Cyclosporine plus eltrombopag in the treatment of aplastic anemia with or without antithymocyte immunoglobulin: A multicenter real‐world retrospective study

Abstract Aims To compare cyclosporine (CSA) combining eltrombopag (EPAG) with or without antithymocyte globulin (ATG) in aplastic anemia (AA) patients in the real world. Methods AA patients who received ATG combining CSA and EPAG (Group A) and CSA + EPAG (Group B) as front‐line treatment in 13 medical centers in China were enrolled. The efficacy and safety were compared. Results A total of 89 patients were enrolled with 51 patients in Group A and 38 patients in Group B. The 6‐month overall response (OR)/complete response (CR) was 73.3%/24.4% and 60.6%/27.3% in Groups A and B ( p > .1). For severe AA patients, the 6‐month OR was 74.1% versus 50% and 6‐month CR was 25.9% versus 20% in Groups A and B ( p > 0.1). Multivariate analysis showed gender affects the 6‐month OR with females better OR ( p = .017, OR 6.045, 95% CI: 1.377–26.546) and time from disease onset to treatment affected the 12‐month CR ( p = .026, OR 0.263, 95% CI: 0.081–0.852). No difference was found in side effects except ATG infusion reaction and serum sickness. Mortality was 7.8% in Group A and no patient died in Group B. Conclusions CSA + EPAG had a similar response and less side effects compared with standard immunosuppressive therapy + EPAG in newly diagnosed AA.