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

Mattia Algeri; Franco Locatelli

doi:10.1016/j.medj.2025.100844

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

Med. 2025 Sep 12;6(9):100844. doi: 10.1016/j.medj.2025.100844.

Authors

Mattia Algeri¹, Franco Locatelli²

Affiliations

¹ Department 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.
² Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, IRCCS, Bambino Gesù Children's Hospital, Rome, Italy; Catholic University of the Sacred Heart, Rome, Italy. Electronic address: franco.locatelli@opbg.net.

PMID: 40945502
DOI: 10.1016/j.medj.2025.100844

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.

MeSH terms

Fatigue / drug therapy
Fatigue / etiology
Hemoglobins / metabolism
Humans
Pyruvate Kinase*
alpha-Thalassemia* / drug therapy
beta-Thalassemia* / drug therapy

Substances

Pyruvate Kinase
Hemoglobins