Using elexacaftor/tezacaftor/ivacaftor + ivacaftor in preschool children with cystic fibrosis
https://doi.org/10.18093/0869-0189-2025-35-2-213-220
Abstract
In recent years, new drugs have been discovered to treat cystic fibrosis (CF), which directly target the primary defects of CFTR: correctors and potentiators.
The aim was to evaluate the efficacy and safety of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children with CF aged 2 – 6 years in routine clinical practice.
Methods. An open observational study enrolled patients diagnosed with CF aged 2 – 6 years (n = 8) who received elexacaftor/tezacaftor/ivacaftor and ivacaftor. The following parameters were evaluated over time: height, weight, body mass index (BMI), sweat chlorides, fecal elastase 1, alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, and glucose.
Results. During therapy, there was an absolute decrease in the level of sweat chlorides from an average of 110.12 (SD – 10.35) mmol/L at visit 1 to 61.37 (SD – 9.15) mmol/L (p < 0.05) after 1 month of treatment. The sweat chlorides level at visit 6 was 52.62 (SD – 7.67) mmol/l (p < 0.05). The body weight increased from 15.5 kg (SD – 2.4) to 18.8 kg (SD – 1.36) (p < 0.05), height – from 99.88 cm to 110.37 cm. The BMI varied from 15.76 (SD – 0.44) kg/m2 at baseline to 16.26 (SD – 0.23) by the 9th month (p < 0.05) and 15.02 (SD – 0.13) kg/m2 by the 12th month of follow-up (p > 0.05). The level of fecal
elastase-1 grew from 143.14 μg/g of stool (SD – 77.12) to 240.3 μg/g of stool (SD – 77.31) at 6 months after the start of therapy, and was 232.7 μg/g of stool (SD – 128.58) by the 12th month of follow-up (p > 0.05). Undesirable side effects were mild and did not require discontinuation of the drug.
Conclusion. For the first time in Russia, an open observational study was conducted as part of routine clinical practice to evaluate the effectiveness and safety of targeted therapy using elexacaftor/tezacaftor/ivacaftor and ivacaftor in children aged 2 – 6 years. Clinical and laboratory efficacy and safety have been demonstrated during 12 months of continuous follow-up.
Keywords
CFTR,
cystic fibrosis,
children,
sweat test,
targeted therapy,
CFTR modulators,
elexacaftor,
tezacaftor,
ivacaftor
Supporting agencies: There was no financial support for the study
About the Authors
N. A. Ilyenkova
Federal State Budgetary Educational Institution of Higher Education “Krasnoyarsk State Medical University named after Professor V.F.Voyno-Yasenetsky” of the Ministry of Health of the Russian Federation
Russian Federation
Competing Interests:
Russian Federation
Natalya A. Ilyenkova, Doctor of Medicine, Professor, Head of the Department
Department of Children’s Diseases with Postgraduate Physician Training Course
660022; ul. Partizana Zheleznyaka 1; Krasnoyarsk region
tel.: (391) 264-09-61
Competing Interests:
The authors declare no conflicts of interest
V. V. Chikunov
Federal State Budgetary Educational Institution of Higher Education “Krasnoyarsk State Medical University named after Professor V.F.Voyno-Yasenetsky” of the Ministry of Health of the Russian Federation
Russian Federation
Competing Interests:
Russian Federation
Vladimir V. Chikunov, Candidate of Medicine, Associate Professor
Department of Children’s Diseases with Postgraduate Physician Training Course
660022; ul. Partizana Zheleznyaka 1; Krasnoyarsk region
tel.: (391) 264-09-61
Competing Interests:
The authors declare no conflicts of interest
S. A. Shive
Federal State Budgetary Scientific Institution “Federal Research Center Krasnoyarsk Scientific Center of the Siberian Branch of the Russian Academy of Sciences” –
separate division of the Research Institute for Medical Problems of the North, Ministry of Science and Higher Education of the Russian Federation
Russian Federation
Competing Interests:
Russian Federation
Svetlana A. Shive, Pulmonologist
660022; ul. Partizana Zheleznyaka 3g; Krasnoyarsk
tel.: (391) 256-81-35
Competing Interests:
Конфликт интересов авторами не заявлен
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For citations:
Ilyenkova N.A., Chikunov V.V., Shive S.A. Using elexacaftor/tezacaftor/ivacaftor + ivacaftor in preschool children with cystic fibrosis. PULMONOLOGIYA. 2025;35(2):213-220. (In Russ.) https://doi.org/10.18093/0869-0189-2025-35-2-213-220
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