The purpose of this study is to determine whether the treatment with Ivacaftor remains effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR mutation) in the real life setting, after the drug has been approved by the Health authorities.
The aims of our study are: 1. to describe the treated population at initiation of treatment, 2. to evaluate clinical parameters during the year before Ivacaftor was started, at initiation of treatment and during at least one year of treatment, until June 2014. 3. to evaluate the tolerance and safety of this treatment.
Study Type
OBSERVATIONAL
Enrollment
57
Hôpital Cochin
Paris, France
FEV1 (in liters and in % predicted)
pulmonary function
Time frame: until one years after initiation of treatment
pulmonary exacerbations
Time frame: until one years after initiation of treatment
number of hospitalizations and number of days of hospitalization per year
Time frame: until one year after initiation of treatment
number of antibiotic treatments and number of days of antibiotic treatments
number of oral antibiotic treatments and number of days of oral antibiotic treatments, number of IV courses and days of IV antibiotics per year
Time frame: until one year after initiation of treatment
respiratory colonization
Evolution of bacteria and fungi in sputum
Time frame: until least one year after initiation of treatment
nutritional status
Weight (and BMI-Zscore for children)
Time frame: until one year after initiation of treatment
Adverse events
Dates and reasons for interruption and discontinuation of treatment with Ivacaftor Adverse events, indicating what in the physician's opinion might be due to Ivacaftor
Time frame: until one year after initiation of treatment
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