Romiplostim with ciclosporin A in patients with aplastic anaemia naive to immunosuppressive therapy: A phase 2/3 study.

ABSTRACT: Romiplostim has been shown to restore multi-lineage haematopoiesis and is effective in patients with aplastic anaemia (AA) refractory to immunosuppressive therapy (IST). This open-label, phase 2/3 study (NCT04095936) recruited adult AA patients in Japan and Korea who had not received prior IST and evaluated the efficacy and safety of romiplostim plus ciclosporin A (CsA). Romiplostim was initiated at 10 μg/kg once weekly through Week 4 and adjusted between 0 and 20 μg/kg from Week 5 onwards. CsA was administered at 5-6 mg/kg/day in two divided doses through Week 26. A total of 24 patients (median [range] age, 52 [19-80] years) were enrolled, and 22 (91.7%) completed the study. Four patients (16.7%) had very severe AA (VSAA), 13 (54.2%) had severe AA (SAA) and seven (29.2%) had transfusion-d

SUBMITTER: Lee JW 

PROVIDER: S-EPMC12378949 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature