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<p>Thrombopoietin receptor agonists (TPO-RAs) are used as a second-line treatment for children with chronic immune thrombocytopenic purpura (ITP) who are refractory to first-line therapies or dependent on corticosteroids, due to their proven efficacy and safety profiles. However, there is a scarcity of comparative real-world data (RWD) for specific TPO-RA agents within pediatric chronic ITP patients. This retrospective study aims to review the efficacy and safety of TPO-RAs (eltrombopag, romiplostim, and avatrombopag) in a real-world clinical setting. Data on pediatric patients with chronic ITP who received TPO mimetics between 2012 and 2023 were collected and analyzed.Thirty-two pediatric patients with chronic ITP, aged 2.75–16 years, underwent 49 TPO-mimetic treatments. Among these, 27 patients were treated with eltrombopag, 16 with romiplostim, and 6 with avatrombopag. The overall response rate was 82%, with eltrombopag achieving an 85% response rate, avatrombopag 83%, and romiplostim 75%. All responders maintained platelet counts &gt; 50 × 10^9/L. Treatment-related adverse events were non-significant with high treatment satisfaction.The results revealed that eltrombopag, romiplostim, and avatrombopag demonstrated comparable efficacy in pediatric chronic ITP, with no serious adverse events.</p>

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Evaluation of Three Thrombopoietin Receptor Agonists in Pediatric Chronic ITP: A Real-World Single-Center Study

  • 期刊论文

  • 发表日期：

    2026年4月1日

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  • Liana Binti Yusof,
  • Jason Patel,
  • Jayashree Motwani,
  • Esraa Mohamed Ahmed Hasan

摘要

Thrombopoietin receptor agonists (TPO-RAs) are used as a second-line treatment for children with chronic immune thrombocytopenic purpura (ITP) who are refractory to first-line therapies or dependent on corticosteroids, due to their proven efficacy and safety profiles. However, there is a scarcity of comparative real-world data (RWD) for specific TPO-RA agents within pediatric chronic ITP patients. This retrospective study aims to review the efficacy and safety of TPO-RAs (eltrombopag, romiplostim, and avatrombopag) in a real-world clinical setting. Data on pediatric patients with chronic ITP who received TPO mimetics between 2012 and 2023 were collected and analyzed.Thirty-two pediatric patients with chronic ITP, aged 2.75–16 years, underwent 49 TPO-mimetic treatments. Among these, 27 patients were treated with eltrombopag, 16 with romiplostim, and 6 with avatrombopag. The overall response rate was 82%, with eltrombopag achieving an 85% response rate, avatrombopag 83%, and romiplostim 75%. All responders maintained platelet counts > 50 × 10^9/L. Treatment-related adverse events were non-significant with high treatment satisfaction.The results revealed that eltrombopag, romiplostim, and avatrombopag demonstrated comparable efficacy in pediatric chronic ITP, with no serious adverse events.