ECE2010 Poster Presentations Growth factors (15 abstracts)
Two years of GH therapy improves body composition in adults with Prader–Willi Syndrome
Rasmus Sode-Carlsen 1 , Stense Farholt 1 , Kai Fr. Rabben 2 , Jens Bollerslev 3 , Thomas Schreiner 3 , Jens Sandahl Christiansen 4 & Charlotte Höybye 5
1Department of Paediatrics, Aarhus University Hospital Skejby, Aarhus, Denmark; 2Frambu, Siggerud, Norway; 3Department of Endocrinology, Rikshospitalet, Oslo, Norway; 4Department of Endocrinology M, Aarhus University Hospital, Aarhus, Denmark; 5Department of Endocrinology, Metabolism and Diabetology, Karolinska University Hospital, Stockholm, Sweden.
Introduction: Prader–Willi syndrome (PWS) presents clinically with a multitude of findings, including abnormal body composition and partial GH deficiency. Until now three studies have reported beneficial effects upon body composition of GH treatment in adults with PWS. However, only one of these studies had the optimal randomised controlled design.
Aim: The aim of this study was to confirm and substantiate the results from previous studies.
Patients and methods: Forty-six patients, 25 women, 21 men, age 29 years (16–41) (median and range) with genetically verified PWS participated in a multinational Scandinavian study. The patients were randomised to treatment with GH (0.6–0.8 mg daily) (Norditropin SimpleXx) or placebo for 12 months, the following 12 months all patients were treated with GH according to their IGF1 value. Body composition was measured yearly by dual X-ray absorptiometry. The study was approved by the local ethical committees.
Results: Body fat changed −2.05 vs +2.60 kg (P<0.001) and lean body mass +2.39 vs −0.00 kg (P=0.006) the first year. During the second year non significant changes was found in body fat +0.70 kg (P=0.37) and lean body mass 1.10 kg (P=0.15) in the group primarily randomised to GH, whereas the change in body fat was −4.51 kg (P<0.001) and lean body mass +2.15 kg (P<0.001) in the group primarily randomised to placebo.
Conclusion: In this first large scale, long-term placebo-controlled study the improvement in body composition by GH treatment in adults with PWS was confirmed. No side effects were observed. Based on our 2 years results, findings persist during long-term therapy.
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Endocrine Abstracts
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