{"title":"Epidemiology of advanced cystic fibrosis lung disease in the modulator era.","authors":"Julia Westhoff, Lutz Naehrlich","doi":"10.1097/MCP.0000000000001115","DOIUrl":null,"url":null,"abstract":"<p><strong>Purpose of review: </strong>Advanced cystic fibrosis lung disease remains the main cause of death in people with cystic fibrosis (pwCF). Cystic fibrosis transmembrane regulator (CFTR) modulators have changed the disease burden for eligible pwCF with access to this therapy.</p><p><strong>Recent findings: </strong>Real-world data show that there are no safety concerns for patients with advanced cystic fibrosis lung disease treated with highly effective triple CFTR modulator therapy. The improvements are comparable to those in other people with cystic fibrosis and in part even better. Mortality and rates of lung transplantation have decreased since the approval of CFTR modulator therapy and, especially, highly effective triple CFTR modulator therapy. Nevertheless, at least 10% of people with cystic fibrosis are not eligible for highly effective CFTR modulator therapy, and the development of alternative treatments remains important.</p><p><strong>Summary: </strong>The approval of highly effective CFTR modulator therapies has been a breakthrough in treatment for most people with cystic fibrosis, especially those with advanced lung disease, improving survival and reducing the burden of the disease.</p>","PeriodicalId":11090,"journal":{"name":"Current Opinion in Pulmonary Medicine","volume":null,"pages":null},"PeriodicalIF":2.8000,"publicationDate":"2024-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Current Opinion in Pulmonary Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1097/MCP.0000000000001115","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"RESPIRATORY SYSTEM","Score":null,"Total":0}
引用次数: 0
Abstract
Purpose of review: Advanced cystic fibrosis lung disease remains the main cause of death in people with cystic fibrosis (pwCF). Cystic fibrosis transmembrane regulator (CFTR) modulators have changed the disease burden for eligible pwCF with access to this therapy.
Recent findings: Real-world data show that there are no safety concerns for patients with advanced cystic fibrosis lung disease treated with highly effective triple CFTR modulator therapy. The improvements are comparable to those in other people with cystic fibrosis and in part even better. Mortality and rates of lung transplantation have decreased since the approval of CFTR modulator therapy and, especially, highly effective triple CFTR modulator therapy. Nevertheless, at least 10% of people with cystic fibrosis are not eligible for highly effective CFTR modulator therapy, and the development of alternative treatments remains important.
Summary: The approval of highly effective CFTR modulator therapies has been a breakthrough in treatment for most people with cystic fibrosis, especially those with advanced lung disease, improving survival and reducing the burden of the disease.
期刊介绍:
Current Opinion in Pulmonary Medicine is a highly regarded journal offering insightful editorials and on-the-mark invited reviews, covering key subjects such as asthma; cystic fibrosis; infectious diseases; diseases of the pleura; and sleep and respiratory neurobiology. Published bimonthly, each issue of Current Opinion in Pulmonary Medicine introduces world renowned guest editors and internationally recognized academics within the pulmonary field, delivering a widespread selection of expert assessments on the latest developments from the most recent literature.