title: A 4-Year, Open-Label, Multicenter, Randomized Trial of Genotropin® Growth Hormone in Patients with Idiopathic Short Stature: Analysis of 4-Year Data Comparing Efficacy, Efficiency, and Safety between an Individualized, Target-Driven Regimen and Standard Dosing.
abstract: OBJECTIVE
  Growth hormone (GH) treatment regimens for children with non-GH-deficient, idiopathic short stature (ISS) have not been optimized. To compare the efficacy, efficiency, and safety of an individualized, target-driven GH regimen with standard weight-based dosing after 4 years of treatment.
  
  
  METHODS
  This is a 4-year, open-label, multicenter, randomized trial comparing individualized, formula-based dosing of Genotropin® versus a widely used ISS dose of Genotropin®. Subjects were prepubertal, had a bone age of 3-10 years for males and 3-9 years for females, were naive to GH treatment, and had a height standard deviation score (Ht SDS) of -3 to -2.25, a height velocity <25th percentile for their bone age, and peak stimulated GH >10 ng/ml. After the first 2 years, the individualized-dosing group was further randomized to either 0.18 or 0.24 mg/kg/week.
  
  
  RESULTS
  At 4 years, subjects in all treatment regimens achieved similar average height gains of +1.3 SDS; however, the individualized dosing regimen utilized less GH to achieve an equivalent height gain.
  
  
  CONCLUSIONS
  Individualized, formula-based GH dosing, followed by a dose reduction after 2 years, provides a more cost-effective growth improvement in patients with ISS than currently employed weight-based regimens.
authors.affiliation: University of Maryland School of Medicine, Baltimore, Md., USA.
authors.forename: Debra R
authors.initials: DR
authors.lastname: Counts
authors.forename: Lawrence A
authors.initials: LA
authors.lastname: Silverman
authors.forename: Natasa
authors.initials: N
authors.lastname: Rajicic
authors.forename: Mitchell E
authors.initials: ME
authors.lastname: Geffner
authors.forename: Ron S
authors.initials: RS
authors.lastname: Newfield
authors.forename: Paul
authors.initials: P
authors.lastname: Thornton
authors.forename: Mauri
authors.initials: M
authors.lastname: Carakushansky
authors.forename: Oscar
authors.initials: O
authors.lastname: Escobar
authors.forename: Robert
authors.initials: R
authors.lastname: Rapaport
authors.forename: Lynne
authors.initials: L
authors.lastname: Levitsky
authors.forename: Deborah
authors.initials: D
authors.lastname: Rotenstein
authors.forename: Judith
authors.initials: J
authors.lastname: Hey-Hadavi
authors.forename: Michael P
authors.initials: MP
authors.lastname: Wajnrajch
mesh_terms: D001827:Body Height / Q000187:drug effects*; D001846:Bone Development / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D003362:Cost-Benefit Analysis; D004305:Dose-Response Relationship, Drug; D005260:Female; D006130:Growth Disorders / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D010290:Parents; D057285:Precision Medicine; D011994:Recombinant Proteins / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D019382:Human Growth Hormone