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

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

摘要

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。

查看原文本刊更多论文

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

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.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Med MEDICINE, RESEARCH & EXPERIMENTAL-

CiteScore

17.70

自引率

0.60%

发文量

102

期刊介绍： Med is a flagship medical journal published monthly by Cell Press, the global publisher of trusted and authoritative science journals including Cell, Cancer Cell, and Cell Reports Medicine. Our mission is to advance clinical research and practice by providing a communication forum for the publication of clinical trial results, innovative observations from longitudinal cohorts, and pioneering discoveries about disease mechanisms. The journal also encourages thought-leadership discussions among biomedical researchers, physicians, and other health scientists and stakeholders. Our goal is to improve health worldwide sustainably and ethically. Med publishes rigorously vetted original research and cutting-edge review and perspective articles on critical health issues globally and regionally. Our research section covers clinical case reports, first-in-human studies, large-scale clinical trials, population-based studies, as well as translational research work with the potential to change the course of medical research and improve clinical practice.