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December 8, 2025BloodOpen Access

Real-world comparative effects of curative and disease-modifying therapies on ineffective erythropoiesis in beta-thalassemia

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Authors

ACAaron ChengJKJanet Kwiatkowski

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Overview

Real-world analysis shows curative therapies may improve ineffective erythropoiesis indicators in beta-thalassemia patients.

Key Points

  • This research aims to compare effects of various therapies on ineffective erythropoiesis and related parameters in beta-thalassemia patients.
  • Enrolled patients with beta-thalassemia from specialized thalassemia centers.
  • Categorized subjects into treatment groups: allo-HSCT, luspatercept, auto-HSCT, regular transfusions.
  • Measured biomarkers including HAMP, ERFE, EPO, GDF-15 via blood samples.
  • Statistical comparisons performed using Kruskal–Wallis test with Dunn's post hoc correction.
  • Patients receiving allo-HSCT showed restored erythropoiesis with normalized EPO, GDF-15, and HAMP levels.
  • Luspatercept users had elevated markers of ineffective erythropoiesis compared to allo and auto-HSCT groups.
  • Iron homeostasis was fully restored in allo-HSCT patients, indicating effective treatment.

Cite This Study

Cheng et al. (2025) studied this question.

synapsesocial.com/papers/69362f6c4fa91c937236dfc1https://doi.org/10.1182/blood-2025-176
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