Expectations about CFTR modulators among physicians from Italian Cystic
fibrosis centers: a survey about the evolution of clinical practice
paradigms for cystic fibrosis
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 for F508del,
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.