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Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis
- Source :
- American Journal of Respiratory and Critical Care Medicine, American Journal of Respiratory and Critical Care Medicine, American Thoracic Society, 2020, 201 (2), pp.188-197. ⟨10.1164/rccm.201906-1227OC⟩, American Journal of Respiratory and Critical Care Medicine, 2020, 201 (2), pp.188-197. ⟨10.1164/rccm.201906-1227OC⟩
- Publication Year :
- 2019
-
Abstract
- International audience; Rationale: Lumacaftor-ivacaftor is a CFTR (cystic fibrosis transmembrane conductance regulator) modulator combination recently approved for patients with cystic fibrosis (CF) homozygous for the Phe508del mutation.Objectives: To evaluate the safety and effectiveness of lumacaftor-ivacaftor in adolescents (≥12 yr) and adults (≥18 yr) in a real-life postapproval setting.Methods: The study was conducted in the 47 CF reference centers in France. All patients who initiated lumacaftor-ivacaftor from January 1 to December 31, 2016, were eligible. Patients were evaluated for lumacaftor-ivacaftor safety and effectiveness over the first year of treatment following the French CF Learning Society's recommendations.Measurements and Main Results: Among the 845 patients (292 adolescents and 553 adults) who initiated lumacaftor-ivacaftor, 18.2% (154 patients) discontinued treatment, often owing to respiratory (48.1%, 74 patients) or nonrespiratory (27.9%, 43 patients) adverse events. In multivariable logistic regression, factors associated with increased rates of discontinuation included adult age group, percent predicted FEV1 (ppFEV1) less than 40%, and numbers of intravenous antibiotic courses during the year before lumacaftor-ivacaftor initiation. Patients with continuous exposure to lumacaftor-ivacaftor showed an absolute increase in ppFEV1 (+3.67%), an increase in body mass index (+0.73 kg/m2), and a decrease in intravenous antibiotic courses by 35%. Patients who discontinued treatment had significant decrease in ppFEV1, without improvement in body mass index or decrease in intravenous antibiotic courses.Conclusions: Lumacaftor-ivacaftor was associated with improvement in lung disease and nutritional status in patients who tolerated treatment. Adults who discontinued lumacaftor-ivacaftor, often owing to adverse events, were found at high risk of clinical deterioration.
- Subjects :
- Male
Cystic Fibrosis
Gastrointestinal Diseases
[SDV]Life Sciences [q-bio]
Aminopyridines
Quinolones
Critical Care and Intensive Care Medicine
Logistic regression
Aminophenols
Cystic fibrosis
Body Mass Index
Ivacaftor
chemistry.chemical_compound
0302 clinical medicine
Deprescriptions
Forced Expiratory Volume
Medicine
030212 general & internal medicine
Fatigue
2. Zero hunger
biology
Lumacaftor
Headache
lumacaftor–ivacaftor
Cystic fibrosis transmembrane conductance regulator
3. Good health
Anti-Bacterial Agents
Drug Combinations
Treatment Outcome
Administration, Intravenous
Female
France
medicine.drug
Pulmonary and Respiratory Medicine
Adult
medicine.medical_specialty
Metrorrhagia
Adolescent
Nutritional Status
03 medical and health sciences
Young Adult
Internal medicine
Product Surveillance, Postmarketing
Humans
Benzodioxoles
Adverse effect
Bronchial Spasm
business.industry
Editorials
Myalgia
medicine.disease
Discontinuation
Dyspnea
Logistic Models
030228 respiratory system
chemistry
Cough
Multivariate Analysis
biology.protein
postmarketing study
business
Body mass index
Subjects
Details
- ISSN :
- 15354970 and 1073449X
- Volume :
- 201
- Issue :
- 2
- Database :
- OpenAIRE
- Journal :
- American journal of respiratory and critical care medicine
- Accession number :
- edsair.doi.dedup.....0db392b714dac9ccdd6c21aebbb545e5