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

IF 11.8 Q1 MEDICINE, RESEARCH & EXPERIMENTAL

Med Pub Date : 2025-09-12 DOI:10.1016/j.medj.2025.100844

Mattia Algeri, Franco Locatelli

引用次数: 0

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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ENERGIZE试验：mitapivat是否准备好成为NTDT管理的中心舞台？

ENERGIZE试验表明，一种新型丙酮酸激酶激活剂mitapivat可以快速提高血红蛋白水平，减少患者报告的疲劳，这表明在治疗非输血依赖型地中海贫血方面迈出了一步。对α-和β-地中海贫血亚型均有疗效。需要更长时间的随访来证明米他哌啶持续的效果和对疾病相关并发症的影响。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Med MEDICINE, RESEARCH & EXPERIMENTAL-

CiteScore

17.70

自引率

0.60%

发文量

102

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