CFTR调节剂对纵向囊性纤维化生存和死亡率的影响：回顾和二次分析。

IF 3 Q2 RESPIRATORY SYSTEM

Pulmonary Therapy Pub Date : 2025-09-01 Epub Date: 2025-07-11 DOI:10.1007/s41030-025-00303-4

Jaime L Rubin, Craig McKinnon, Gabriel Ghizzi Pedra, Devon A Morgan, Kimberly Zweig, Theodore G Liou

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引用次数: 0

摘要

囊性纤维化（CF）跨膜传导调节剂（CFTRm）已经改变了CF的治疗方式，将治疗从仅仅控制症状转变为解决导致CF的潜在缺陷。CFTRm于2012年首次进入临床实践，随后又有更多的CFTRm组合，包括elexaftor /tezacaftor/ivacaftor （ELX/TEZ/IVA）于2019年获得批准，可治疗大多数CF基因型。方法：我们选取同行评审的文献进行叙述性回顾（1990年1月至2025年1月），描述CF生存率和死亡年龄的纵向趋势，并评估CFTRm的影响，特别是ELX/TEZ/IVA。为了补充现有文献，我们使用最新的可用数据对美国CF基金会患者登记处（U.S. cfpr, 1990-2023）的历史纵向趋势进行了二次分析。结果：已发表研究的定量数据表明，生存和死亡的中位年龄随着时间的推移而增加，但不同地区的幅度不同。大多数队列和基于登记的研究是在CFTRm尚未广泛应用的情况下进行的，这限制了CFTRm对长期生存趋势影响的评估。在美国cfpr的二次分析中，中位生存年龄从1990年的29.0岁增加到2012年CFTRm引入前的38.6岁，到2023年增加到68.0岁，显示了CFTRm引入后的显著改善。线性回归分析显示，中位生存年龄的增加从CFTRm前的每年0.48岁增加到2019年批准ELX/TEZ/IVA后的每年4.79岁。结论：研究结果提供了CFTRm显著提高生存率的初步证据。跨地域的长期随访数据将更深入地了解CFTRm对预测的CF生存和死亡率的全面影响。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Impact of CFTR Modulators on Longitudinal Cystic Fibrosis Survival and Mortality: Review and Secondary Analysis.

查看原文本刊更多论文

Impact of CFTR Modulators on Longitudinal Cystic Fibrosis Survival and Mortality: Review and Secondary Analysis.

Introduction: Cystic fibrosis (CF) transmembrane conductance regulator modulators (CFTRm) have transformed CF care, shifting treatment from only managing symptoms to also addressing the underlying defects that cause CF. CFTRm first entered clinical practice in 2012 and was followed by additional CFTRm combinations-including the approval of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in 2019-which treats most CF genotypes.

Methods: We identified peer-reviewed literature for a narrative review (January 1990 to January 2025) describing longitudinal trends in CF survival and age of death and assessing the influence of CFTRm, particularly ELX/TEZ/IVA. To supplement the existing literature, a secondary analysis of historical, longitudinal trends in the United States CF Foundation Patient Registry (U.S. CFFPR, 1990-2023) was conducted using recent available data.

Results: Quantitative data from published studies show that the median age of survival and death increased over time but with varying magnitudes across regions. Most cohort and registry-based studies were conducted in settings where CFTRm were not yet widely available, limiting the evaluation of CFTRm effects on survival trends over time. In the secondary U.S. CFFPR analysis, the median survival age increased from 29.0 years in 1990 to 38.6 years in 2012 prior to the introduction of CFTRm and to 68.0 years in 2023, demonstrating substantial improvement following the introduction of CFTRm. Linear regression analyses showed gains in median survival age increased from 0.48 years per year prior to CFTRm to 4.79 years per year after approval of ELX/TEZ/IVA in 2019.

Conclusions: Study results provide initial evidence of the impact of CFTRm to meaningfully improve survival. Longer-term follow-up data across geographies will provide a deeper understanding of the full impact of CFTRm on predicted CF survival and mortality.

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来源期刊

Pulmonary Therapy Medicine-Pulmonary and Respiratory Medicine

CiteScore

5.20

自引率

3.30%

发文量

审稿时长

6 weeks

期刊介绍： Aims and Scope Pulmonary Therapy is an international, open access, peer-reviewed (single-blind), and rapid publication journal. The scope of the journal is broad and will consider all scientifically sound research from pre-clinical, clinical (all phases), observational, real-world, and health outcomes research around the use of pulmonary therapies, devices, and surgical techniques. Areas of focus include, but are not limited to: asthma; chronic obstructive pulmonary disease; idiopathic pulmonary fibrosis; pulmonary hypertension; cystic fibrosis; lung cancer; respiratory tract disorders; allergic rhinitis and other respiratory allergies; influenza, pneumococcal infection, respiratory syncytial virus and other respiratory infections; and inhalers and other device therapies. The journal is of interest to a broad audience of pharmaceutical and healthcare professionals and publishes original research, reviews, case reports/series, trial protocols and short communications such as commentaries and editorials. Pulmonary Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of quality research, which may be considered of insufficient interest by other journals. Rapid Publication The journal’s publication timelines aim for a rapid peer review of 2 weeks. If an article is accepted it will be published 3–4 weeks from acceptance. The rapid timelines are achieved through the combination of a dedicated in-house editorial team, who manage article workflow, and an extensive Editorial and Advisory Board who assist with peer review. This allows the journal to support the rapid dissemination of research, whilst still providing robust peer review. Combined with the journal’s open access model this allows for the rapid, efficient communication of the latest research and reviews, fostering the advancement of pulmonary therapies. Open Access All articles published by Pulmonary Therapy are open access. Personal Service The journal’s dedicated in-house editorial team offer a personal “concierge service” meaning authors will always have an editorial contact able to update them on the status of their manuscript. The editorial team check all manuscripts to ensure that articles conform to the most recent COPE, GPP and ICMJE publishing guidelines. This supports the publication of ethically sound and transparent research. 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