Metabolic health profile in young adults with Prader-Willi syndrome: results of a 2-year randomized, placebo-controlled, crossover GH trial.
BACKGROUND
Patients with Prader-Willi syndrome (PWS) have an increased fat mass and decreased lean body mass. GH-treated young adults with PWS who have attained adult height benefit from continuation of growth hormone (GH) treatment, as GH maintained their improved body composition, whereas fat mass increased during the placebo period. Adults with PWS are predisposed to T2DM and cardiovascular disease. Whether GH affects metabolic health profile of this patient group is unknown.


OBJECTIVE
To investigate the effects of GH vs placebo on metabolic health, in young adults with PWS who were GH-treated for many years during childhood and had attained adult height (AH).


METHODS
A 2-year, randomized, double-blind, placebo-controlled crossover study with stratification for gender and BMI in 27 young adults with PWS. Intervention with GH (0·67 mg/m2/day) and placebo, both for 1-year duration.


RESULTS
Compared to placebo, GH treatment resulted in similar glucose and insulin levels during oral glucose tolerance test. Only fasting glucose and insulin were slightly higher during GH vs placebo (+0·2 mmol/l and +18·4 pmol/l), although both remained within normal ranges in both phases. Blood pressure and lipid profile were similar after GH vs placebo. At baseline (AH) and during GH, no patients had metabolic syndrome, while 1 developed it during placebo treatment.


CONCLUSIONS
Growth hormone treatment has no adverse effects on metabolic health profile. Thus, GH-treated young adults with PWS who have attained AH benefit from continuation of GH treatment without safety concerns regarding metabolic health.
Dutch Growth Research Foundation, Rotterdam, The Netherlands.|Department of Pediatrics, Subdivision of Endocrinology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
R J
RJ
Kuppens
Dutch Growth Research Foundation, Rotterdam, The Netherlands.|Department of Pediatrics, Subdivision of Endocrinology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
N E
NE
Bakker
Dutch Growth Research Foundation, Rotterdam, The Netherlands.|Department of Pediatrics, Subdivision of Endocrinology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
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EP
Siemensma
Dutch Growth Research Foundation, Rotterdam, The Netherlands.|Department of Pediatrics, Subdivision of Endocrinology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
S H
SH
Donze
Department Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands.
T
T
Stijnen
Dutch Growth Research Foundation, Rotterdam, The Netherlands.|Department of Pediatrics, Subdivision of Endocrinology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.
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Hokken-Koelega
D000293:Adolescent; D001786:Blood Glucose / Q000032:analysis; D018592:Cross-Over Studies; D005260:Female; D005951:Glucose Tolerance Test; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007328:Insulin / Q000097:blood; D008297:Male; D011218:Prader-Willi Syndrome / Q000188:drug therapy* / Q000503:physiopathology; D055815:Young Adult
D001786:Blood Glucose; D007328:Insulin; D019382:Human Growth Hormone