Hong Yaonan, Liu Qi, Sun Zhuonan, Wang Peicheng, Wang Xu, Su Ziying, Li Yuzhu, Liu Wenbin, Hu Huijin, Shen Yingying, Ye Baodong, Zhou Yuhong, Liu Shan, Wu Dijiong
The First School of Clinical Medicine, Zhejiang Chinese Medical University, Hangzhou, People's Republic of China.
Department of Hematology, The First Affiliated Hospital of Zhejiang Chinese Medical University (Zhejiang Provincial Hospital of Chinese Medicine), Hangzhou, People's Republic of China.
Hematology. 2025 Dec;30(1):2548990. doi: 10.1080/16078454.2025.2548990. Epub 2025 Aug 26.
The present study aimed to compare the efficacy and safety of hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) for hepatitis-associated aplastic anemia (HAAA). Studies comparing HSCT with IST in HAAA were retrieved from inception to July 22, 2024, including 12 studies with a total of 544 cases for meta-analysis. Meta-analysis demonstrated significantly superior outcomes in the HSCT group versus IST, which was manifested as lower overall mortality ( < 0.01), higher overall response rate ( < 0.001), and improved five-year overall survival ( < 0.05), yielding a pooled RR of 1.67 (95% CI: 1.15-2.44), 0.75 (95% CI: 0.66-0.86) and 0.88 (95% CI: 0.78-0.99), respectively. However, no benefit was observed in one-year survival ( = 0.08). Further subgroup analysis indicated that the advantage of mortality ( < 0.05, RR = 1.67, 95% CI: 1.10-2.55) and five-year overall survival ( = 0.05, RR = 0.84, 95% CI: 0.71-1.00) only achieved in patients under 20 years old. There was no significant difference in the overall response and one-year overall survival for each age group. Additionally, for the IST selection, a combination of cyclosporine (CSA) and antithymocyte globulin/antilymphocyte globulin (ATG/ALG) was preferred over the CSA-only regimen (effectiveness of 78.57% . 50.00%), although the difference was not statistically significant ( = 0.10, RR = 1.56, 95% CI: 0.92-2.66). This study showed that HSCT had a higher effective rate, greater long-term survival and lower mortality compared to IST, especially for patients under 20 years old, who should receive HSCT treatment as possible.
本研究旨在比较造血干细胞移植（HSCT）和免疫抑制治疗（IST）用于肝炎相关性再生障碍性贫血（HAAA）的疗效和安全性。检索了从开始到2024年7月22日比较HSCT与IST治疗HAAA的研究，包括12项研究共544例用于荟萃分析。荟萃分析表明HSCT组相对于IST组有显著更好的结果，表现为总体死亡率更低（<0.01）、总体缓解率更高（<0.001）以及五年总生存率提高（<0.05），合并相对危险度分别为1.67（95%可信区间：1.15 - 2.44）、0.75（95%可信区间：0.66 - 0.86）和0.88（95%可信区间：0.78 - 0.99）。然而，在一年生存率方面未观察到益处（P = 0.08）。进一步的亚组分析表明，死亡率（<0.05，RR = 1.67，95%可信区间：1.10 - 2.55）和五年总生存率（P = 0.05，RR = 0.84，95%可信区间：0.71 - 1.00）的优势仅在20岁以下患者中实现。各年龄组在总体缓解和一年总生存率方面无显著差异。此外，对于IST的选择，环孢素（CSA）与抗胸腺细胞球蛋白/抗淋巴细胞球蛋白（ATG/ALG）联合方案优于仅使用CSA的方案（有效率分别为78.57%对50.00%），尽管差异无统计学意义（P = 0.10，RR = 1.56，95%可信区间：0.92 - 2.66）。本研究表明，与IST相比，HSCT有效率更高、长期生存率更高且死亡率更低，尤其是对于20岁以下患者，应尽可能接受HSCT治疗。
