Long-term growth hormone therapy changes the natural history of body composition and motor function in children with prader-willi syndrome.
BACKGROUND
Children with Prader-Willi syndrome (PWS) have decreased muscle mass, hypotonia, and impaired linear growth. Recombinant human GH (hGH) treatment reportedly improves body composition and physical function in children with PWS, but these studies lack long-term control data. To assess the impact of hGH therapy begun early in life on the natural history of PWS, we compared height, body composition, and strength in similar-age children with PWS naïve to hGH with those treated with hGH for 6 yr.


OBJECTIVE
Forty-eight children with PWS were studied: 21 subjects (aged 6-9 yr) treated with hGH for 6 yr (beginning at 4-32 months, mean 13 +/- 6 months) were compared with 27 children of similar age (5-9 yr) prior to treatment with hGH. Percent body fat, lean body mass, carbohydrate/lipid metabolism, and motor strength were compared using analysis of covariance.


RESULTS
PWS children treated with hGH demonstrated lower body fat (mean, 36.1 +/- 2.1 vs. 44.6 +/- 1.8%, P < 0.01), greater height (131 +/- 2 vs. 114 +/- 2 cm; P < 0.001), greater motor strength [increased standing broad jump 22.9 +/- 2.1 vs. 14.6 +/- 1.9 in. (P < 0.001) and sit-ups 12.4 +/- 0.9 vs. 7.1 +/- 0.7 in 30 sec (P < 0.001)], increased high-density lipoprotein cholesterol (58.9 +/- 2.6 vs. 44.9 +/- 2.3 mg/dl, P < 0.001), decreased low-density lipoprotein (100 +/- 8 vs. 131 +/- 7 mg/dl, P < 0.01), and no difference in fasting glucose or insulin.


CONCLUSIONS
hGH treatment in children with PWS, begun prior to 2 yr of age, improves body composition, motor function, height, and lipid profiles. The magnitude of these effects suggests that long-term hGH therapy favorably alters the natural history of PWS to an extent that exceeds risks and justifies consideration for initiation during infancy.
Carrel
Aaron L
AL
Department of Pediatrics, University of Wisconsin, American Family Children's Hospital, 600 Highland Avenue H4-436, Madison, Wisconsin 53792, USA. alcarrel@wisc.edu
Myers
Susan E
SE
Whitman
Barbara Y
BY
Eickhoff
Jens
J
Allen
David B
DB
D015502:Absorptiometry, Photon; D000704:Analysis of Variance; D000886:Anthropometry; D001823:Body Composition / Q000187:drug effects*; D050260:Carbohydrate Metabolism / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D005260:Female; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000378:metabolism; D050356:Lipid Metabolism / Q000187:drug effects; D008055:Lipids / Q000097:blood; D008297:Male; D053580:Muscle Strength / Q000187:drug effects*; D011218:Prader-Willi Syndrome / Q000097:blood / Q000188:drug therapy*; D011446:Prospective Studies; D011994:Recombinant Proteins / Q000627:therapeutic use; D016896:Treatment Outcome
D008055:Lipids; D011994:Recombinant Proteins; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I