Body composition changes in children with cystic fibrosis while receiving targeted therapy: A pilot study

Introduction. The primary treatment for cystic fibrosis is conservative, primarily mucolytic and antibacterial. Dietary therapy plays a significant role, targeting mechanisms such as pancreatic enzyme replacement therapy and enteral caloric supplements that improve nutritional status.

Aim. To study the effect of targeted therapy on changes in the body composition of children with cystic fibrosis.

Materials and methods. A single-center retrospective study examined 67 bioimpedancemetry protocols (Diamant AIST mini) in 31 patients aged 4 to 17 years with cystic fibrosis receiving nutritional support before and after targeted therapy. There were 15 boys (48%) and 16 girls (52%). As part of the observational study, the patients were divided into 2 groups: 11 (35%) received targeted therapy for 1 year, and 20 children (65%) had their nutritional status assessed before targeted therapy. All children received enzyme replacement therapy and nutritional support with enteral nutrition mixtures recommended for patients with cystic fibrosis. Body composition assessments were performed annually during planned hospitalization.

Results. No differences in the Z-score indicators of height, body weight and body mass index were found in the groups. The body composition varied. When using targeted therapy, statistically significant changes in fat mass, its proportion, and deviations in the proportion of dry cell mass were revealed (p = 0.046, p = 0.020, p = 0.021, respectively). With targeted therapy, patients demonstrated an increase in body fat mass and its proportion, along with an increase in the proportion of skeletal cell mass. In Group 2 without targeted therapy, fat mass, its proportion, and skeletal cell mass were below age-appropriate norms, despite adequate replacement therapy and nutritional support.

Conclusions. Targeted therapy improved disease control but altered body composition in patients receiving it. Nutritional support remains an essential treatment for patients with cystic fibrosis.

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