Growth hormone treatment completely normalizes adult height and improves body composition in Prader-Willi syndrome: experience from KIGS (Pfizer International Growth Database).
BACKGROUND
Abnormal body composition, with low muscle mass and increased fat mass, as well as short adult stature are common features in Prader-Willi syndrome (PWS), as in growth hormone (GH) deficiency.


METHODS
We followed a cohort of 22 genetically verified patients with PWS from the start of GH (Genotropin) treatment at the median age of 6.9 years (4.9-11.3) to near-adult height at 18.1 years (16.4-21.2). The patients were treated with a median GH dose of 0.03 mg/kg/day (0.02-0.03) for a median duration of 10.2 years (6.9-11.5).


RESULTS
All patients reached near-adult height within midparental height median -0.5 SDS (-1.4 to 0.7) and 0.9 SDS (0.1-1.9) for girls and boys, respectively. The body composition improved but did not normalize. Only 7 of the 22 patients were reported to be in puberty. None of the patients were reported to be on sex hormone substitution which might contribute to not reaching a normal body composition. No serious side effects were reported when the caloric intake was controlled to maintain an appropriate body weight.


CONCLUSIONS
GH treatment in children with Prader-Wili syndrome normalizes adult height and improves body composition.
Lindgren
A C
AC
Paediatric Endocrinology Unit, Department of Woman and Child Health, Karolinska University Hospital and Stockholm Centre of Eating Disorders, Stockholm, Sweden. ann.christin.lindgren@ki.se
Lindberg
A
A
D015502:Absorptiometry, Photon; D001823:Body Composition / Q000187:drug effects*; D001827:Body Height / Q000187:drug effects*; D002648:Child; D002675:Child, Preschool; D016208:Databases, Factual; D005260:Female; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D011218:Prader-Willi Syndrome / Q000188:drug therapy* / Q000503:physiopathology
D019382:Human Growth Hormone