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.