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December 8, 2025BloodOpen Access

Safety data from the non-transfusion-dependent dose-confirmation cohort: A phase 2a study of luspatercept in pediatric patients with β-thalassemia

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Authors

YAYeşim AydınokPMPatricia Martín-RegueiraGFGiovanni Battista Ferrero

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Overview

Phase 2a study shows luspatercept's safety in pediatric patients with β-thalassemia, indicating minimal adverse effects.

Key Points

  • Evaluate the safety of luspatercept in pediatric patients with non-transfusion-dependent β-thalassemia.
  • Ongoing phase 2a study with staggered design for ages 6–17
  • Focus on non-transfusion-dependent dose-confirmation cohort
  • Patients received luspatercept 1.0 mg/kg subcutaneously every 3 weeks for 4 cycles
  • All patients experienced at least one treatment-emergent adverse event
  • No serious treatment-emergent adverse events or dose-limiting toxicities were reported
  • Safety profile consistent with prior data from adult patients with β-thalassemia

Cite This Study

Aydınok et al. (2025) studied this question.

synapsesocial.com/papers/69362f364fa91c937236d2fbhttps://doi.org/10.1182/blood-2025-1135
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Also Consider

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

  1. 1A phase 2 study of luspatercept in adults and adolescents with α-thalassemia: Findings from the dose-confirmation cohort in adolescents2025
  2. 2A clinical study on the Efficacy and safety of luspatercept in Chinese patients with non-transfusion-dependent β-thalassemia2025
  3. 3Outcomes with luspatercept in patients with β-thalassemia: a systematic review and meta-analysis2026
  4. 4Luspatercept in β-thalassemia: Meta-analysis of efficacy and safety across transfusion-dependent and non-transfusion-dependent populations2025
  5. 5Outcomes with luspatercept in patients with β-thalassemia: A systematic review and meta-analysis2025