P172 CFTR Modulators in children with cystic fibrosis: real-life evidence in Turkey

The aim of the study was to evaluate the effect of CFTR modulators on clinical and laboratory findings of cystic fibrosis patients. Patients under the age of 18 who were followed up with the diagnosis of CF and using modulator therapy for at least 1 month between December 2018 and December 2022 were included. The clinical and laboratory data of the patients were evaluated retrospectively. 51 patients were included. The median age was 12 (8.3–15.1) years. 20 (39.2%) patients had homozygous and 16 (31.3%) patients had heterozygous F508del mutations. 10 (19.6%) patients were receiving ivacaftor, 39 (76.4%) patients were receiving elexacaftor-tezacaftor-ivacaftor, 2 (3.9%) patients were receiving lumacaftor-ivacaftor. The mean duration of using modulators were 6.3 (min-max;1–48) months. 2 (3.9%) patients were using continuous oxygen therapy, 2 (3.9%) patients were using continuous oxygen and BIPAP therapy. Median BMI z score at pretreatment,1 month and 3 months were; –0.7 (IQR: –1.4–0.4), –0.2(IQR: –1–0.5), –0.2 (IQR:-0.7–0.4). Median FEV1%at pre-treatment,1 month and 3 months were; 81% (IQR:60.5–100.3),102.5% (IQR:84.2–112.5), 98.5% (IQR:79.7–111.7). Median cystic fibrosis quality of life questionnaire respiratory score (CFQR-R) at pre-treatment, 1 month and 3 months were; 66.6 (IQR:45.8–76.3), 88.8 (IQR:75–100),91 (IQR:83.3–100) respectively. A significant increase was observed in BMI z score, FEV1% and CFQR-R scores at the third months compared to pre-treatment (p < 0.01). Side effects were observed in 15 (29,4%) patients. Eight patients (15.6%) had maculopapular rash, 3 (5.9%)patients had elevated transaminases, 2 (3.9%) patients had increased bilirubin without elevated transaminases, 1 (1.9%) patient had dyspeptic complaints, and 1 (1.9%)patient had obliviousness and tremors were observed. In our study, it was observed that patients using CFTR modulator therapy had positive contributions to quality of life, respiratory functions, and weight gain from the beginning of treatment even in three months.