Department of Therapeutic Center of Anemia, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
BMC Immunol. 2020 Mar 6;21(1):10. doi: 10.1186/s12865-020-0340-x.
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, I = 58.8%) and the pooled HR for failure-free survival (FFS) was 1.962 (95% CI 1.43-2.493, P = 0.000, I = 0%). The pooled relative risk (RR) for overall response rate (ORR) was 1.691 (95% CI 1.433-1.996, P = 0.000, I = 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.
同种异体造血干细胞移植（allo-HSCT）和免疫抑制治疗（IST）是获得性再生障碍性贫血（AA）的两种主要竞争治疗策略。allo-HSCT 是否优于 IST 作为 AA 患者的一线治疗一直存在争议。为了比较 allo-HSCT 与 IST 作为 AA 患者一线治疗的疗效和安全性，我们对评估两种主要竞争治疗策略对 AA 影响的现有研究进行了荟萃分析。
该荟萃分析共纳入了 15 项研究，总计 5336 例患者。总生存（OS）的合并风险比（HR）为 0.4（95%CI 0.074-0.733，P=0.016，I²=58.8%），无失败生存（FFS）的合并 HR 为 1.962（95%CI 1.43-2.493，P=0.000，I²=0%）。总反应率（ORR）的合并相对风险（RR）为 1.691（95%CI 1.433-1.996，P=0.000，I²=11.6%）。
尽管与接受一线 IST 的 AA 患者相比，接受一线 allo-HSCT 的患者生存时间显著延长，但对于新诊断为 AA 的患者，初始治疗的选择仍需要全面评估供体可用性、患者年龄、预期生活质量、IST 后疾病复发或克隆演变的风险，以及辅助使用艾曲泊帕的可能性。
