Seminars in respiratory and critical care medicine
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Lung infection is the leading cause of death in cystic fibrosis (CF), and antimicrobial therapies are the backbone of infection management. While many different strategies may be applied, rigorous microbiological surveillance, intensive eradication therapy, and long-term maintenance therapy based on inhaled antibiotics may be considered the main strategy for infection control in individuals with CF. While most of the existing evidence is based on infection with Pseudomonas aeruginosa, other important pathogens causing lung inflammation and deterioration exist and should be treated despite the evidence gap. In this chapter, we describe the approaches to the antimicrobial treatment of the most important pathogens in CF and the evidence behind.
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Semin Respir Crit Care Med · Apr 2023
Advanced Cystic Fibrosis Lung Disease and Lung Transplantation in the Era of Cystic Fibrosis Transmembrane Conductance Regulator Modulators.
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have changed the clinical landscape of cystic fibrosis (CF) by improving clinically significant outcome measures and quality of life of people with CF (pwCF). There are now long-term data showing improved 5-year survival with the use of ivacaftor, and the field continues to evolve at a rapid pace with the continued development of highly effective CFTR modulators. While the randomized controlled trials of CFTR modulators excluded patients with severe lung disease (forced expiratory volume in 1 second <40% predicted), observational data based on case reports and registry data show similar benefits in those with advanced lung disease. ⋯ While the widespread availability of elexacaftor/tezacaftor/ivacaftor over the past 2 years has been associated with a sharp drop in the number of people referred for consideration for lung transplantation and the number of people wait-listed for lung transplantation, it is difficult to accurately determine the true impact due to the confounding effect of the coronavirus disease 2019 pandemic. It is expected that lung transplantation will remain an important treatment for a smaller number of pwCF. Lung transplantation offers survival benefits in CF, and there remains an imperative to ensure timely consideration of lung transplantation in patients with advanced disease to further reduce the number of pwCF dying without consideration of lung transplant.
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Semin Respir Crit Care Med · Apr 2023
ReviewDiagnosis and Management of Cystic Fibrosis Exacerbations.
With the improving survival of cystic fibrosis (CF) patients and the advent of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) therapy, the clinical spectrum of this complex multisystem disease continues to evolve. One of the most important clinical events for patients with CF in the course of this disease is acute pulmonary exacerbation (PEx). ⋯ This work has now led to several large-scale clinical trials designed to clarify the treatment paradigm for CF PEx. The primary goal of this review is to provide a summary and update of the pathophysiology, clinical and microbial epidemiology, outcome and treatment of CF PEx, biomarkers for exacerbation, and the impact of highly effective modulator therapy on these events moving forward.
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In cystic fibrosis, a new era has started with the approval and use of highly effective cystic fibrosis transport regulator (CFTR) modulator therapy. As pulmonary function is increasing and exacerbation rate significantly decreases, the current meaning of fungal pulmonary diseases is questioned. During the past couple of decades, several studies have been conducted regarding fungal colonization and infection of the airways in people with cystic fibrosis. ⋯ Whether the percentage will decrease or not will be the task of future evaluations in studies and registry analysis. Using the established definition for different categories of fungal diseases is recommended and should be taken into account if patients are deteriorating without responding to antibiotic treatment. Drug-drug interactions, in particular when using azoles, should be recognized and therapies need to be adjusted accordingly.
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Semin Respir Crit Care Med · Apr 2023
Impact of Digital Technologies on Clinical Care for Adults with Cystic Fibrosis.
The coronavirus disease 2019 pandemic accelerated the implementation of digital technologies, which have now become embedded as essential tools for the management of chronic disease, including cystic fibrosis (CF). Despite subsequent easing of restrictions and because of improved clinical stability resulting from the introduction of highly effective modulator therapy, digital technologies including video and telephone consultations and remote monitoring are likely to remain integral to the future delivery of CF health care. In this article, we explore some of the key developments in digital technologies, barriers to their adoption, and how the CF community is likely to embrace lessons learned from the recent pandemic to help modernize and reshape the future of CF care.