title: Two years of growth hormone therapy in young children with Prader-Willi syndrome: physical and neurodevelopmental benefits.
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/m(2)/day) or 1 year of observation without GH treatment and then placed on GH (1.5 mg/m(2).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.
authors.affiliation: Department of Pediatrics, Saint Louis University, St. Louis, Missouri 63104, USA. myersse@slu.edu
authors.forename: Susan E
authors.initials: SE
authors.lastname: Myers
authors.forename: Barbara Y
authors.initials: BY
authors.lastname: Whitman
authors.forename: Aaron L
authors.initials: AL
authors.lastname: Carrel
authors.forename: Victoria
authors.initials: V
authors.lastname: Moerchen
authors.forename: M Tracy
authors.initials: MT
authors.lastname: Bekx
authors.forename: David B
authors.initials: DB
authors.lastname: Allen
mesh_terms: D001823:Body Composition / Q000187:drug effects; D001921:Brain / Q000187:drug effects* / Q000254:growth & development*; D002657:Child Development / Q000187:drug effects*; D002675:Child, Preschool; D003071:Cognition / Q000187:drug effects; D013006:Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D009043:Motor Activity / Q000187:drug effects*; D011218:Prader-Willi Syndrome / Q000188:drug therapy*; D013060:Speech / Q000187:drug effects
chemical_list: D013006:Growth Hormone