CFTRm use according to disease stage and selection of CFTRm indicated in same patient group
Table 4 shows the distribution of responses on CFTRm use by disease stage and patient groups (percentage of physicians who would consider use in all patients). Systematic use in asymptomatic patients would be considered in 19, 27 and 17% of pediatric F508del homozygous, adult F508del homozygous and patients with residual function mutations, respectively while 54 to 58% of respondents would evaluate CFTRm use case by case (data not shown). Favorable opinion of use in all patients increases with severity of clinical presentation with 53-56% positive responses for mild disease stage, 69-78% in case of fully expressed stable disease and 90-92% for fully expressed disease with worsening symptoms. About half of respondents would consider CFTRm in terminal disease stage in patients >12 years F508delhomozygous and RF while most physicians would evaluate use case by case for non adherent patients (data not shown). Half of respondents (51%) would choose to use tezacaftor/ivacaftor association to start CFTR modulator therapy in F508del homozygous patients compared to 17% who would start with lumacaftor/ivacaftor. The main very important reasons to start with lumacaftor/ivacaftor were clinical experience (60%) and safety profile (80%) while strong motivation for starting with tezacaftor/ivacaftor was based on expectation of efficacy for 53% of health care professionals. No significant center-effect or correlation between answers was found.