: The ENERGIZE trial1 represents a step forward in managing non-transfusion-dependent thalassemia, demonstrating that mitapivat, a novel pyruvate kinase activator, may rapidly increase hemoglobin levels and reduce patient-reported fatigue. Efficacy was shown in both α- and β-thalassemia subtypes. Longer follow-up is required to document mitapivat-sustained effects and impact on disease-related complications.

The ENERGIZE trial: Is mitapivat ready to take center stage in NTDT management?

Algeri, Mattia;
2025-01-01

Abstract

: The ENERGIZE trial1 represents a step forward in managing non-transfusion-dependent thalassemia, demonstrating that mitapivat, a novel pyruvate kinase activator, may rapidly increase hemoglobin levels and reduce patient-reported fatigue. Efficacy was shown in both α- and β-thalassemia subtypes. Longer follow-up is required to document mitapivat-sustained effects and impact on disease-related complications.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/20.500.12317/113289
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