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The ENERGIZE trial: Is mitapivat ready to take center stage in NTDT management?
Mattia Algeri1, Franco Locatelli2
1Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy; Department of Health Sciences, Magna Graecia University, Catanzaro, Italy.
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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