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December 8, 2025BloodOpen Access

A phase 2 study of luspatercept in adults and adolescents with α-thalassemia: Findings from the dose-confirmation cohort in adolescents

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Authors

YLYongrong LaiMLMeng‐Yao LuYZYing Zhao

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Overview

Phase 2 study demonstrates safety and tolerability of luspatercept in adolescents with α-thalassemia, indicating potential for broader use in anemia treatment.

Key Points

  • This study aims to evaluate the safety and tolerability of luspatercept in adolescents with α-thalassemia.
  • Phase 2, open-label, multicenter study
  • Included 6 adolescents aged 12 to <18 years with α-thalassemia
  • Patients received 1 dose of luspatercept at 1.0 mg/kg subcutaneously
  • Cohorts defined by transfusion-dependent (TD) and non-transfusion-dependent (NTD) status
  • Safety and tolerability assessed during a 21-day treatment cycle.
  • No dose-limiting toxicities or new safety signals reported
  • Two treatment-emergent adverse events occurred; one was grade 1 dizziness and another was a grade 2 upper respiratory infection
  • Luspatercept demonstrated acceptable safety and tolerability in adolescents with α-thalassemia

Cite This Study

Lai et al. (2025) studied this question.

synapsesocial.com/papers/69362f574fa91c937236daa5https://doi.org/10.1182/blood-2025-4689
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Also Consider

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

  1. 1Safety data from the non-transfusion-dependent dose-confirmation cohort: A phase 2a study of luspatercept in pediatric patients with β-thalassemia2025 · 1 citations
  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