Cystic fibrosis modulator therapy can reverse cystic bronchiectasis

This report describes the reversal of cystic bronchiectasis following a year of CF modulator therapy, providing exciting new insights into the pathogenesis of bronchiectasis in CF. AbstractBronchiectasis is often considered progressive and irreversible, so cases of regression or reversal are an important step in understanding the underlying pathophysiological mechanisms. Cystic fibrosis, (CF) caused by pathogenic variants in thecystic fibrosis transmembrane conductance regulator (CFTR) gene has been a success story in personalized medicine. The recent development ofCFTR modulator therapies has revolutionized care. Dramatic improvements in lung function, sputum production, daytime functioning, and quality of life are seen within weeks. However, the effect of long-term exposure to elexacaftor + tezacaftor + ivacaftor (ETI) on the structural abnormalities is at present unknown. This case series outlines three adults with CF who have demonstrated progressive improvement in the cylindrical, varicose and importantly cystic changes of bronchiectasis with prolonged ETI treatment. This raises the exciting question of reversibility of bronchiectasis as well as the mechanisms involved in the maintenance and progression of bronchiectasis as it relates to CF.
Source: Respirology Case Reports - Category: Respiratory Medicine Authors: Tags: CASE SERIES Source Type: research