Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease.

Abstract

OBJECTIVE:

To investigate the short-term adverse events and effectiveness of lumacaftor/ivacaftor combination treatment in adults with cystic fibrosis (CF) and severe lung disease in a real life setting.

METHODS:

A multicentre observational study investigated adverse events, treatment discontinuation, FEV1 and body mass index (BMI) one month and three months after lumacaftor/ivacaftor initiation in adults with CF and FEV1 below 40% predicted.

RESULTS:

Respiratory adverse events (AEs) were reported by 27 of 53 subjects (51%) and 16 (30%) discontinued treatment. The mean absolute change in FEV1 was +2.06% after one month of treatment (P=0.086) and +3.19% after 3 months (P=0.009). BMI was unchanged.

CONCLUSIONS:

Treatment with lumacaftor/ivacaftor in patients with CF and severe lung disease was discontinued more frequently than reported in clinical trials, due to respiratory AEs. Nevertheless, the patients who continued treatment had an increase in lung function comparable to what was observed in pivotal trials.

• Authors: Dominique Hubert, Raphaël Chiron, Boubou Camara, Dominique Grenet, Anne Prévotat, Laurence Bassinet, Stéphane Dominique, Gilles Rault, Julie Macey, Isabelle Honoré, Reem Kanaan, Sylvie Leroy, Nadine Desmazes Dufeu, Pierre-Régis Burgel
• Journal: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society (J Cyst Fibros) Vol. 16 Issue 3 Pg. 388-391 (May 2017) ISSN: 1873-5010 [Electronic] Netherlands
• PMID: 28325531 (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
• Aminopyridines
• Benzodioxoles
• Drug Combinations
• Membrane Transport Modulators
• Quinolones
• lumacaftor, ivacaftor drug combination
• Cystic Fibrosis Transmembrane Conductance Regulator

Topics

• Adult
• Aminophenols (administration & dosage, adverse effects)
• Aminopyridines (administration & dosage, adverse effects)
• Benzodioxoles (administration & dosage, adverse effects)
• Cystic Fibrosis (diagnosis, drug therapy, genetics)
• Cystic Fibrosis Transmembrane Conductance Regulator (genetics)
• Drug Combinations
• Drug Monitoring (methods)
• Female
• France
• Humans
• Male
• Membrane Transport Modulators (administration & dosage, adverse effects)
• Mutation
• Outcome and Process Assessment, Health Care
• Quinolones (administration & dosage, adverse effects)
• Respiratory Function Tests (methods)
• Severity of Illness Index
• Withholding Treatment (statistics & numerical data)