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December 8, 2025BloodOpen Access

Adding eltrombopag to immunosuppressive treatment with cyclosporine as front-line therapy for moderate aplastic anemia improves trilineage hematologic response: One-year analysis of the randomised, placebo-controlled, double-blind Phase III trial eltrombopag for moderate aplastic anemia (EMAA Trial)

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Authors

MHMichael HeuserFSFlore Sicre-de-FontbruneAMAstrid Marx‐Hofmann

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Overview

Randomised trial demonstrates improved hematologic response in moderate aplastic anemia patients receiving eltrombopag with cyclosporine, suggesting a potential new standard of care.

Key Points

  • This research aims to evaluate the efficacy of eltrombopag combined with cyclosporine in treating moderate aplastic anemia.
  • Prospective, randomized, placebo-controlled, double-blind Phase III trial.
  • Included 85 treatment-naïve patients with moderate aplastic anemia requiring treatment.
  • Patients received either placebo or eltrombopag while both groups were treated with cyclosporine.
  • The overall response rate was significantly higher in the eltrombopag arm at 24 weeks (71.4%) than in the placebo arm (42.5%).
  • The complete response rate was 11.4% in the eltrombopag arm compared to 5.0% in the placebo arm.
  • Adding eltrombopag to placebo patients without complete response after 24 weeks increased overall response rate to 73.5% by week 48.

Cite This Study

Heuser et al. (2025) studied this question.

synapsesocial.com/papers/69362f764fa91c937236e36chttps://doi.org/10.1182/blood-2025-980
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Eltrombopag plus cyclosporine A for moderate aplastic anemia (EMAA): a placebo-controlled, double-blind, phase 3 trial2026
  2. 2Outcomes of eltrombopag combined with immunosuppressive therapy in adults with severe aplastic anemia: A large, propensity-matched retrospective analysis2025
  3. 3Efficacy and influencing factors of immunosuppressive therapy combined with or without eltrombopag in children with severe aplastic anemia2025
  4. 4Efficacy and influencing factors of immunosuppressive therapy combined with or without eltrombopag in children with severe aplastic anemia2025 · 2 citations
  5. 5A Multicenter, Retrospective Study Comparing Immunosuppressive Therapy Combined with Eltrombopag to Immunosuppressive Therapy Alone as Front-Line Treatment for Pediatric Severe Aplastic Anemia.2025 · 2 citations