Effects on growth and metabolism of growth hormone treatment for 3 years in 36 children with Prader-Willi syndrome.
OBJECTIVE
Prader-Willi syndrome (PWS) is a complex genetic disorder whose many manifestations include obesity and short stature. Diabetes, osteoporosis, and scoliosis are common. We evaluated the effects of human growth hormone (hGH).


METHODS
A prospective cohort study of 36 children (1-15 years of age) with genetically confirmed PWS who were given hGH (mean dose 0.033 ± 0.006 mg/kg/day) for 36 months. At baseline and once yearly, we evaluated growth, insulin-like growth factor-1 (IGF-1), body composition, bone mineral density (BMD), glucose tolerance, serum lipids, and spinal radiographs.


RESULTS
Height gain over the 3-year period was 1.2 SD score. Lean body mass increased significantly during each treatment year. Total body fat decreased by 5.42 and 1.17% in the 1st and 2nd years, respectively. BMD remained unchanged during therapy. IGF-1 and homeostasis model assessment index of insulin resistance increased, and glucose intolerance was found in 22.7% of patients at baseline and 0% at 3 years. None of the patients had diabetes. Their lipid profile improved. Scoliosis was present in 27.8% of the patients at baseline and 47.2% at 3 years.


CONCLUSIONS
GH treatment in children with PWS has multiple beneficial effects on growth and body composition. Tolerance is good, with an improvement in glucose metabolism, although IGF-1 levels and insulin resistance parameters should be monitored closely. The high rate of scoliosis warrants monitoring by a pediatric orthopedic surgeon.
Colmenares
A
A
Pediatric Endocrinology and Diabetes Unit, Centre de référence pour le syndrome de Prader-Willi, Paris, France.
Pinto
G
G
Taupin
P
P
Giuseppe
A
A
Odent
T
T
Trivin
C
C
Laborde
K
K
Souberbielle
J C
JC
Polak
M
M
D001823:Body Composition / Q000187:drug effects; D015519:Bone Density / Q000187:drug effects; D050260:Carbohydrate Metabolism / Q000187:drug effects; D002648:Child; D015331:Cohort Studies; D005260:Female; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007333:Insulin Resistance / Q000502:physiology; D007334:Insulin-Like Growth Factor I / Q000378:metabolism; D050356:Lipid Metabolism / Q000187:drug effects; D008297:Male; D011218:Prader-Willi Syndrome / Q000188:drug therapy* / Q000378:metabolism*; D011446:Prospective Studies; D012600:Scoliosis / Q000209:etiology
D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I