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Growth, Body Composition, and Strength of Children with Cystic Fibrosis
Treated with Elexacaftor/Tezacaftor/Ivacaftor (ETI)
Abstract
Many children treated with cystic fibrosis transmembrane conductance
regulator (CFTR) modulators increase their body mass index (BMI). To
extend this observation, we prospectively monitored rates of change for
growth, body mass and composition indices and z scores, forearm
strength, and FEV1 during ETI therapy for a cohort of 27 children, ages
6-11, assessed potential risk factors for concerning change rates; and
documented variability of individual change rates. Body composition was
assessed by bioelectrical impedance analysis (BIA). Z score calculations
used matched data from US children. Outcomes rates of change were
analyzed using a linear mixed effects regression model. At enrollment,
median BMI z-score was 0.6, percent body fat was 22.7, and percent
predicted (pp) FEV1 was 100. During ETI treatment, mean z scores for
annualized change rates of BMI (0.02±0.07), fat mass index (FMI)
(0.02±0.08), and fat free mass index (-0.03±0.07) were not different
from zero. Percent increase of FMI (mean:12.0±29.0) often exceeded that
for skeletal muscle mass index (SMMI)(4.7±4.9). Older children and girls
experienced the largest weight gain. Individual body mass and
composition index rates of change varied extensively. FEV1 increase was
independent of body composition and muscle strength change rates.
Preadolescent children, as a group, experienced growth and body
composition trajectories expected of US children, adding muscle mass,
but preferentially adding fat mass when treated with ETI. Individual
outcomes variation suggests potential benefit for body composition
monitoring and interventions to promote healthy physical maturation
during ETI therapy.