Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting.

Abstract

BACKGROUND:

Ivacaftor has been shown to improve lung function and body weight in patients with CF and a gating mutation. Real-world evaluation is warranted to examine its safety and effectiveness over the long term.

METHODS:

A retrospective observational multicentre study collected clinical data in the year before and the 2years after ivacaftor initiation in patients with CF and a Gly551Asp-CFTR mutation.

RESULTS:

Fifty-seven patients were included. Mean absolute change in FEV1% predicted improved from baseline to Year 1 (8.4%; p<0.001) and Year 2 (7.2%; p=0.006). Statistically significant benefits were observed with increased body mass index, fewer Pseudomonas aeruginosa and Staphylococcus aureus positive cultures, and decreased IV antibiotics and maintenance treatment prescriptions (including azithromycin, Dornase alpha and nutritional supplements). No significant adverse events were reported.

CONCLUSION:

The clinical benefits of ivacaftor reported in previous clinical trials were confirmed in a real-world setting two years post-initiation, also reducing treatment burden.

• Authors: Dominique Hubert, Clémence Dehillotte, Anne Munck, Valérie David, Jinmi Baek, Laurent Mely, Stéphane Dominique, Sophie Ramel, Isabelle Danner Boucher, Sylvaine Lefeuvre, Quitterie Reynaud, Virginie Colomb-Jung, Prissile Bakouboula, Lydie Lemonnier
• Journal: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society (J Cyst Fibros) Vol. 17 Issue 1 Pg. 89-95 (01 2018) ISSN: 1873-5010 [Electronic] Netherlands
• PMID: 28711222 (Publication Type: Journal Article, Multicenter Study, Observational Study)
• Copyright: Copyright © 2017 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

Chemical References

• Aminophenols
• Anti-Bacterial Agents
• Chloride Channel Agonists
• Quinolones
• Cystic Fibrosis Transmembrane Conductance Regulator
• ivacaftor

Topics

• Adolescent
• Adult
• Aminophenols (therapeutic use)
• Anti-Bacterial Agents (therapeutic use)
• Child
• Chloride Channel Agonists (therapeutic use)
• Cystic Fibrosis (diagnosis, drug therapy, epidemiology, genetics)
• Cystic Fibrosis Transmembrane Conductance Regulator (genetics)
• Female
• France (epidemiology)
• Humans
• Male
• Pseudomonas aeruginosa (isolation & purification)
• Quinolones (therapeutic use)
• Respiratory Function Tests
• Respiratory System (microbiology, physiopathology)
• Staphylococcus aureus (isolation & purification)
• Time