Triple combination cystic fibrosis transmembrane conductance regulator modulator therapy in the real world – opportunities and challenges

Purpose of review The purpose of this review is to describe on-going and upcoming real-world studies that will aid the cystic fibrosis (CF) community in understanding the long-term efficacy, safety and challenges in utilizing this therapy and managing care. Recent findings The triple combination of elexacaftor, tezacaftor and ivacaftor (ETI) has been demonstrated to improve lung function, weight and quality of life in children and adults with CF with at least one copy of Phe508del. Treatment with ETI will potentially be available for approximately 90% of the CF population and change the face of CF. Summary In spite of early demonstrations of short-term efficacy and safety, for medications that may be given for much of someone's life, continued assessment of these outcomes is necessary. Furthermore, the CF community must evaluate and address the issues that arise with increased longevity including parenthood, preventive care management and the potential comorbidities of aging.
Source: Current Opinion in Pulmonary Medicine - Category: Respiratory Medicine Tags: CYSTIC FIBROSIS: Edited by Peter J. Barry and Barry J. Plant Source Type: research