P171 Clinical experience with the introduction of a programme with CFTR modulators in a paediatric cystic fibrosis centre in Poland - a preliminary report

In 2022, Poland joined the elite group of countries where CFTR modulators (mCFTR) are reimbursed. The aim this prospective study was analysis selected nutritional and pulmonary function parameters their impact on patient health-related quality life in CF children before 3 months after introduction mCFTR therapy. patients aged 2–18 consecutively enrolled program were assessed during trimonthly visits. status lung (FEV1, FVC, FEV1/FVC, LCI), evaluated. Subjects from 12 years age completed CFQ-R. From Jul to Dec 90 started therapy, 75 them performed tests (mean 12.9 ± 3.6): 23 received tezacaftor/ivacaftor (TI), 52 elexacaftor/tezacaftor/ivacaftor (ETI). TI group, FEV1 increased 13 (57%) (average 8,99% 6,20, p = 0,001), FVC 11 (48%) 4.82% 4.23, 0.01), FEV1/FVC 5.42% 4.76, 0.003). LCI decreased 15 (65%) 1.06 1.22, < 0.001). ETI 39 (75%) 10,45% 6,46), 33 (63%) 5.65% 4.25), 38 (73%) 7.0 4.79); 47 (90%) 1.95 1.44), 0.001. Average weight gain 0.65 1.43 kg 2.57 2.16 (p Change CFQ-R Physical domain correlated with change (R –0.29), FEV1%pp. 0.30), z-score 0,32), FVC% pp 0.29), 0,34), 0.05). A significant improvement Respiratory also noted 0.002). After therapy improved most group. Patients treated compared those achieved better clinical results. evaluation test