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.