Cystic fibrosis year in review 2024

IF 5.4 2区医学 Q1 RESPIRATORY SYSTEM

Journal of Cystic Fibrosis Pub Date : 2025-03-01 DOI:10.1016/j.jcf.2025.02.012

Amel Alameeri , Burcu Capraz Yavuz , Francesca Lucca , Ivan Bambir , Paulina Famulska , Renata W․F․ Cohen

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引用次数: 0

Abstract

The year 2024 marks a pivotal moment in the field of cystic fibrosis (CF) treatment, characterised by significant advancements in clinical care and an expanding body of literature on CF transmembrane conductance regulator (CFTR) modulators. These CFTR therapies have transformed the landscape of CF management, offered systemic benefits, and established new guidelines for assessing clinical manifestations and therapies. Additionally, progress has been made in newborn screening (NBS), diagnosis, and understanding outcomes for individuals with CF-related metabolic syndrome or inconclusive diagnostic results. However, amidst these clinical milestones, disparities in global access to CFTR modulators (CFTRm) persist, threatening to exacerbate existing inequities in CF care. This review provides a focused overview of the most impactful articles from 2024, highlighting both the clinical advancements and the pressing global accessibility challenges that define this transformative era in CF research and treatment.

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囊性纤维化年回顾2024年。

2024年标志着囊性纤维化（CF）治疗领域的关键时刻，其特点是临床护理取得重大进展，CF跨膜传导调节剂（CFTR）调节剂的文献越来越多。这些CFTR疗法已经改变了CF治疗的格局，提供了系统性的益处，并建立了评估临床表现和治疗的新指南。此外，在新生儿筛查（NBS）、诊断和了解cf相关代谢综合征个体的结局或不确定的诊断结果方面也取得了进展。然而，在这些临床里程碑中，CFTR调节剂（CFTRm）的全球可及性仍然存在差异，有可能加剧CF治疗中现有的不公平现象。本综述对2024年以来最具影响力的文章进行了重点概述，重点介绍了CF研究和治疗变革时代的临床进展和紧迫的全球可及性挑战。

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

Journal of Cystic Fibrosis 医学-呼吸系统

CiteScore

10.10

自引率

13.50%

发文量

1361

审稿时长

50 days

期刊介绍： The Journal of Cystic Fibrosis is the official journal of the European Cystic Fibrosis Society. The journal is devoted to promoting the research and treatment of cystic fibrosis. To this end the journal publishes original scientific articles, editorials, case reports, short communications and other information relevant to cystic fibrosis. The journal also publishes news and articles concerning the activities and policies of the ECFS as well as those of other societies related the ECFS.