title: A four-year, open-label, multi-center, randomized, two-arm study of Genotropin® in patients with idiopathic short stature: comparison of an individualized, target-driven treatment regimen to standard dosing of Genotropin® - analysis of two-year data.
abstract: BACKGROUND
  Several models have been developed to predict growth response to growth hormone (GH) based on auxological and biochemical parameters for children with non-GH-deficient, idiopathic short stature (ISS).
  
  
  OBJECTIVE
  To demonstrate if an individualized, formula-based, target-driven GH regimen for children with ISS would lead to a height (Ht) gain to -1.3 SDS during the first 24 months of treatment of this 4-year study, with less variability than with standard weight-based dosing.
  
  
  METHODS
  A 4-year, open-label, multi-center, randomized, two-arm study comparing formula-based dosing of Genotropin® GH from 0.18 to 0.7 mg/kg/week versus standard FDA-approved ISS dosing of Genotropin® (0.37 mg/kg/week). Subjects (n = 316, 89 females) were prepubertal, 3-14 years of age, bone age 3-10 years (m) and 3-9 years (f), naive to GH treatment, Ht SDS -3 to -2.25, Ht velocity <25th percentile for bone age, and peak GH >10 ng/ml.
  
  
  RESULTS
  The majority (83%) of subjects had Ht SDS within the normal range by 2 years. All subjects displayed catch-up growth consistent with other studies of GH treatment of ISS.
  
  
  CONCLUSIONS
  The formula-based therapy did not meet the primary endpoint achieving targeted gain with lower variability. No new safety concerns were found.
authors.affiliation: University of Maryland, Baltimore, Md., USA.
authors.forename: D R
authors.initials: DR
authors.lastname: Counts
authors.forename: L A
authors.initials: LA
authors.lastname: Silverman
authors.forename: M E
authors.initials: ME
authors.lastname: Geffner
authors.forename: N
authors.initials: N
authors.lastname: Rajicic
authors.forename: J
authors.initials: J
authors.lastname: Hey-Hadavi
authors.forename: P S
authors.initials: PS
authors.lastname: Thornton
authors.forename: M P
authors.initials: MP
authors.lastname: Wajnrajch
mesh_terms: D002648:Child; D002675:Child, Preschool; D004393:Dwarfism, Pituitary / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology*; D005260:Female; D019382:Human Growth Hormone / Q000008:administration & dosage* / Q000009:adverse effects; D006801:Humans; D008297:Male; D013997:Time Factors
chemical_list: D019382:Human Growth Hormone