Abstract
BACKGROUND: 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. RESULTS: 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(2) = 58.8%) and the pooled HR for failure-free survival (FFS) was 1.962 (95% CI 1.43-2.493, P = 0.000, I(2) = 0%). The pooled relative risk (RR) for overall response rate (ORR) was 1.691 (95% CI 1.433-1.996, P = 0.000, I(2) = 11.6%). CONCLUSION: 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.