What type of study is this?

This is a Pre-Registered Trial study.

December 8, 2025BloodOpen Access

Long-term transfusion-free duration and impact on transfusion-related burdens: Results from the ongoing ENERGIZE-T open-label extension study of mitapivat in transfusion-dependent alpha- or beta-thalassemia

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Authors

KMKhaled M. MusallamKhalifa University of Science and Technology

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Implication

Observational analysis shows mitapivat improves clinical outcomes in transfusion-dependent alpha- or beta-thalassemia, suggesting enhanced patient quality of life.

Key Points

Long-term transfusion-free duration reached a maximum of 84.3 weeks with mitapivat treatment.
Patients experienced a 56% reduction in annualized transfusion visits, equating to over 2800 mg less iron intake per year.
Assessment of clinical outcomes was evaluated based on cumulative data from the double-blind period and open-label extension.
This analysis highlights the potential improvements in quality of life for patients with transfusion-dependent thalassemia.

Cite This Study

Khaled M. Musallam (2025) studied this question.

synapsesocial.com/papers/69362f574fa91c937236dab9 https://doi.org/10.1182/blood-2025-4697

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Implication

Observational analysis shows mitapivat improves clinical outcomes in transfusion-dependent alpha- or beta-thalassemia, suggesting enhanced patient quality of life.

Long-term transfusion-free duration and impact on transfusion-related burdens: Results from the ongoing ENERGIZE-T open-label extension study of mitapivat in transfusion-dependent alpha- or beta-thalassemia

Discussion

Member takes

Implication

Key Points

Cite This Study

Long-term transfusion-free duration and impact on transfusion-related burdens: Results from the ongoing ENERGIZE-T open-label extension study of mitapivat in transfusion-dependent alpha- or beta-thalassemia

Discussion

Member takes

Implication

Key Points

Cite This Study

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