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<p>Thrombopoietin receptor agonists (TPO-RAs) have become pivotal in managing immune thrombocytopenia (ITP), yet data from Indian cohorts remain limited. This study evaluated real-world efficacy, safety, and predictors of TPO-RA response in adults with ITP. We conducted a retrospective analysis of 106 ITP patients treated with eltrombopag or romiplostim at a tertiary-care center between 2017 and 2025. Treatment responses were defined as complete response (CR: ≥100 × 10⁹/L without bleeding), response (R: ≥30 × 10⁹/L with ≥ 2-fold increase from baseline), and no response (NR). Failure-free survival (FFS) was calculated from TPO-RA initiation to refractoriness, relapse, or death. Logistic regression identified predictors of response. The median age was 38 years, with a female predominance (69.8%). Most patients had chronic ITP (65.1%) and a median illness duration of 23 months. Eltrombopag was used in 77.4% and romiplostim in 22.6%. The response rate was 52.8% at day 30, 57.5% at day 60, and 61.3% at day 90, with CR in 32.1%. The median time to first response was 12 days (IQR 8–19), and median FFS was 17 months. Adverse events were infrequent: thrombocytosis (9.4%), transaminitis (3.8%), and thrombosis or reticulin fibrosis (0.9% each). On multivariate analysis, mean platelet volume &gt; 12 fL (OR 2.95) and steroid refractoriness (OR 2.7) independently predicted response. TPO-RAs were effective and well-tolerated in Indian adults with ITP, achieving outcomes comparable to international data. Elevated MPV and steroid refractoriness emerged as simple, clinically relevant predictors of favourable response.</p>

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Mean Platelet Volume and Steroid Refractoriness as Independent Predictors of Response to Thrombopoietin Receptor Agonists in Immune Thrombocytopenia

  • 期刊论文

  • 发表日期：

    2026年5月13日

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  • Sarthak Wadhera,
  • Rudra Narayan Swain,
  • Aarushi Sahni,
  • Charanpreet Singh,
  • Aditya Jandial,
  • Arihant Jain,
  • Gaurav Prakash,
  • Alka Khadwal,
  • Reena Das,
  • Pankaj Malhotra

摘要

Thrombopoietin receptor agonists (TPO-RAs) have become pivotal in managing immune thrombocytopenia (ITP), yet data from Indian cohorts remain limited. This study evaluated real-world efficacy, safety, and predictors of TPO-RA response in adults with ITP. We conducted a retrospective analysis of 106 ITP patients treated with eltrombopag or romiplostim at a tertiary-care center between 2017 and 2025. Treatment responses were defined as complete response (CR: ≥100 × 10⁹/L without bleeding), response (R: ≥30 × 10⁹/L with ≥ 2-fold increase from baseline), and no response (NR). Failure-free survival (FFS) was calculated from TPO-RA initiation to refractoriness, relapse, or death. Logistic regression identified predictors of response. The median age was 38 years, with a female predominance (69.8%). Most patients had chronic ITP (65.1%) and a median illness duration of 23 months. Eltrombopag was used in 77.4% and romiplostim in 22.6%. The response rate was 52.8% at day 30, 57.5% at day 60, and 61.3% at day 90, with CR in 32.1%. The median time to first response was 12 days (IQR 8–19), and median FFS was 17 months. Adverse events were infrequent: thrombocytosis (9.4%), transaminitis (3.8%), and thrombosis or reticulin fibrosis (0.9% each). On multivariate analysis, mean platelet volume > 12 fL (OR 2.95) and steroid refractoriness (OR 2.7) independently predicted response. TPO-RAs were effective and well-tolerated in Indian adults with ITP, achieving outcomes comparable to international data. Elevated MPV and steroid refractoriness emerged as simple, clinically relevant predictors of favourable response.