Clinical Endocrinology

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

Authors


D. A. M. Festen, Dutch Growth and Research Foundation, Westzeedijk 106, 3016 AH Rotterdam, the Netherlands. Tel.: +31 10 2251533; Fax: +31 10 2250133; E-mail: d.festen@erasmusmc.nl, d.festen@kindengroei.nl

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.

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