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ASH 2024 | Iron overload parameters in transfusion-dependent β-thalassemia treated with luspatercept

Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, comments on data from the Phase IIIb long-term rollover study (NCT04064060) following the BELIEVE trial (NCT02604433) of luspatercept in patients with transfusion-dependent beta-thalassemia (TDT). Luspatercept has resulted in a reduction in ferritin levels, whereas the impact on liver iron concentration was not significant. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.

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Transcript (AI-generated)

That is related to luspatercept. Luspatercept is another, let’s say, molecule which may play a role in reducing the ineffective erythropoiesis in thalassemia and mainly the abstract is related to the transfusion-dependent thalassemia patient. 

Now we have a long-term follow-up already, almost five years, and these abstracts look at the iron distribution in these patients and what I can say is that definitely there is a reduction in ferritin levels in patients, whereas the liver iron concentration, which is the other, let’s say, marker for iron overload, it declined much less without actually significant impact...

That is related to luspatercept. Luspatercept is another, let’s say, molecule which may play a role in reducing the ineffective erythropoiesis in thalassemia and mainly the abstract is related to the transfusion-dependent thalassemia patient. 

Now we have a long-term follow-up already, almost five years, and these abstracts look at the iron distribution in these patients and what I can say is that definitely there is a reduction in ferritin levels in patients, whereas the liver iron concentration, which is the other, let’s say, marker for iron overload, it declined much less without actually significant impact. This data require further evaluation, further analysis, and probably they support the idea of immobilization of the iron from the liver or from other sites. So we are not yet at the stage to confirm that treating patients with luspatercept may also impact on the iron overload but this data will be followed over time.

 

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Disclosures

Membership on an entity’s Board of Directors or advisory committees: Novo Nordisk, Vertex Pharmaceuticals, Pfizer,  Silence, Sanofi-Genzyme, Pharmacosmos,  Bristol Myers Squibb (Celgene), Vifor,  Agios Pharmaceuticals, Inc.