Abstract
CFTR modulators (CFTRm) were introduced recently but they are already
profoundly changing Cystic Fibrosis (CF) landscape. This survey was
conducted in 2018 to evaluate how their perception and use evolved in
Italy, with focus on factors that could influence physician treatment
decisions. Response rate was 75.6% and the majority of physicians
(81%) had been working in CF for over 5 years. While traditional
parameters such as lung function and nutritional status remain key
evaluation criteria in relation to initiation and monitoring of CFTRm,
pulmonary exacerbations ranked at least at the same level of importance
in both pediatric and adolescent/adult patients homozygous forF508del , as well as those with residual function mutations.
Increasing interest is shown for tools that can help detect early
manifestations of disease such as Lung Clearance Index and imaging.
Patient-related outcomes, such as ability to conduct daily activities,
are also deemed relevant in decision to start and continue CFTRm.
Physician decision to initiate treatment according to clinical
presentation was similar in all groups, showing that more importance was
given to severity/instability of disease rather than mutation type or
age range. A relatively low percentage of physicians would treat
asymptomatic patients, in particular very young or those with residual
function mutations, showing reluctance to treat early in some patient
groups in the absence of clear manifestations of CF. Increasing
experience with CFTRm will allow to gain more long term evidence and
will help shape new guidelines.