Efficacy of targeted therapy with lumacaftor/ivacaftor in children with cystic fibrosis (12-month follow-up)

Lumacaftor/ivacaftor is a combination of two low-molecular-weight drugs aimed at eliminating the main defect of cystic fibrosis (MV) at the cellular level. Restoration of the function of the chlorine (CFTR) channel in MV is necessary for normalization of mucociliary clearance, nutritional status of patients, improvement of other clinical and laboratory parameters. This CFTR modulator is approved for the treatment of homozygotes according to the F508del variant, the most common among patients with MV. Objective: to study the effectiveness of lumacaftor/ivacaftor CFTR modulator therapy in children with cystic fibrosis after 12 months of treatment and follow-up. Materials and methods. The data of 50 patients from among 135 children over 6 years of age who received lumacaftor/ivacaftor, presented in the “Register of patients with cystic fibrosis of the Russian Federation” for 2021-2022, were analyzed. The indicators of external respiration function (FVD) - FEV1, FVC, sweat conductivity, anthropometric data at the start of targeted therapy and after 12 months of treatment were analyzed. Results. During 12 months of treatment, there was an increase in body weight from Iu (Q1-Q3) 36.2 (27.5-46.1) to 40.5 (30.8-50.0) kg, p<0.001, height - from 148.8 (134.8-159.3) to 155.0 (137.9-162.5) cm, p<0.001. Body mass index (BMI) (kg / m2) at the start of therapy was 16.4 (14.7-17.8), and a year later - 17.4 (15.9-19.1), p<0.001. The conductivity of sweat before the start of therapy was equivalent to 113.0 (104.0-122.0) mmol / l NaCl, and after 12 months of therapy with lumacaftor/ ivacaftor - 90.0 (75.5-103.0) mmol \\ l NaCl, p<0.001. There were no statistically significant changes in the indicators of external respiration function according to spirometry data. Conclusion: after 12 months of lumacaftor/ivacaftor therapy, statistically significant positive dynamics of weight, height, BMI and sweat test were observed in children with MV older than 6 years compared with the indicators before the start of targeted therapy.

low-molecular-weight drug, cystic fibrosis, CFTR modulator

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