Pediatria. Journal named after G.N. Speransky | VOL. 101
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ПЕРВЫЕ РЕЗУЛЬТАТЫ ТЕРАПИИ ДВУМЯ CFTR-МОДУЛЯТОРАМИ ПРИ МУКОВИСЦИДОЗЕ В ДЕТСКОМ ВОЗРАСТЕ

Publication Date Jun 17, 2022

Abstract

In the treatment of cystic fibrosis (CF), new methods of precision medicine have emerged. This is due to the discovery of pathogenetic modulator drugs that eliminate the main defects in pathogenic variants of the CFTR gene. Objective of the study: to analyze the efficacy and safety of two CFTR modulators in children with CF. Materials and methods of research: a single-center, experimental, open, controlled, non-randomized study was conducted from November 2021 to March 2022, 2 groups were studied: 1st group [23 patients (7 M/16 F), homozygous for F508del with confirmed homozygosity and exclusion of the F508Del; L467F complex Allela] received ivacaftor/lumacaftor; 2nd group [20 patients (6 M/14 F), compound heterozygous for F508del or other pathogenic variants of the CFTR gene] received an elexacaftor/tezacaftor/ivacaftor. We studied the dynamics of indicators at the start and after 3 months: sweat test, body mass index (BMI), external respiration (ER), levels of ALT, AST, total bilirubin, blood pressure (Bp). Results: when using ivacaftor/lumacaphore in 23 patients, the median age ME (Q1–Q3) 14.8 (11.3–16.2) years, a statistically significant increase in body weight: from 38.8 (28.6–48,4) kg up to 44.8 (29.4–52.1) kg after 3 months (p=0.007), decrease in sweat conductivity (mmol/l) Me (Q1–Q3) 113.0 (100–120) at the start, 89.0 (76–92.5) after 3 months (p=0.012) was noted. Unwanted side effects (USEs) are registered in 47.8% of cases. When evaluating the effectiveness of the elekfator/thezacaftor/ivakaftor drug in 20 patients...

Concepts

Q1 Q3 Increase In Body Weight Unwanted Side Effects Sweat Conductivity CFTR Modulator Body Mass Index External Respiration Cystic Fibrosis Forced Vital Capacity Sweat Test

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No potential conflict of interest was reported by the authors. The conception and design of the study, acquisition of data, analysis and interpretatio...

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