilin, Wang Jieru, Wang Yan, Pang Aiming, Yang Donglin, Chen Xin, Zhang Rongli, Wei Jialin, Ma Qiaoling, Zhai Weihua, He Yi, Jiang Erlie, Han Mingzhe, Feng Sizhou

机构信息

Haematopoietic 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 College, Tianjin, 300020, China.

Tianjin Institutes of Health Science, Tianjin, 301600, China.

出版信息

Sci Rep. 2024 Mar 2;14(1):5178. doi: 10.1038/s41598-024-55843-7.

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.

摘要

本研究旨在评估造血干细胞移植(HSCT)治疗肝炎相关性再生障碍性贫血(HAAA)患者的安全性和疗效,并探索优化治疗结果的策略。我们回顾性分析了2008年至2022年期间在中国一家大型血液病医院接受HSCT的35例HAAA患者。接受HSCT的HAAA患者通常表现为重度(28.6%)和极重度(65.7%)再生障碍性贫血。男性患者占主导(68.6%),中位发病年龄为23岁(范围9 - 44岁)。单倍体相合供者HSCT和同胞全相合供者HSCT的比例相当。5年总生存率(OS)为74.0%,II - IV级急性和慢性移植物抗宿主病(GVHD)的累积发生率分别为37.1%和22.4%。诊断至HSCT间隔≥75天、急性GVHD以及HSCT后肝脏事件(如肝脏GVHD和转氨酶或胆红素升高三倍)显著降低5年OS。在多变量模型中,接受性别匹配移植物的受者OS更好,而供者为年轻男性的受者II - IV级急性GVHD发生率较低。更高的人类白细胞抗原(HLA)匹配度(HLA≥7/10)是与更好的OS和无移植失败生存率(GFFS)相关的独立预后因素。诊断至HSCT间隔≥75天可预测HAAA患者移植后肝脏事件。总之,HSCT是治疗HAAA的一种安全有效的方法。早期移植、谨慎选择供者以及改善移植后肝脏事件对于优化治疗结果至关重要。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/aefa/10908854/882af857ea2e/41598_2024_55843_Fig1_HTML.jpg

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