Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease.
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, FEV1and body mass index (BMI) one month and three months after lumacaftor/ivacaftor initiation in adults with CF and FEV1below 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 FEV1was +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.
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.
Dominique
D
Hubert
Pulmonary Department, Arnaud de Villeneuve Hospital, Montpellier, France.
Raphaël
R
Chiron
Pulmonary Department, University Hospital Grenoble Alpes, Grenoble, France.
Boubou
B
Camara
Adult CF and Lung Transplantation Centre, Foch Hospital, Suresnes, France.
Dominique
D
Grenet
Pulmonary Department and Adult CF Centre, Lille University Hospital, Lille, France.
Anne
A
Prévotat
Pulmonary Department, Centre Intercommunal de Créteil, Créteil, France.
Laurence
L
Bassinet
Pulmonary Department, Charles Nicolle Hospital, Rouen University Hospital, Rouen, France.
Stéphane
S
Dominique
CF centre, Centre héliomarin de Perharidy, Roscoff, France.
Gilles
G
Rault
Pulmonary Department and adult CF centre, Bordeaux University Hospital, Bordeaux, France.
Julie
J
Macey
Pulmonary Department and Adult CF Centre, Cochin Hospital, AP-HP, Paris, France; Université Paris Descartes, Sorbonne Paris Cité, Paris, France.
Isabelle
I
Honoré
Pulmonary Department and Adult CF Centre, Cochin Hospital, AP-HP, Paris, France; Université Paris Descartes, Sorbonne Paris Cité, Paris, France.
Reem
R
Kanaan
FHU OncoAge, Côte d'Azur University, Nice, France.
Sylvie
S
Leroy
Adult CF Centre, CHU Nord, AP-HM, Marseille, France.
Nadine
N
Desmazes Dufeu
Pulmonary Department and Adult CF Centre, Cochin Hospital, AP-HP, Paris, France; Université Paris Descartes, Sorbonne Paris Cité, Paris, France.
Pierre-Régis
PR
Burgel
D000328:Adult; D000627:Aminophenols / Q000008:administration & dosage / Q000009:adverse effects; D000631:Aminopyridines / Q000008:administration & dosage / Q000009:adverse effects; D052117:Benzodioxoles / Q000008:administration & dosage / Q000009:adverse effects; D003550:Cystic Fibrosis / Q000175:diagnosis / Q000188:drug therapy / Q000235:genetics; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics*; D004338:Drug Combinations; D016903:Drug Monitoring / Q000379:methods; D005260:Female; D005602:France; D006801:Humans; D008297:Male; D049990:Membrane Transport Modulators / Q000008:administration & dosage / Q000009:adverse effects; D009154:Mutation; D010043:Outcome and Process Assessment, Health Care; D015363:Quinolones / Q000008:administration & dosage / Q000009:adverse effects; D012129:Respiratory Function Tests / Q000379:methods; D012720:Severity of Illness Index; D028761:Withholding Treatment / Q000706:statistics & numerical data
D000627:Aminophenols; D000631:Aminopyridines; D052117:Benzodioxoles; D004338:Drug Combinations; D049990:Membrane Transport Modulators; D015363:Quinolones; C000599212:lumacaftor, ivacaftor drug combination; D019005:Cystic Fibrosis Transmembrane Conductance Regulator
CFTR correctors; Cystic fibrosis; Lumacaftor/ivacaftor