Materials and methods
Participation to the survey was proposed through an online platform to all physicians from Italian CF centers and was voluntary and anonymous. No IRB approval was required considering the study type. Questionnaire was composed of 16 item including 3 profiling questions. The questionnaire focused on the three groups of patients for whom CFTR modulators were authorized at the time: F508del homozygous patients below and above 12 years of age and patients with RF mutations. Questions explored expectations of efficacy of CFTRm, criteria used to start therapy, monitoring parameters, treatment preferences according to disease stage and treatment preference in F508del homozygous patients. A Likert scale in 4 points (not at all, slightly, somewhat, a lot) was used to rank the answers. Descriptive statistical analysis was performed to depict the distribution of answers. CHI-squared test was used to investigate a possible center effect influencing the answers (clusterization) a statistical significance was considered when p-value was <0.05. Cohen’s kappa coefficient (κ ) was calculated to evaluate possible correlation between different answers, consideringk =1 as complete concordance.