Trial reportBritish journal of haematology2025
Romiplostim with ciclosporin A in patients with aplastic anaemia naïve to immunosuppressive therapy: A phase 2/3 study.
Trial report in British journal of haematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04095936 (A Phase 2/3 Study of AMG531 Combined With Ciclosporin A in Patients With Aplastic Anemia Previously Untreated With Immunosuppressive Therapy), which is not on this map. Cited by 8 papers, 2 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 2/3 Study of AMG531 Combined With Ciclosporin A in Patients With Aplastic Anemia Previously Untreated With Immunosuppressive Therapy
Who cites it
8 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Efficacy and Safety of Romiplostim in the Treatment of Aplastic Anemia: A Systematic Review and Single-Arm Meta-Analysis.Current medical science · 2026Pooled it
- Romiplostim in the management of severe aplastic anemia: a comprehensive clinical review.Annals of hematology · 2026Pooled it
- Romiplostim with ciclosporin A in patients with aplastic anaemia naïve to immunosuppressive therapy: A phase 2/3 study.British journal of haematology · 2025Trial
- Evidence- and opinion-based consensus from Delphi round 1 of the Asian aplastic anemia recommendation development.International journal of hematology · 2026Article
- 2026 Korean expert recommendations for the use of thrombopoietin receptor agonists in severe aplastic anemia.Blood research · 2026Review
- Efficacy and safety of romiplostim with horse anti-thymocyte globulin and cyclosporine in acquired aplastic anemia.International journal of hematology · 2026Article
- Rapid early hematological response in a newly diagnosed patient with very severe aplastic anemia: a case report of high-dose romiplostim, hetrombopag and IST combination therapy.Frontiers in medicine · 2026Article
- Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
16 authors.
Funding
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-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.
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Registered trials
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