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


METHODS
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/m(2)/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/m(2)/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.
Festen
Dederieke A M
DA
Dutch Growth and Research Foundation, Rotterdam, the Netherlands. d.festen@erasmusmc.nl
de Lind van Wijngaarden
Roderick
R
van Eekelen
Marielle
M
Otten
Barto J
BJ
Wit
Jan M
JM
Duivenvoorden
Hugo J
HJ
Hokken-Koelega
Anita C S
AC
D000293:Adolescent; D000465:Algorithms; D000886:Anthropometry; D001823:Body Composition / Q000187:drug effects*; D001827:Body Height / Q000502:physiology; D015992:Body Mass Index; D002648:Child; D002675:Child, Preschool; D005260:Female; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D018972:Insulin-Like Growth Factor Binding Protein 3; D018969:Insulin-Like Growth Factor Binding Proteins / Q000097:blood; D007334:Insulin-Like Growth Factor I / Q000032:analysis; D008297:Male; D011218:Prader-Willi Syndrome / Q000188:drug therapy* / Q000378:metabolism / Q000503:physiopathology
C515497:IGFBP3 protein, human; D018972:Insulin-Like Growth Factor Binding Protein 3; D018969:Insulin-Like Growth Factor Binding Proteins; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I