Research Article

Two years of growth hormone therapy in young children with Prader–Willi syndrome: Physical and neurodevelopmental benefits†

  1. Susan E. Myers1,*,
  2. Barbara Y. Whitman1,
  3. Aaron L. Carrel2,
  4. Victoria Moerchen3,
  5. M. Tracy Bekx2,
  6. David B. Allen2

Article first published online: 13 NOV 2006

DOI: 10.1002/ajmg.a.31468

Issue

American Journal of Medical Genetics Part A

American Journal of Medical Genetics Part A

Volume 143A, Issue 5, pages 443–448, 1 March 2007

Additional Information

How to Cite

Myers, S. E., Whitman, B. Y., Carrel, A. L., Moerchen, V., Bekx, M. T. and Allen, D. B. (2007), Two years of growth hormone therapy in young children with Prader–Willi syndrome: Physical and neurodevelopmental benefits. American Journal of Medical Genetics Part A, 143A: 443–448. doi: 10.1002/ajmg.a.31468

Author Information

  1. 1

    Department of Pediatrics, Saint Louis University, St. Louis, Missouri

  2. 2

    Department of Pediatrics, University of Wisconsin, Madison, Wisconsin

  3. 3

    Department of Physical Therapy and Rehabilitative Science, University of Maryland School of Medicine, Baltimore, Maryland

*Division of Endocrinology, Department of Pediatrics, Saint Louis University, Cardinal Glennon Children's Hospital, 1465 South Grand Boulevard, St. Louis, Missouri 63104.

  1. †

    How to cite this article: Myers SE, Whitman BY, Carrel AL, Moerchen V, Bekx MT, Allen DB. 2006. Two years of growth hormone therapy in young children with Prader–Willi syndrome: Physical and neurodevelopmental benefits. Am J Med Genet Part A 143A:443–448.

Publication History

  1. Issue published online: 22 FEB 2007
  2. Article first published online: 13 NOV 2006
  3. Manuscript Accepted: 27 JUL 2006
  4. Manuscript Received: 16 FEB 2006

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Keywords:

  • Prader–Willi syndrome;
  • growth hormone therapy;
  • body composition;
  • psychomotor development;
  • infants and toddlers

Abstract

Infants with Prader–Willi syndrome (PWS) typically display failure to thrive and decreased muscle mass with excess body fat for age. Growth hormone (GH) therapy in children with PWS improves, but does not normalize, body composition and muscle strength and agility. The objective of this study was to determine the effects of earlier GH therapy on anthropometric measurements, body composition, and psychomotor development in affected PWS infants and toddlers. Twenty-five subjects, ages 4–37 months, were randomized to 2 years of GH therapy (1 mg/m2/day) or 1 year of observation without GH treatment and then placed on GH (1.5 mg/m2·day) for 1 year only. Anthropometric measurements were obtained by standard methods: percent body fat, lean body mass, and total body bone mineral density by dual x-ray absorptiometry; motor constructs of mobility and stability by the Toddler Infant Motor Evaluation; and cognitive and language function by the Capute Scales of Infant Language and Cognitive Development. GH-treated PWS subjects demonstrated normalization of length/height standard deviation scores (SDS), faster head growth, increased lean body mass accrual, and decreased percent body fat (P < 0.005 for all parameters), as well as improved language (P = 0.05) and cognitive (P = 0.02) quotient Z-scores compared with similarly aged untreated PWS subjects after 1 year into the study. PWS subjects treated before their first birthday spoke their first words at a mean age of 14.4 ± 2.8 months and walked independently at 23.3 ± 4.8 months. GH therapy was well-tolerated; however, one PWS subject experienced scoliosis progression. As greater benefits were seen in our study with early treatment, prompt referral to a pediatric endocrinologist for consideration of GH therapy is recommended for PWS at an early age. © 2006 Wiley-Liss, Inc.

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