Čes-slov Pediat 2024, 79(5):293-298 | DOI: 10.55095/CSPediatrie2024/026

Musculoskeletal health in children with cystic fibrosisComprehensive Report

Kvido Malaska, Ondřej Souček

Pediatrická klinika, 2. lékařská fakulta, Univerzita Karlova a FN v Motole, Praha

Cystic fibrosis (CF) is a genetically determined disease that primarily affects lungs and pancreas, but also a number of other organ systems, including bones and skeletal muscles. Osteoporosis, i.e. reduced bone mineral density, together with an increased incidence of fractures, occurs in up to 28 % of individuals with CF and has a complex etiology. Osteoporosis is more common in older individuals, individuals with malnutrition, more severe clinical manifestations and impaired lung functions. Some of these factors also cause reduced muscle strength of respiratory and skeletal muscles and insufficient serum concentrations of 25-hydroxyvitamin D (25-OHD), an indicator of vitamin D reserves in the body and one of the main regulators of bone metabolism. Substitution with cholecalciferol increases the concentration of 25-OHD only in some individuals with CF. Modern treatment with CFTR (cystic fibrosis transmembrane conductance regulator) modulators leads to improvement of lung functions and nutritional status and, according to several recent pilot studies, probably has a positive effect on bone density and serum 25-OHD concentrations. New randomized controlled trials will verify the effect of vitamin D substitution on bone density and 25-OHD concentrations and clarify the effect of CFTR modulators on musculoskeletal complications.

Keywords: cystic fibrosis, bone mineral density, 25-hydroxyvitamin D, muscle strength, CFTR modulators, children

Received: April 15, 2024; Revised: April 15, 2024; Accepted: April 16, 2024; Published: November 1, 2024  Show citation