Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*)

Overview

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.

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

Conditions

Interventions

Eligibility

Locations (1)

Outcomes