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Growth hormone treatment in adults with Prader-Willi syndrome: the Scandinavian study

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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.

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Acknowledgments

The Scandinavian study on GH treatment in adults with PWS was supported by grants from Novo Nordisk Scandinavia AB, Malmö, Sweden and Novo Nordisk, Bagsværd, Denmark, The A.P. Møller Foundation for the Advancement of Medical Science, Research Initiative of Aarhus University Hospital, Aarhus University Hospital Skejby Research Foundation, Aase and Ejnar Danielsen Foundation, The Danish Prader-Willi Syndrome Association, The Augustinus Foundation.

Conflict of interest

Jens Sandahl Christiansen and Charlotte Höybye are members of the advisory board on the International Outcome study of NordiNet, NordiNet investigators and have received lecture fees from NovoNordisk.

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Authors and Affiliations

  1. Department of Paediatrics, Centre for Rare Diseases, Aarhus University Hospital Skejby, 8200, Aarhus N, Denmark

    Rasmus Sode-Carlsen & Stense Farholt

  2. Frambu, 1400, Siggerud, Norway

    Kai Fr. Rabben

  3. Department of Endocrinology, Oslo University Hospital, Rikshospitalet, University of Oslo, Oslo, Norway

    Jens Bollerslev & Thomas Schreiner

  4. Department of Radiology, Aarhus University Hospital, 8000, Aarhus C, Denmark

    Anne Grethe Jurik

  5. Department of Endocrinology M, Aarhus University Hospital, 8000, Aarhus C, Denmark

    Jens Sandahl Christiansen

  6. Department of Endocrinology, Metabolism and Diabetology, Karolinska University Hospital, 17176, Stockholm, Sweden

    Charlotte Höybye

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  1. Rasmus Sode-Carlsen
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  2. Stense Farholt
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Sode-Carlsen, R., Farholt, S., Rabben, K.F. et al. Growth hormone treatment in adults with Prader-Willi syndrome: the Scandinavian study. Endocrine 41, 191–199 (2012). https://doi.org/10.1007/s12020-011-9560-4

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