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Growth hormone may be viable treatment for adults with Prader-Willi syndrome
Sode-Carlsen R. J Clin Endocrinol Metab. 2010;95:4943-4950.
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One year of treatment with growth hormone led to significant increases in lean body mass and reductions in visceral and subcutaneous abdominal fat volume in adults with Prader-Willi syndrome.
In a study sponsored by Novo Nordisk, researchers randomly assigned 46 patients to receive placebo or, depending on the patient’s weight, 0.3 mg or 0.4 mg of GH (Norditropin SimpleXx, Novo Nordisk) per day for the first 4 weeks of the study. They then increased doses to 0.6 mg or 0.8 mg daily for 11 months. Forty patients completed the study, with six departing for various reasons.
Compared with the placebo arm, the GH group experienced a 22.9-mL reduction in visceral abdominal fat (–38.1 mL to –7.8 mL; P=.004) and a 70.7-mL reduction in subcutaneous abdominal fat (–115 mL to –26.3 mL; P=.003). Similarly, thigh adipose tissue also decreased by 21.3 mL (–38 mL to –4.7 mL; P=.013) and thigh muscle increased by 6.1 mL (1.9 mL -10.2 mL; P=.005) among those receiving GH.
Results also indicated, however, that, while visceral abdominal fat declined in men (P=.04), levels remained unchanged in women (P=.15).
A 2.25-kg growth in lean body mass was also observed in the GH arm (0.725 kg-3.77 kg; P=.005), according to the researchers, along with a 4.2-kg reduction in fat mass (–6.4 kg to –2 kg; P<.001) compared with the placebo group.
Patients receiving GH also experienced a 125-mcg/L increase in insulin-like growth factor I levels (84 mcg/L-167mcg/L; P<.001) as well as a 0.27-mmol/L decrease in LDL.
The researchers noted no major adverse events in the GH treatment arm, and they observed no changes in physical performance after study completion.
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