Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Case Reports
. 2023 Jun 12;11(7):e01172.
doi: 10.1002/rcr2.1172. eCollection 2023 Jul.

Cystic fibrosis modulator therapy can reverse cystic bronchiectasis

Peter G Middleton  1   2 , Nicholas J Simmonds  3   4
Affiliations
Case Reports

Cystic fibrosis modulator therapy can reverse cystic bronchiectasis

Peter G Middleton et al. Respirol Case Rep. .

Abstract

Bronchiectasis 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 the cystic fibrosis transmembrane conductance regulator (CFTR) gene has been a success story in personalized medicine. The recent development of CFTR 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.

Keywords: bronchiectasis; cystic fibrosis; elexacaftor; ivacaftor; tezacaftor.

PubMed Disclaimer

Conflict of interest statement

Peter Middleton reports grants from Vertex Pharmaceuticals, during the conduct of the study; personal fees from Vertex Pharmaceuticals, outside the submitted work. Nicholas Simmonds reports personal fees from Vertex Pharmaceuticals, Chiesi, Gilead, Menarini, Zambon, outside the submitted work.

Figures

FIGURE 1
FIGURE 1
CT scans before (A) and after (B) elexacaftor + tezacaftor + ivacaftor for case 1
FIGURE 2
FIGURE 2
CT scans (A) before and (B) after elexacaftor + tezacaftor + ivacaftor for case 2
FIGURE 3
FIGURE 3
CT scans (A) before and (B) after elexacaftor + tezacaftor + ivacaftor for case 3
See this image and copyright information in PMC

References

    1. Barker AF. Bronchiectasis. N Engl J Med. 2002;346:1383–93. - PubMed
    1. Cole PJ. Inflammation: a two‐edged sword—the model of bronchiectasis. Eur J Respir Dis Suppl. 1986;147:6–15. - PubMed
    1. O'Donnell AE. Bronchiectasis—a clinical review. N Engl J Med. 2022;387:533–45. - PubMed
    1. Flume PA, Chalmers JD, Olivier KN. Advances in bronchiectasis: endotyping, genetics, microbiome, and disease heterogeneity. Lancet. 2018;392:880–90. - PMC - PubMed
    1. Middleton PG, Mall MA, Drevinek P, Lands LC, McKone EF, Polineni D, et al. Elexacaftor—tezacaftor—ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381:1809–19. - PMC - PubMed

Publication types

LinkOut - more resources