JOURNAL ARTICLE
RANDOMIZED CONTROLLED TRIAL
RESEARCH SUPPORT, NON-U.S. GOV'T
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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.

CONCLUSION: GH treatment in children with Prader-Wili syndrome normalizes adult height and improves body composition.

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