Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, ivacaftor, lumacaftor
Brief summary
The purpose of the study is to examine the real-life safety and effectiveness of the novel combination ivacaftor+lumacaftor in eligible patients with cystic fibrosis (CF). All patients with CF were eligible if they were 12 years and older, started ivacaftor+lumacaftor outside of a clinical trial between December 15th 2017 and December 15th 2018 in an accredited CF center in France. Patient followed-up is based on standardized recommendation of the French Cystic Fibrosis Society. Each patient is followed 1 year.
Detailed description
Each patient is followed one year with visits at months 1, 3, 6 and 12. At each visit, the following data are recorded: * Treatment discontinuation or not. If the treatment was discontinued, reasons for discontinuation * Adverse effects * Lung function (spirometry) * Body mass index * Pulmonary exacerbations (intravenous antibiotics) * Sputum microbiology * Liver enzymes are measured at each visit At the initial and 12 visits, a yearly CF examination is proposed to the patients: * Blood tests * Chest CT scans * Body plethysmography
Interventions
DRUGIvacaftor+lumacaftor
1 year follow-up after initiation of ivacaftor+lumacaftor
Sponsors
Effi-Stat
Societe Francaise de la Mucoviscidose
URC-CIC Paris Descartes Necker Cochin
Assistance Publique - Hôpitaux de Paris
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patient aged 12 years or older. * Patient with Cystic Fibrosis with presence of two mutations DF508 in the CFTR gene * Patient treated with ivacaftor+lumacaftor (Orkambi)
Exclusion criteria
* Refusal to participate in the study * Start of Orkambi as part of a clinical trial
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Rates of treatment discontinuation | 1 year |
| Timing of treatment discontinuation | 1 year |
| Causes of treatment discontinuation | 1 year |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pulmonary exacerbations | 1 year | Intravenous antibiotic courses |
| Forced expiratory volume in 1 sec (FEV1) | 1 year | to evaluate lung function |
| Chloride concentration | 1 year | Sweat test before and during treatment |
| Forced vital capacity (FVC) | 1 year | to evaluate lung function |
| Body mass index | 1 year | Nutritional status |
Countries
France
Outcome results
None listed