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September 15, 2021BloodOpen Access

Long-term outcomes in patients with severe aplastic anemia treated with immunosuppression and eltrombopag: a phase 2 study

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Authors

BPBhavisha A. PatelEGEmma M. GroarkeJLJennifer Lotter

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Overview

Clinical trial reveals distinct relapse patterns and clonal evolution in severe aplastic anemia treated with immunosuppression and eltrombopag, highlighting earlier event timing without increased...

Key Points

  • To assess long-term complications, cumulative relapse rates, and clonal evolution in patients with severe aplastic anemia treated with eltrombopag combined with standard immunosuppressive therapy.
  • Secondary endpoint analysis of a phase 2 interventional clinical trial (NCT01623167) enrolling 178 patients with severe aplastic anemia between 2012 and 2020.
  • Patients received standard immunosuppressive therapy (IST) alongside eltrombopag (EPAG) and cyclosporine (CSA) maintenance, followed for a median of 4 years.
  • Relapse rates and clonal evolution incidence were evaluated over time and compared with a historical cohort treated with IST alone.
  • At a median 4-year follow-up, the cumulative relapse rate was 39% in responding patients receiving CSA maintenance, occurring primarily after CSA dose reduction/EPAG discontinuation at 6 months and CSA cessation at 2 years.
  • Clonal evolution occurred in 15% of all treated patients at 4 years, with high-risk evolution (myeloid malignancy or chromosome 7 abnormality) detected in 5.7%, which was associated with reduced overall survival.
  • Neither overall relapse nor high-risk clonal evolution occurred at higher frequencies than in historical IST-treated controls, though the median time to both complications was earlier in the IST and EPAG group.

Cite This Study

Patel et al. (2021) studied this question.

synapsesocial.com/papers/69dc69344264bdb3843593behttps://doi.org/10.1182/blood.2021012130
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Also Consider

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

  1. 1Long-term outcomes of intensive immunosuppressive therapy combined with eltrombopag in patients with severe aplastic anemia2025
  2. 2Efficacy and influencing factors of immunosuppressive therapy combined with or without eltrombopag in children with severe aplastic anemia2025
  3. 3Outcomes of eltrombopag combined with immunosuppressive therapy in adults with severe aplastic anemia: A large, propensity-matched retrospective analysis2025
  4. 4A 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
  5. 5<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