One year of growth hormone treatment in adults with Prader-Willi syndrome improves body composition: results from a randomized, placebo-controlled study.
BACKGROUND
Prader-Willi syndrome (PWS) is a multisymptomatic disease that shares many similarities with the GH deficiency syndrome, including altered body composition with more body fat than lean body mass.


OBJECTIVE
Our objective was to investigate the effect of GH on body composition in adults with PWS.


METHODS
Forty-six adults with PWS were randomized to GH or placebo treatment for 12 months in a double-blind trial.


METHODS
We evaluated change in regional body composition of the abdomen and thigh as measured by computed tomography and change in total body composition as measured by dual-energy x-ray absorptiometry.


RESULTS
Forty patients completed the study. Baseline median IGF-I sd score was -0.4. GH treatment increased IGF-I by 125 μg/liter (1.51 sd score), and based upon computed tomography, body composition improved with a decrease in visceral fat mass of 22.9 ml (P = 0.004), abdominal sc fat mass 70.9 ml (P = 0.003), and thigh fat mass 21.3 ml (P = 0.013), whereas thigh muscle mass increased 6.0 ml (P = 0.005). By dual-energy x-ray absorptiometry, lean body mass improved 2.25 kg (P = 0.005), and total fat mass decreased 4.20 kg (P < 0.001). No major side effects were seen.


CONCLUSIONS
Unrelated to the GH-IGF-I levels at baseline, our results showed that long-term treatment with GH effectively improved body composition and represents a safe, potential treatment option, relieving some of the negative consequences of PWS.
Sode-Carlsen
Rasmus
R
Centre for Rare Diseases, Department of Pediatrics, Aarhus University Hospital Skejby, Aarhus N, Denmark. Rasmus.Sode-Carlsen@ki.au.dk
Farholt
Stense
S
Rabben
Kai Fr
KF
Bollerslev
Jens
J
Schreiner
Thomas
T
Jurik
Anne Grethe
AG
Christiansen
Jens Sandahl
JS
Höybye
Charlotte
C
D050153:Abdominal Fat / Q000000981:diagnostic imaging; D015502:Absorptiometry, Photon; D000328:Adult; D000704:Analysis of Variance; D001823:Body Composition; D004311:Double-Blind Method; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007328:Insulin / Q000097:blood; D007334:Insulin-Like Growth Factor I / Q000378:metabolism; D011218:Prader-Willi Syndrome / Q000097:blood / Q000000981:diagnostic imaging / Q000628:therapy*; D011863:Radioimmunoassay; D016896:Treatment Outcome
D007328:Insulin; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I