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

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-dependent non-severe AA (NSAA). A haematological response at Week 27 was observed in 10/24 patients (overall response, 41.7%; 95% confidence interval, 22.1%-63.4%). At Week 27, the subgroup overall response rates were 0.0% in VSAA, 46.2% in SAA and 57.1% in NSAA. Nearly 92% of patients experienced at least one treatment-emergent adverse event (TEAE), but no drug-related Grade ≥3 TEAEs were reported. One patient developed myelodysplastic syndromes. Treatment with romiplostim plus CsA was effective and well tolerated in patients with AA who had not previously received IST.