Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent β-Thalassemia - Summary - MDSpire
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Restoration of Effective Erythropoiesis and Iron Homeostasis Following Exagamglogene Autotemcel Treatment in Patients with Transfusion-Dependent β-Thalassemia

  • By

  • Sujit Sheth

  • Selim Corbacioglu

  • Josu de la Fuente

  • Mattia Algeri

  • Joachim Rupprecht

  • Kevin H. M. Kuo

  • Ami J. Shah

  • Peter Lang

  • Hayley Merkeley

  • Ben Carpenter

  • Markus Y. Mapara

  • Robert I. Liem

  • Stephan Grupp

  • Yogi Chopra

  • Amanda M. Li

  • Janet L. Kwiatkowski

  • Melanie Kirby-Allen

  • Maria Domenica Cappellini

  • Antonis Kattamis

  • Sakellarios Zairis

  • Tina Liu

  • William Hobbs

  • Haydar Frangoul

  • Franco Locatelli

  • Roland Meisel

  • June 7, 2026

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Objective:

To analyze key biomarkers of erythropoiesis and iron homeostasis before and after discontinuation of iron removal therapy in patients treated with exagamglogene autotemcel (exa-cel) for transfusion-dependent β-thalassemia (TDT).

Approach:
  • Study Design: Phase 3, open-label, single dose study (CLIMB THAL-111) followed by a long-term follow-up study (CLIMB-131) involving patients aged 12–35 years with TDT.
  • Participants: Patients with a confirmed diagnosis of TDT and a history of significant RBC transfusions.
  • Outcomes: Primary endpoint was transfusion independence; secondary endpoints included changes in hemoglobin levels, markers of iron overload, and erythropoiesis.
Key Findings:
  • Infusion of exa-cel led to transfusion independence in 91.4% of participants in the study.
  • Mean total hemoglobin reached 13.1 g/dL and mean HbF was 11.9 g/dL, as reported in the study.
  • Analysis of biomarkers indicated restoration of effective erythropoiesis and improved iron homeostasis post-treatment, according to study findings.
Interpretation:

Limitations:
  • The study was limited to a specific age group (12–35 years).
  • Long-term effects beyond 6 years of follow-up were not fully assessed.
Conclusion:

Exagamglogene autotemcel treatment shows potential in addressing ineffective erythropoiesis and iron overload in TDT patients, based on study results.

Sources:

Original Source(s)

Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion-Dependent β-Thalassemia

  • by Sujit Sheth, Selim Corbacioglu, Josu de la Fuente, Mattia Algeri, Joachim Rupprecht, Kevin H. M. Kuo, Ami J. Shah, Peter Lang, Hayley Merkeley, Ben Carpenter, Markus Y. Mapara, Robert I. Liem, Stephan Grupp, Yogi Chopra, Amanda M. Li, Janet L. Kwiatkowski, Melanie Kirby-Allen, Maria Domenica Cappellini, Antonis Kattamis, Sakellarios Zairis, Tina Liu, William Hobbs, Haydar Frangoul, Franco Locatelli, Roland Meisel

  • June 7, 2026

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