title: Increased height standard deviation scores in response to growth hormone therapy to near-adult height in older children with delayed skeletal maturation: results from the ANSWER Program.
abstract: BACKGROUND
  A primary goal of recombinant human growth hormone therapy (GHT) in children is attaining normal adult height. In this study, children with growth hormone deficiency (GHD) (including isolated idiopathic growth hormone deficiency [IGHD] and multiple pituitary hormone deficiency [MPHD]), idiopathic short stature (ISS), and Turner syndrome (TS) were evaluated for near-adult height (NAH) and percent achieving NAH within the normal range after approximately 4 years of GHT.
  
  
  METHODS
  Data from the American Norditropin®
  
  
  METHODS
  Web-Enabled Research (ANSWER) Program were analyzed for NAH from age at treatment start (ATS) (i.e., referral age as defined by age at enrollment in the study) to last clinic visit using one of the following two criteria: 1) age ≥18 years, or 2) if male: ≥16 years and height velocity (HV) <2 cm/year; if female: ≥15 years and HV <2 cm/year. All patients had a baseline height standard deviation score (HSDS) ≤ -2, and either GHD (n = 201), ISS (n = 19), or TS (n = 41). The main outcome measures included HSDS and corrected HSDS (HSDS-target HSDS) in response to GH treatment, and correlation of ATS with NAH HSDS.
  
  
  RESULTS
  Mean (± SD) chronological and bone ages at baseline were 14.0 ± 2.1 years and 11.7 ± 2.0 years, respectively, and mean GHT duration was 4.0 ± 1.6 years. Mean HSDS (baseline to NAH; GHD: -2.7 to -1.0; ISS: -2.8 to -1.4; TS: -3.0 to -1.8) and mean corrected HSDS (baseline to NAH; GHD: -2.1 to -0.3; ISS: -2.1 to -0.6; TS: -1.8 to -0.6) increased across diagnostic indications. Percentages of patients reaching near-adult HSDS > -2 were GHD: 87.6%; ISS: 78.9%; TS: 65.8%. Significant negative correlations were found between ATS and NAH HSDS when analyzed by sex.
  
  
  CONCLUSIONS
  Despite a relatively advanced childhood age, the majority of GH-treated patients attained mean near-adult HSDS within the normal range (HSDS > -2). Negative correlations of ATS with near-adult HSDS indicate that an earlier age at treatment start would likely have resulted in greater adult height achieved in both male and female patients.
authors.affiliation: Department of Pediatrics, Thomas Jefferson University, Philadelphia, PA 19107 USA ; Nemours/AI DuPont Hospital for Children, Wilmington, DE 19803 USA.
authors.forename: Judith L
authors.initials: JL
authors.lastname: Ross
authors.affiliation: Department of Pediatrics, Penn State College of Medicine, The Milton S. Hershey Medical Center, Hershey, PA 17033 USA.
authors.forename: Peter A
authors.initials: PA
authors.lastname: Lee
authors.affiliation: Department of Clinical Development, Medical and Regulatory Affairs, Novo Nordisk, Inc., 800 Scudders Mill Road, Plainsboro, NJ 08536 USA.
authors.forename: Robert
authors.initials: R
authors.lastname: Gut
authors.affiliation: Department of Clinical Development, Medical and Regulatory Affairs, Novo Nordisk, Inc., 800 Scudders Mill Road, Plainsboro, NJ 08536 USA.
authors.forename: John
authors.initials: J
authors.lastname: Germak
keywords: Human growth hormone; Near-adult height; Short stature