Aplastic anemia: dramatic increase in hematologic response with eltrombopag

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  • Addition of eltrombopag (Promacta) to horse antithymocyte globulin (Atgam, ATG) and cyclosporine in patients with previously untreated severe aplastic anemia resulted in dramatic increases in hematologic response compared with historical outcomes.

Why this matters

  • There have been numerous unsuccessful efforts to improve upon outcomes in this setting.

Study design

  • Phase 1/2 study to investigate the efficacy and safety of adding eltrombopag to ATG and cyclosporine as first treatment in 92 patients with previously untreated severe aplastic anemia.
  • Patients were enrolled in 3 consecutive cohorts.
  • Funding: National Heart, Lung, and Blood Institute.

Key results

  • Best results were observed in the cohort in which eltrombopag was...