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January 6, 2026American Journal of Hematology

Hetrombopag Added to Cyclosporine as the First‐Line Treatment for Patients With Non‐Severe Aplastic Anemia: A Phase 2 Multicenter Trial

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Authors

LZLele ZhangRLRuonan LiQLQian Liang

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Overview

Phase 2 trial shows hetrombopag improves quality of life with a 81.5% response rate in non-severe aplastic anemia.

Key Points

  • To evaluate the efficacy and safety of hetrombopag added to cyclosporine for treating non-severe aplastic anemia.
  • Phase 2, single-arm trial
  • Involved 54 adults with newly diagnosed non-severe aplastic anemia (NSAA)
  • Patients received hetrombopag in combination with cyclosporine
  • Overall response rate was 81.5% (44/54) after 24 weeks
  • In transfusion-dependent NSAA subgroup, the response rate was 88.0% (22/25)
  • A significant increase in complete responses observed from 24.0% to 44.0% with extended treatment duration from 16 to 24 weeks

Cite This Study

Zhang et al. (2026) studied this question.

synapsesocial.com/papers/695d854b3483e917927a46fehttps://doi.org/10.1002/ajh.70183
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