title: Growth hormone treatment in adults with Prader-Willi syndrome: the Scandinavian study.
abstract: Prader-Willi syndrome (PWS) is characterized by short stature, muscular hypotonia, cognitive dysfunction, and hyperphagia usually leading to severe obesity. Patients with PWS share similarities with growth hormone deficiency (GHD). Few studies have dealt with growth hormone (GH) treatment in PWS adults. The purpose of the Scandinavian study was to evaluate the effects of GH on body composition, lipid and glucose metabolism, physical performance and safety parameters in adults with PWS. Twenty-five women and 21 men with PWS were randomized to treatment with GH or placebo during 1 year followed by 2 years of open labeled GH treatment. At baseline 1/3 had normal BMI, six patients severe GHD, ten impaired glucose tolerance and seven diabetes. At 1 year insulin-like growth factor I (IGF-I) SDS had increased by 1.51 (P < 0.001) and body composition improved in the GH treated group. Visceral fat decreased by 22.9 ml (P = 0.004), abdominal subcutaneous fat by 70.9 ml (P = 0.003) and thigh fat by 21.3 ml (P = 0.013), whereas thigh muscle increased 6.0 ml (P = 0.005). Lean body mass increased 2.25 kg (P = 0.005), and total fat mass decreased 4.20 kg (P < 0.001). The positive effects on body composition were maintained after 2 years of GH treatment. Peak expiratory flow increased by 12% (P < 0.001) at 2 years of GH treatment. Lipid and glucose metabolism were unchanged, however, three patients developed diabetes at 2 years of GH treatment. In conclusion GH treatment had beneficial effects on the abnormal body composition without serious adverse events making it a logic treatment option in adults with PWS.
authors.affiliation: Department of Paediatrics, Centre for Rare Diseases, Aarhus University Hospital Skejby, 8200 Aarhus N, Denmark.
authors.forename: Rasmus
authors.initials: R
authors.lastname: Sode-Carlsen
authors.forename: Stense
authors.initials: S
authors.lastname: Farholt
authors.forename: Kai Fr
authors.initials: KF
authors.lastname: Rabben
authors.forename: Jens
authors.initials: J
authors.lastname: Bollerslev
authors.forename: Thomas
authors.initials: T
authors.lastname: Schreiner
authors.forename: Anne Grethe
authors.initials: AG
authors.lastname: Jurik
authors.forename: Jens Sandahl
authors.initials: JS
authors.lastname: Christiansen
authors.forename: Charlotte
authors.initials: C
authors.lastname: Höybye
mesh_terms: D000328:Adult; D001823:Body Composition / Q000187:drug effects; D015331:Cohort Studies; D048909:Diabetes Complications / Q000097:blood / Q000188:drug therapy / Q000378:metabolism / Q000503:physiopathology; D004311:Double-Blind Method; D005260:Female; D005500:Follow-Up Studies; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007333:Insulin Resistance; D007334:Insulin-Like Growth Factor I / Q000032:analysis; D008297:Male; D008875:Middle Aged; D010366:Peak Expiratory Flow Rate / Q000187:drug effects; D011218:Prader-Willi Syndrome / Q000097:blood / Q000188:drug therapy* / Q000378:metabolism / Q000503:physiopathology; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use; D012537:Scandinavian and Nordic Countries; D012720:Severity of Illness Index; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I
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