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September 5, 2025Blood AdvancesOpen Access

Efficacy and safety of luspatercept in non-transfusion-dependent β-thalassemia: long-term results from the BEYOND study

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Authors

ATAlì TaherVVVip ViprakasitAKAntonis Kattamis

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Overview

Observational analysis shows luspatercept improves hemoglobin in patients with non-transfusion-dependent β-thalassemia, highlighting data on safety outcomes.

Key Points

  • Luspatercept led to a sustained increase in hemoglobin levels in patients with non-transfusion-dependent β-thalassemia for up to ~4.6 years of treatment.
  • Overall, 94.8% of patients receiving luspatercept achieved a hemoglobin increase of ≥1.0 g/dL during any 12-week interval, compared to 22.4% for placebo.
  • The analysis from the BEYOND trial followed participants for 26 months, assessing both efficacy and adverse events associated with luspatercept.
  • Luspatercept presents a valuable long-term treatment option, addressing chronic anemia effectively while maintaining a consistent safety profile.

Cite This Study

Taher et al. (2025) studied this question.

synapsesocial.com/papers/68c238d2b210217d6477900ehttps://doi.org/10.1182/bloodadvances.2025016554
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Also Consider

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

  1. 1A clinical study on the Efficacy and safety of luspatercept in Chinese patients with non-transfusion-dependent β-thalassemia2025
  2. 2Luspatercept in β-thalassemia: Meta-analysis of efficacy and safety across transfusion-dependent and non-transfusion-dependent populations2025
  3. 3Safety data from the non-transfusion-dependent dose-confirmation cohort: A phase 2a study of luspatercept in pediatric patients with β-thalassemia2025 · 1 citations
  4. 4Impact of long-term luspatercept treatment on iron parameters in patients with transfusion-dependent and non-transfusion-dependent β-thalassemia: Results from the phase 3b long-term follow-up study2025
  5. 5Outcomes with luspatercept in patients with β-thalassemia: a systematic review and meta-analysis2026