PMID- 28325531
OWN - NLM
STAT- MEDLINE
DCOM- 20180312
LR  - 20191210
IS  - 1873-5010 (Electronic)
IS  - 1569-1993 (Linking)
VI  - 16
IP  - 3
DP  - 2017 May
TI  - Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic 
      fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease.
PG  - 388-391
LID - S1569-1993(17)30086-3 [pii]
LID - 10.1016/j.jcf.2017.03.003 [doi]
AB  - 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, 
      FEV(1) and body mass index (BMI) one month and three months after 
      lumacaftor/ivacaftor initiation in adults with CF and FEV(1) 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 FEV(1) 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.
CI  - Copyright (c) 2017 European Cystic Fibrosis Society. Published by Elsevier B.V. All 
      rights reserved.
FAU - Hubert, Dominique
AU  - Hubert D
AD  - Pulmonary Department and Adult CF Centre, Cochin Hospital, AP-HP, Paris, France; 
      Universite Paris Descartes, Sorbonne Paris Cite, Paris, France. Electronic 
      address: dominique.hubert@aphp.fr.
FAU - Chiron, Raphael
AU  - Chiron R
AD  - Pulmonary Department, Arnaud de Villeneuve Hospital, Montpellier, France.
FAU - Camara, Boubou
AU  - Camara B
AD  - Pulmonary Department, University Hospital Grenoble Alpes, Grenoble, France.
FAU - Grenet, Dominique
AU  - Grenet D
AD  - Adult CF and Lung Transplantation Centre, Foch Hospital, Suresnes, France.
FAU - Prevotat, Anne
AU  - Prevotat A
AD  - Pulmonary Department and Adult CF Centre, Lille University Hospital, Lille, 
      France.
FAU - Bassinet, Laurence
AU  - Bassinet L
AD  - Pulmonary Department, Centre Intercommunal de Creteil, Creteil, France.
FAU - Dominique, Stephane
AU  - Dominique S
AD  - Pulmonary Department, Charles Nicolle Hospital, Rouen University Hospital, Rouen, 
      France.
FAU - Rault, Gilles
AU  - Rault G
AD  - CF centre, Centre heliomarin de Perharidy, Roscoff, France.
FAU - Macey, Julie
AU  - Macey J
AD  - Pulmonary Department and adult CF centre, Bordeaux University Hospital, Bordeaux, 
      France.
FAU - Honore, Isabelle
AU  - Honore I
AD  - Pulmonary Department and Adult CF Centre, Cochin Hospital, AP-HP, Paris, France; 
      Universite Paris Descartes, Sorbonne Paris Cite, Paris, France.
FAU - Kanaan, Reem
AU  - Kanaan R
AD  - Pulmonary Department and Adult CF Centre, Cochin Hospital, AP-HP, Paris, France; 
      Universite Paris Descartes, Sorbonne Paris Cite, Paris, France.
FAU - Leroy, Sylvie
AU  - Leroy S
AD  - FHU OncoAge, Cote d'Azur University, Nice, France.
FAU - Desmazes Dufeu, Nadine
AU  - Desmazes Dufeu N
AD  - Adult CF Centre, CHU Nord, AP-HM, Marseille, France.
FAU - Burgel, Pierre-Regis
AU  - Burgel PR
AD  - Pulmonary Department and Adult CF Centre, Cochin Hospital, AP-HP, Paris, France; 
      Universite Paris Descartes, Sorbonne Paris Cite, Paris, France.
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
PT  - Observational Study
DEP - 20170318
PL  - Netherlands
TA  - J Cyst Fibros
JT  - Journal of cystic fibrosis : official journal of the European Cystic Fibrosis 
      Society
JID - 101128966
RN  - 0 (Aminophenols)
RN  - 0 (Aminopyridines)
RN  - 0 (Benzodioxoles)
RN  - 0 (Drug Combinations)
RN  - 0 (Membrane Transport Modulators)
RN  - 0 (Quinolones)
RN  - 0 (lumacaftor, ivacaftor drug combination)
RN  - 126880-72-6 (Cystic Fibrosis Transmembrane Conductance Regulator)
SB  - IM
CIN - J Cyst Fibros. 2017 May;16(3):311-312. PMID: 28433526
MH  - Adult
MH  - *Aminophenols/administration & dosage/adverse effects
MH  - *Aminopyridines/administration & dosage/adverse effects
MH  - *Benzodioxoles/administration & dosage/adverse effects
MH  - *Cystic Fibrosis/diagnosis/drug therapy/genetics
MH  - Cystic Fibrosis Transmembrane Conductance Regulator/*genetics
MH  - Drug Combinations
MH  - Drug Monitoring/methods
MH  - Female
MH  - France
MH  - Humans
MH  - Male
MH  - Membrane Transport Modulators/administration & dosage/adverse effects
MH  - Mutation
MH  - Outcome and Process Assessment, Health Care
MH  - *Quinolones/administration & dosage/adverse effects
MH  - Respiratory Function Tests/methods
MH  - Severity of Illness Index
MH  - Withholding Treatment/statistics & numerical data
OTO - NOTNLM
OT  - CFTR correctors
OT  - Cystic fibrosis
OT  - Lumacaftor/ivacaftor
EDAT- 2017/03/23 06:00
MHDA- 2018/03/13 06:00
CRDT- 2017/03/23 06:00
PHST- 2016/11/29 00:00 [received]
PHST- 2017/02/25 00:00 [revised]
PHST- 2017/03/01 00:00 [accepted]
PHST- 2017/03/23 06:00 [pubmed]
PHST- 2018/03/13 06:00 [medline]
PHST- 2017/03/23 06:00 [entrez]
AID - S1569-1993(17)30086-3 [pii]
AID - 10.1016/j.jcf.2017.03.003 [doi]
PST - ppublish
SO  - J Cyst Fibros. 2017 May;16(3):388-391. doi: 10.1016/j.jcf.2017.03.003. Epub 2017 
      Mar 18.