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not-yet-known not-yet-known not-yet-known unknown Growth, Body Composition, and Strength of Children with Cystic Fibrosis Treated with Elexacaftor/Tezacaftor/Ivacaftor (ETI)
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  • Tom Boat,
  • Md Hossain,
  • Aisaku Nakamura,
  • Michelle Hjelm,
  • William Hardie,
  • Matthew Wackler,
  • Alyssa Amato,
  • Carolyn Dress
Tom Boat
Cincinnati Children's Hospital Medical Center

Corresponding Author:[email protected]

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Md Hossain
Cincinnati Children's Hospital Medical Center
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Aisaku Nakamura
Cincinnati Children's Hospital Medical Center
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Michelle Hjelm
Cincinnati Children's Hospital Medical Center
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William Hardie
Cincinnati Children's Hospital Medical Center
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Matthew Wackler
Cincinnati Children's Hospital Medical Center
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Alyssa Amato
Cincinnati Children's Hospital Medical Center
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Carolyn Dress
Corewell Health Helen DeVos Children's Hospital
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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.
06 Aug 2024Submitted to Pediatric Pulmonology
15 Aug 2024Submission Checks Completed
15 Aug 2024Assigned to Editor
15 Aug 2024Review(s) Completed, Editorial Evaluation Pending
18 Aug 2024Reviewer(s) Assigned
15 Sep 2024Editorial Decision: Revise Major