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

Harriette R. Mogul, Phillip D.K. Lee, Barbara Y. Whitman, William B. Zipf, Michael Frey, Susan Myers, Mindy Cahan, Belinda Pinyerd, A. Louis Southren

Research output: Contribution to journalArticle

59 Scopus citations

Abstract

Context: GH replacement in Prader-Willi syndrome (PWS) children has well-defined benefits and risks and is used extensively worldwide. Its use in PWS adults has been limited by documentation of benefits and risks, as determined by larger multisite studies. Objectives: Our objective was to evaluate the effectiveness and safety of GH in GH-deficient genotype-positive PWS adults. Design: We conducted a 12-month open-label multicenter trial with 6-month dose-optimization and 6-month stable treatment periods. Setting: The study was conducted at outpatient treatment facilities at four U.S. academic medical centers. Patients: Lean and obese PWS adults with diverse cognitive skills, behavioral traits, and living arrangements were recruited from clinical populations. Intervention: Human recombinant GH (Genotropin) was initiated at 0.2 mg/d with monthly 0.2-mg increments to a maximum 1.0 mg/d, as tolerated. Main Outcomes Measures: Lean body mass and percent fat were measured by dual-energy x-ray absorptiometry. Results: Lean body mass increased from 42.65 ± 2.25 (SE) to 45.47 ± 2.31kg(P ≤ 0.0001), and percent fat decreased from 42.84 ± 1.12 to 39.95 ± 1.34% (P = 0.025) at a median final dose of 0.6 mg/d in 30 study subjects who completed 6-12 months of GH. Mean fasting glucose of 85.3 ± 3.4 mg/dl, hemoglobin A1c of 5.5 ± 0.2%, fasting insulin of 5.3 ± 0.6 μU/ml, area under the curve for insulin of 60.4 ± 7.5 μU/ml, and homeostasis model assessment of insulin resistance of 1.1 ± 0.2 were normal at baseline in 38 study initiators, including five diabetics, and remained in normal range. Total T3 increased 26.7% from 127.0 ± 7.8 to 150.5 ± 7.8 ng/dl (P = 0.021) with normalization in all subjects, including six (20%) with baseline T3 values at least 2 SD below the mean. Mildly progressive ankle edema was the most serious treatment-emergent adverse event (five patients). Conclusions: This multicenter study demonstrates that GH improves body composition, normalizes T3, and is well tolerated without glucose impairment in PWS genotype adults.

Original languageEnglish (US)
Pages (from-to)1238-1245
Number of pages8
JournalJournal of Clinical Endocrinology and Metabolism
Volume93
Issue number4
DOIs
StatePublished - Apr 2008
Externally publishedYes

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Endocrinology
  • Clinical Biochemistry
  • Biochemistry, medical

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