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August 20, 2026Blood

Eltrombopag plus cyclosporine A for moderate aplastic anemia (EMAA): a placebo-controlled, double-blind, phase 3 trial

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Authors

BHBritta HöchsmannPSPhilippe SchafhausenFBFabian Beier

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Overview

Randomized double-blind trial demonstrates that adding eltrombopag to cyclosporine A significantly increases response rates in moderate aplastic anemia, indicating an effective first-line regimen.

Key Points

  • To determine whether adding eltrombopag to standard cyclosporine A improves hematologic response rates in patients with moderate aplastic anemia and clinically significant cytopenia.
  • Investigator-initiated, prospective, multicenter, randomized, double-blind, placebo-controlled phase 3 trial (NCT02773225; EudraCT2014-000174-19).
  • Assigned patients with moderate aplastic anemia to receive eltrombopag plus cyclosporine A (n=41) or placebo plus cyclosporine A (n=44) as first-line therapy.
  • Patients in the placebo arm without a complete response at week 24 unblinded and switched to receive eltrombopag plus continued cyclosporine A through week 48.
  • At week 24, the primary endpoint of overall response rate in evaluable patients (n=75) was 71.4% (95% CI, 54.8%-83.8%) in the eltrombopag plus cyclosporine A group versus 42.5% (95% CI, 28.5%-57.8%) in the placebo group (p=0.011).
  • Placebo-arm patients without complete response who added eltrombopag after week 24 achieved an overall response rate of 73.5% by week 48 (p=0.009 vs. week 24).
  • Relapse rates and failure-free survival did not differ significantly between treatment arms, and eltrombopag was well tolerated with no new safety signals.

Cite This Study

Höchsmann et al. (2026) studied this question.

synapsesocial.com/papers/6a86b57f8a91293e6a1ccf4ehttps://doi.org/10.1182/blood.2025032682
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Also Consider

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

  1. 1Adding 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)2025
  2. 2<scp>Eltrombopag</scp> Added to Standard Immunosuppressive Treatment as Front‐Line Therapy for Severe Aplastic Anemia: Long‐Term Outcomes of the Phase‐3 Randomized Superiority <scp>EBMT</scp> ‐ <scp>SAAWP RACE</scp> Study2026
  3. 3Avatrombopag as alternative therapy for severe aplastic anemia patients who are intolerant or unresponsive to eltrombopag2024
  4. 4Long-term outcomes in patients with severe aplastic anemia treated with immunosuppression and eltrombopag: a phase 2 study2021 · 111 citations
  5. 5Hetrombopag Added to Cyclosporine as the First‐Line Treatment for Patients With Non‐Severe Aplastic Anemia: A Phase 2 Multicenter Trial2026