title: Limited efficacy of growth hormone (GH) during transition of GH-deficient patients from adolescence to adulthood: a phase III multicenter, double-blind, randomized two-year trial.
abstract: BACKGROUND
  Treatment of GH-deficient adolescents in transition to adulthood remains challenging.
  
  
  OBJECTIVE
  The objective was to assess the safety and efficacy of GH in GH-deficient adolescents in transition.
  
  
  METHODS
  Fifty-eight GH-deficient adolescents (mean age, 15.8 +/- 1.8 yr; 33 males) at near completion of their linear growth participated in the study.
  
  
  METHODS
  Baseline studies were done while subjects were on GH. Subjects were retested (insulin-induced hypoglycemia) 4 wk after GH discontinuation and reclassified as persistently GH-deficient or controls (n = 18). GH-deficient subjects were randomized to GH (n = 25, approximately 20 microg/kg.d) or placebo (n = 15).
  
  
  METHODS
  The multicenter study was conducted over a 2-yr period.
  
  
  RESULTS
  Changes in body composition, bone mineral density (BMD), quality of life (QOL), cardiovascular and metabolic markers were measured.
  
  
  RESULTS
  All groups had normal measures of lipid and carbohydrate metabolism, body composition, BMD, cardiac function, muscle strength, and QOL at baseline and after 2 yr. IGF-I concentrations decreased in all, but less so in the GH-group (P = 0.013). There was a greater increase in lean body mass (lesser adiposity) in the GH group than placebo at 12 months, but not at 24 months.
  
  
  CONCLUSIONS
  1) GH-deficient patients properly treated in childhood can have normal BMD, body composition, cardiac function, muscle strength, carbohydrate and lipid metabolism, and QOL when reaching adult height; and 2) continuation of GH therapy for 2 yr did not change these measures as compared to placebo-treated or control subjects. GH-deficient adolescents in good metabolic status at the time of epiphyseal fusion may safely discontinue GH for at least 2 yr. Follow-up is needed to determine whether GH therapy is eventually warranted in subjects treated with GH during childhood.
authors.affiliation: Division of Endocrinology, Nemours Children's Clinic, 807 Children's Way, Jacksonville, Florida 32207, USA. nmauras@nemours.org
authors.forename: Nelly
authors.initials: N
authors.lastname: Mauras
authors.forename: Ora Hirsch
authors.initials: OH
authors.lastname: Pescovitz
authors.forename: Vivek
authors.initials: V
authors.lastname: Allada
authors.forename: Michael
authors.initials: M
authors.lastname: Messig
authors.forename: Michael P
authors.initials: MP
authors.lastname: Wajnrajch
authors.forename: Barbara
authors.initials: B
authors.lastname: Lippe
mesh_terms: D000293:Adolescent; D001823:Body Composition; D015519:Bone Density; D050260:Carbohydrate Metabolism; D004311:Double-Blind Method; D015150:Echocardiography, Doppler; D005080:Exercise Test; D005260:Female; D020249:Hormone Replacement Therapy; D019382:Human Growth Hormone / Q000009:adverse effects / Q000172:deficiency* / Q000627:therapeutic use*; D006801:Humans; D018972:Insulin-Like Growth Factor Binding Protein 3 / Q000097:blood; D007334:Insulin-Like Growth Factor I / Q000032:analysis; D050356:Lipid Metabolism; D008297:Male; D011788:Quality of Life
chemical_list: D018972:Insulin-Like Growth Factor Binding Protein 3; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I