Randomized controlled GH trial: effects on anthropometry, body composition and body proportions in a large group of children with Prader-Willi syndrome

Summary Background Prader–Willi syndrome (PWS) children have impaired growth, and abnormal body composition. Previous 1‐year controlled studies showed improvement of height and body composition during GH‐treatment. Objective To evaluate growth, body composition and body proportions during GH‐treatment in a large group of PWS children. Design/patients We performed a randomized controlled GH trial in 91 prepubertal PWS children (42 infants, 49 children, aged 3–14 years). After stratification for age, infants were randomized to GH‐treatment (GH‐group; 1 mg/m2/day; n = 20), or no treatment (control group; n = 22) for 1 year. In the second year all infants were treated with GH. After stratification for BMI, children > 3 years of age were randomized to GH‐treatment (GH‐group; 1 mg/m2/day; n = 27) or no treatment (control group; n = 22) for 2 years. Anthropometric parameters were assessed once in every 3 months. Body composition was measured by Dual Energy X‐ray Absorptiometry. Results Median (interquartile range, iqr) height SDS increased during 2 years of GH in infants from –2·3 (–2·8 to –0·7) to –0·4 (–1·1–0·0) and in prepubertal children from –2·0 (–3·1 to –1·7) to –0·6 (–1·1 to –0·1). In non‐GH‐treated children height SDS did not increase. Head circumference completely normalized during 1 and 2 years of GH in infants and children, respectively. Body fat percentage and body proportions improved in GH‐treated children, but did not completely normalize. Lean body mass SDS improved compared to the control group. Serum IGF‐I increased to levels above the normal range in most GH‐treated children. Conclusions Our randomized study shows that GH‐treatment in PWS children significantly improves height, BMI, head circumference, body composition and body proportions. PWS children are highly sensitive to GH, suggesting that monitoring of serum IGF‐I is indicated.