muscle function and physical activity in the modern era of cystic fibrosis

Conditions: Cystic Fibrosis

Summary: cystic fibrosis (cf) is characterized by various extrapulmonary manifestations, including altered skeletal muscle function, with both quantitative (e.g. reduced muscle mass) and qualitative (e.g. impaired oxidative function) impairments that may have a negative impact on exercise tolerance and quality of life. these abnormalities have traditionally been related to disease (e.g. systemic inflammation) or behavioral factors (e.g. increased physical inactivity). however, most of the studies that observed these abnormalities and tried to shed light on the underlying factors were either small or conducted before the widespread of cftr (cystic fibrosis transmembrane conductance regulator) modulators that have profound impact on the trajectory of the disease. while several studies suggested that the major recent improvements in therapeutics, including highly effective cftr modulators, may have positive effects on skeletal muscle function, either directly (e.g. improved mitochondrial function) or indirectly (e.g. reduction in physical inactivity), no studies to date have thoroughly investigated this issue in a representative sample of people with cf. the absence of recent data on muscle function and physical activity levels casts doubt on the relevance of recommendations on exercise training in this population that were published before the widespread use of highly effective cftr modulators. this study aims to compare muscle function, measured according to the latest recommendations of the european cystic fibrosis society (saynor et al., 2023), and physical activity of children and adults with cf under cftr modulators, compared to age- and sex-matched healthy individuals. we hypothesize that the strength, endurance, muscle power, and physical activity levels of individuals with cystic fibrosis, treated with cftr modulators, remain reduced compared to healthy individuals.


Eligibility criteria:
inclusion criteria: * criteria for inclusion of cf patients: * diagnosis of cystic fibrosis * treated with cftr modulator therapy * men and women (children or adults) aged 10 years or older * affiliated with or entitled to social security coverage * for people ≥ 18 years old: participant expressing its oral non-opposition * for minor child (\<18 years old): participant and its legal representative expressing their oral non-opposition * criteria for inclusion of healthy subjects: * men and women (children or adults) aged 10 years or older * affiliated with or entitled to social security coverage * for people ≥ 18 years old: participant expressing its oral non-opposition * for minor child (\<18 years old): participant and its legal representative expressing their oral non-opposition exclusion criteria: * exclusion criteria for cf patients: * lack of a stable clinical condition (defined here as the presence of a pulmonary exacerbation and/or a significant change in treatments in the three weeks preceding inclusion in the study). * medical contraindication to engage in moderate-intensity physical activity. * knee joint pain incompatible with the measurement of quadriceps strength. * pregnancy. * exclusion criteria for healthy subjects: * known chronic respiratory, cardiovascular, metabolic, renal, or neuromuscular diseases. * presence of benign illnesses and/or acute infections requiring a doctor's visit and occasional treatment in the three weeks preceding inclusion in the study. * medical contraindication to engage in moderate-intensity physical activity. * knee joint pain incompatible with the measurement of quadriceps strength. * pregnancy.

Keywords: cystic fibrosis cftr modulators physical activity muscle function