Harriette R. Mogul, Phillip D. K. Lee, Barbara Y. Whitman, William
B. Zipf, Michael Frey, Susan Myers, Mindy Cahan, Belinda Pinyerd, and A. Louis
Southren
Growth Hormone Treatment of Adults with Prader-Willi Syndrome and
Growth Hormone Deficiency Improves Lean Body Mass, Fractional Body Fat, and
Serum Triiodothyronine without Glucose Impairment: Results from the United
States Multicenter Trial
J. Clin. Endocrinol. Metab., Apr 2008; 93: 1238 -
1245.
The purpose of this study was to evaluate the effectiveness and
safety of growth hormone (GH) treatment in GH-deficient genotype-positive
Prader-Willi syndrome (PWS) adults. Prader-Willi syndrome is a congenital
disease that involves obesity, decreased muscle tone, decreased mental capacity,
and sex glands that produce little or no hormones. Although, GH replacement in
PWS children has well-defined benefits and risks and is used extensively
worldwide, the benefits and risks of its use in PWS adults is not as clear.
This 12-month multicenter trial was conducted at outpatient treatment
facilities at four U.S. academic medical centers. Thirty eight lean and obese
PWS adults were recruited from clinical populations. Human recombinant GH
treatment was initiated at a dose of 0.2 mg/d with monthly 0.2-mg increments to
a maximum 1.0 mg/d, as tolerated. The study measured lean body mass, percent
fat, fasting glucose, hemoglobin, fasting insulin, insulin resistance and serum
T3 uptake (thyroid function test).
Lean body mass increased and percent
fat decreased at a median final dose of 0.6 mg/d in 30 study subjects who
completed 6–12 months of GH. Mean fasting glucose, hemoglobin, fasting insulin,
and insulin resistance were normal at baseline in 38 study initiators, including
five diabetics, and remained in normal range. Total T3 increased 26.7% with
normalization in all subjects including six with baseline T3 values at least 2
SD below the mean. Mildly progressive ankle edema was the most serious treatment
related adverse event (five patients).
This study demonstrates that GH
improves body composition, normalizes T3, and is well tolerated without glucose
impairment in Prader-Willi syndrome genotype adults.
