What type of study is this?

This is a Pre-Registered Trial study.

What question did this study set out to answer?

Assess the long-term duration of transfusion-free periods and their real-world impact on transfusion-related burdens for patients on mitapivat.

December 8, 2025BloodOpen Access

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Authors

Phase 3 trial shows prolonged transfusion-free periods with mitapivat in thalassemia, indicating improved clinical outcomes.

Assess the long-term duration of transfusion-free periods and their real-world impact on transfusion-related burdens for patients on mitapivat.
Adults with transfusion-dependent alpha- or beta-thalassemia received mitapivat or placebo for 48 weeks.
Transfusion independence was defined as being transfusion-free for at least 8 consecutive weeks.
Duration of transfusion-free periods was evaluated during the double-blind phase and the open-label extension.
Mean duration of the longest transfusion-free period was 30.49 weeks during the evaluation period.
Reduction of annualized transfusion visits by 56% and 63.6% fewer packed RBC units received per year.
3 patients maintained transfusion-free status during the open-label extension.

Khaled M. Musallam (2025) studied this question.

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