Criteria used to start and monitor CFTR modulator therapy
Table 2 presents the distribution of responses on criteria used to start CFTRm therapy. The number of exacerbations is perceived by respondents as the most useful parameter to decide to start the therapy with CFTR modulators: this parameter is deemed “somewhat useful” to “very useful” by 95% of respondents for F508del -homozygous pediatric patients, by 97% for F508de l-homozygous adult patients and by 90% for patients with residual function. Following respiratory outcomes, BMI z-score and ability to perform activities of daily living are also considered useful parameters in F508del-homozygous pediatric (“somewhat useful” to “very useful” in 95% and 80% of respondents, respectively) and adult patients (“somewhat useful” to “very useful” in 95% and 90% of respondents, respectively) and in patients with residual function (“somewhat useful” to “very useful” in 86% and 80% of respondents, respectively). Bacterial colonization is deemed somewhat to very helpful for 56-68% of respondents (highest percentage in young homozygous patients). Of note, only exacerbations get close to 50% of opinions of high usefulness (very useful 41-51%). The same parameters are deemed useful to monitor CFTRm therapy and in similar proportions, although growth percentiles and Lung clearance index (LCI) are considered somewhat more important for youngF508del -homozygous patients (Table 3). Lung imaging was also deemed useful in all groups for both initiation and monitoring of therapy (68-75% somewhat/very useful). In addition, sweat chloride is considered somewhat to very useful for about 75% of physicians in all groups of patients.