• J. Cyst. Fibros. · May 2017

    Multicenter Study Observational Study

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

    • 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, and Pierre-Régis Burgel.
    • Pulmonary Department and Adult CF Centre, Cochin Hospital, AP-HP, Paris, France; Université Paris Descartes, Sorbonne Paris Cité, Paris, France. Electronic address: dominique.hubert@aphp.fr.
    • J. Cyst. Fibros. 2017 May 1; 16 (3): 388-391.

    ObjectiveTo 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.MethodsA 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.ResultsRespiratory 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.ConclusionsTreatment 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.Copyright © 2017 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

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