Allogeneic hematopoietic stem cell transplantation (allo-HSCT) and immunosuppressive therapy (IST) are two major competing treatment strategies for acquired aplastic anemia (AA). Whether allo-HSCT is superior to IST as a front-line treatment for patients with AA has been a subject of debate. To compare the efficacy and safety of allo-HSCT with that of IST as a front-line treatment for patients with AA, we performed a meta-analysis of available studies that examined the impact of the two major competing treatment strategies for AA. Fifteen studies including a total of 5336 patients were included in the meta-analysis. The pooled hazard ratio (HR) for overall survival (OS) was 0.4 (95% CI 0.074–0.733, P = 0.016, I2 = 58.8%) and the pooled HR for failure-free survival (FFS) was 1.962 (95% CI 1.43–2.493, P = 0.000, I2 = 0%). The pooled relative risk (RR) for overall response rate (ORR) was 1.691 (95% CI 1.433–1.996, P = 0.000, I2 = 11.6%). Although survival was significantly longer among AA patients undergoing first-line allo-HSCT compared to those undergoing first-line IST, the selection of initial treatment for patients with newly diagnosed AA still requires comprehensive evaluation of donor availability, patient age, expected quality of life, risk of disease relapse or clonal evolution after IST, and potential use of adjunctive eltrombopag.

中文翻译：

---

异基因造血干细胞移植与免疫抑制疗法治疗后天再生障碍性贫血的比较：系统评价和荟萃分析

异基因造血干细胞移植（allo-HSCT）和免疫抑制疗法（IST）是获得性再生障碍性贫血（AA）的两种主要竞争治疗策略。作为AA患者的一线治疗，同种异体造血干细胞移植是否优于IST一直是争论的话题。为了比较allo-HSCT与IST作为AA患者一线治疗的疗效和安全性，我们对现有研究进行了荟萃分析，检查了两种主要竞争性治疗策略对AA的影响。荟萃分析包括15项研究，共5336名患者。总体生存（OS）的合并风险比（HR）为0.4（95％CI 0.074-0.733，P = 0.016，I2 = 58.8％），无故障生存（FFS）的合并HR为1.962（95％CI 1.43–2.493，P = 0.000，I2 = 0％）。总缓解率（ORR）的合并相对风险（RR）为1.691（95％CI 1.433–1.996，P = 0.000，I2 = 11.6％）。尽管接受一线all-HSCT的AA患者比接受一线IST的AA患者生存期明显更长，但为新诊断为AA的患者选择初始治疗仍需要全面评估供体的可用性，患者年龄，预期生活质量，IST后疾病复发或克隆进化的风险以及可能使用辅助性Eltrombopag。