(P11-054-25) Prescribed Growth Hormone’s Effect on Body Composition in Adults With Prader-Willi Syndrome (PWS): A Longitudinal Observational Study Protocol

Objectives: This observational study aims to assess the effect of prescribed growth hormone on body composition in adult patients with Prader-Willi syndrome (PWS). The primary outcome is lean body mass. Secondary outcomes include fat mass, resting energy expenditure, quality of life, and hand grip strength. An exploratory aim will examine the relationship between prescribed growth hormone and brain atrophy.

Methods: This is a longitudinal study, recruiting 40 adults (n = 20 per group) ≥ 18 years of age diagnosed with PWS who are undergoing growth hormone therapy (GHT), and a group of those not utilizing growth hormone. Body composition will be assessed by dual-energy X-ray absorptiometry (DEXA) and bioimpedance spectroscopy (BIS) at baseline, 6 months, 1 year, and 2 years. At these same time points, resting energy expenditure will be assessed by indirect calorimetry, quality of life (SF-36), and hand grip strength by a hand-held dynamometer. Magnetic resonance imaging (MRI) will assess brain volume and focal damage at baseline and 2 years. Adverse events will be monitored. We will use an ANOVA to test for differences in the measured outcomes at baseline. Repeated measures ANOVA will be used to test whether the change in lean body mass and each secondary outcome (as appropriate for each outcome) over time differs significantly between the GHT and non-GHT groups, accounting for both time and between-subject effects. A Wilcoxon signed-rank test (if non-normally distributed) will be used to compare between-group change in brain volume from baseline to 2 years.

Results: We hypothesize that the group receiving GHT will exhibit higher lean body mass, hand grip strength, quality of life, and resting energy expenditure than the non-GHT group at baseline, and this difference will increase over the 2-year period. Additionally, we hypothesize that adults receiving GHT will exhibit less brain atrophy over the 2-year period than the non-GHT group.

Conclusions: The findings of this study will add to the body of literature exploring the efficacy of GHT in adults affected by PWS.

Funding Sources: UF Foundation PWS Research Fund