title: Short Children with CHARGE Syndrome: Do They Benefit from Growth Hormone Therapy?
abstract: OBJECTIVE
  The aim of this study was to evaluate the response to recombinant growth hormone (GH) treatment in short children with CHARGE syndrome.
  
  
  METHODS
  We identified 51 children (28 boys and 23 girls) in KIGS (Pfizer International Growth Database). The median chronological age was 7.6 years at the start of GH therapy and 13.2 years at the latest visit. Evaluation for GH deficiency (n = 33) was based on the following: peak GH level 7.3 μg/l and IGF-I level -2.01 standard deviation score (SDS). Sixteen subjects (9 boys) were followed longitudinally for 2 years.
  
  
  RESULTS
  Birth length (median SDS, -0.47) and weight (-0.97) were slightly reduced. At the start of GH therapy, height was -3.6 SDS, BMI -0.7 SDS, and the GH dose was 0.26 mg/kg/week. At the latest visit after 2.7 years of GH therapy, height had increased to -2.2 SDS and BMI to -0.5 SDS. In the longitudinal group, height increased from -3.72 SDS at the start of GH therapy to -2.92 SDS after 1 year to -2.37 SDS after 2 years of therapy (start - 2 years: p < 0.05), height velocity increased from -1.69 to 2.98 to 0.95 SDS, and BMI and GH dose (mg/kg/week) remained almost unchanged.
  
  
  CONCLUSIONS
  Our data show a positive effect of conventional doses of GH on short-term growth velocity for the longitudinal as well as for the total group, without any safety issues.
authors.affiliation: Division of Pediatric Endocrinology, Department of Pediatrics, University of Erlangen, Erlangen, Germany.
authors.forename: Helmuth G
authors.initials: HG
authors.lastname: Dörr
authors.forename: Margaret
authors.initials: M
authors.lastname: Boguszewski
authors.forename: Jovanna
authors.initials: J
authors.lastname: Dahlgren
authors.forename: David
authors.initials: D
authors.lastname: Dunger
authors.forename: Mitchell E
authors.initials: ME
authors.lastname: Geffner
authors.forename: Anita C
authors.initials: AC
authors.lastname: Hokken-Koelega
authors.forename: Anders
authors.initials: A
authors.lastname: Lindberg
authors.forename: Michel
authors.initials: M
authors.lastname: Polak
authors.forename: Raoul
authors.initials: R
authors.lastname: Rooman
mesh_terms: D000293:Adolescent; D001827:Body Height / Q000187:drug effects*; D058747:CHARGE Syndrome / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D019382:Human Growth Hormone / Q000008:administration & dosage*; D006801:Humans; D008297:Male
chemical_list: D019382:Human Growth Hormone