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December 8, 2025BloodOpen Access

A clinical study on the Efficacy and safety of luspatercept in Chinese patients with non-transfusion-dependent β-thalassemia

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Authors

HLHe LiTNTing NiuXYXinwei Yu

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Overview

Multicenter trial shows improved hemoglobin levels and quality of life in Chinese patients with β-thalassemia, indicating luspatercept's efficacy and safety.

Key Points

  • Investigate the efficacy and safety of luspatercept in Chinese patients with NTD β-thalassemia.
  • Prospective, single-arm, multicenter, open-label design
  • Enrolled 70 patients aged ≥ 18 with NTD β-thalassemia
  • Administered subcutaneous luspatercept 1.0 mg/kg Q3W for 24 weeks
  • Monitored adverse events using NCI CTCAE criteria
  • Achieved mean hemoglobin increase of 2.1 g/dL (+29%) in 21 patients at 24 weeks
  • 67% of patients had hemoglobin increase ≥ 1.0 g/dL
  • No grade 3 or higher adverse events observed
  • Quality of life improved as indicated by decreased fatigue scores

Cite This Study

Li et al. (2025) studied this question.

synapsesocial.com/papers/69362f7d4fa91c937236e47dhttps://doi.org/10.1182/blood-2025-1142
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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. 2Outcomes with luspatercept in patients with β-thalassemia: a systematic review and meta-analysis2026
  3. 3Efficacy and safety of luspatercept in non-transfusion-dependent β-thalassemia: long-term results from the BEYOND study2025 · 10 citations
  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