title: Growth hormone treatment of early growth failure in toddlers with Turner syndrome: a randomized, controlled, multicenter trial.
abstract: BACKGROUND
  Typically, growth failure in Turner syndrome (TS) begins prenatally, and height sd score (SDS) declines progressively from birth.
  
  
  OBJECTIVE
  This study aimed to determine whether GH treatment initiated before 4 yr of age in girls with TS could prevent subsequent growth failure. Secondary objectives were to identify factors associated with treatment response, to determine whether outcome could be predicted by a regression model using these factors, and to assess the safety of GH treatment in this young cohort.
  
  
  METHODS
  This study was a prospective, randomized, controlled, open-label, multicenter clinical trial (Toddler Turner Study, August 1999 to August 2003).
  
  
  METHODS
  The study was conducted at 11 U.S. pediatric endocrine centers.
  
  
  METHODS
  Eighty-eight girls with TS, aged 9 months to 4 yr, were enrolled.
  
  
  METHODS
  Interventions comprised recombinant GH (50 mug/kg.d; n = 45) or no treatment (n = 43) for 2 yr.
  
  
  METHODS
  The main outcome measure was baseline-to-2-yr change in height SDS.
  
  
  RESULTS
  Short stature was evident at baseline (mean length/height SDS = -1.6 +/- 1.0 at mean age 24.0 +/- 12.1 months). Mean height SDS increased in the GH group from -1.4 +/- 1.0 to -0.3 +/- 1.1 (1.1 SDS gain), whereas it decreased in the control group from -1.8 +/- 1.1 to -2.2 +/- 1.2 (0.5 SDS decline), resulting in a 2-yr between-group difference of 1.6 +/- 0.6 SDS (P < 0.0001). The baseline variable that correlated most strongly with 2-yr height gain was the difference between mid-parental height SDS and subjects' height SDS (r = 0.32; P = 0.04). Although attained height SDS at 2 yr could be predicted with good accuracy using baseline variables alone (R(2) = 0.81; P < 0.0001), prediction of 2-yr change in height SDS required inclusion of initial treatment response data (4-month or 1-yr height velocity) in the model (R(2) = 0.54; P < 0.0001). No new or unexpected safety signals associated with GH treatment were detected.
  
  
  CONCLUSIONS
  Early GH treatment can correct growth failure and normalize height in infants and toddlers with TS.
authors.affiliation: Division of Pediatric Endocrinology, University of North Carolina, CB 7039, 3341 Medical Biomolecular Research Building, Chapel Hill, North Carolina 27599-7039, USA. mld@med.unc.edu
authors.forename: Marsha L
authors.initials: ML
authors.lastname: Davenport
authors.forename: Brenda J
authors.initials: BJ
authors.lastname: Crowe
authors.forename: Sharon H
authors.initials: SH
authors.lastname: Travers
authors.forename: Karen
authors.initials: K
authors.lastname: Rubin
authors.forename: Judith L
authors.initials: JL
authors.lastname: Ross
authors.forename: Patricia Y
authors.initials: PY
authors.lastname: Fechner
authors.forename: Daniel F
authors.initials: DF
authors.lastname: Gunther
authors.forename: Chunhua
authors.initials: C
authors.lastname: Liu
authors.forename: Mitchell E
authors.initials: ME
authors.lastname: Geffner
authors.forename: Kathryn
authors.initials: K
authors.lastname: Thrailkill
authors.forename: Carol
authors.initials: C
authors.lastname: Huseman
authors.forename: Anthony J
authors.initials: AJ
authors.lastname: Zagar
authors.forename: Charmian A
authors.initials: CA
authors.lastname: Quigley
mesh_terms: D000365:Age Determination by Skeleton; D001846:Bone Development / Q000187:drug effects; D002675:Child, Preschool; D005260:Female; D006130:Growth Disorders / Q000097:blood / Q000150:complications* / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D018972:Insulin-Like Growth Factor Binding Protein 3; D018969:Insulin-Like Growth Factor Binding Proteins / Q000097:blood; D007334:Insulin-Like Growth Factor I / Q000032:analysis; D014424:Turner Syndrome / Q000097:blood / Q000188:drug therapy*
chemical_list: C515497:IGFBP3 protein, human; D018972:Insulin-Like Growth Factor Binding Protein 3; D018969:Insulin-Like Growth Factor Binding Proteins; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I