Predictive factors of romiplostim response in patients with refractory aplastic anemia: data from two clinical trials.

ABSTRACT: The thrombopoietin receptor agonist (TPO-RA) romiplostim has been shown to be efficacious in the treatment of aplastic anemia (AA) refractory to immunosuppressive therapy. However, there are few reports on predictors of TPO-RA efficacy. We analyzed patient clinical and baseline laboratory data from two clinical trials that enrolled patients with refractory AA, a phase 2 dose-finding study (NCT02094417) and a phase 2/3 study (NCT02773290), to determine predictors of romiplostim efficacy. Data from 66 patients (35 from the phase 2 dose-finding study and 31 from the phase 2/3 study), median age 47.0 (range 20-78) years, were analyzed. Of these, 51.5% (34/66) had non-severe AA, 86.4% (57/66) had received prior therapy with anti-thymocyte globulin plus cyclosporine, and 80.3% (53/66) were trans

SUBMITTER: Jang JH 

PROVIDER: S-EPMC12431918 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature