title: Efficacy and safety of growth hormone treatment in children with hypochondroplasia: comparison with an historical cohort.
abstract: OBJECTIVE
  Hypochondroplasia (HCH) is a skeletal dysplasia characterized by disproportionate short stature. The aims of the study are to evaluate efficacy and safety of recombinant human growth hormone (r-hGH) therapy in HCH children, when compared with a historical cohort of untreated HCH children.
  
  
  METHODS
  Nineteen HCH patients with an initial height standard deviation score (SDS) ≤-2 and a mean age of 9.3 ± 3.1 years were treated with a mean r-hGH dose of 0.053 mg/kg/day over 3 years. Growth charts were derived from the historical cohort (n = 40).
  
  
  RESULTS
  Height gain in the treated population was +0.62 ± 0.81 SDS greater than in the general population, and +1.39 ± 0.9 SDS greater than in the historical untreated HCH cohort (mean gain of 7.4 ± 6.6 cm gain). A negative correlation between height gain and age at treatment initiation was reported (p = 0.04). There was no significant difference in response between patients with fibroblast growth factor receptor 3 mutations and those without. No treatment-related serious adverse events were reported.
  
  
  CONCLUSIONS
  r-hGH treatment is well tolerated and effective in improving growth in HCH patients, particularly when started early. The treatment effect varies greatly and must be evaluated for each patient during treatment to determine the value of continued therapy.
authors.affiliation: Pediatric Endocrinology, Gynecology and Diabetes, Centre des Maladies Endocriniennes Rares de la Croissance, Hôpital Universitaire Necker-Enfants Malades, Paris, France.
authors.forename: Graziella
authors.initials: G
authors.lastname: Pinto
authors.forename: Valérie
authors.initials: V
authors.lastname: Cormier-Daire
authors.forename: Martine
authors.initials: M
authors.lastname: Le Merrer
authors.forename: Dinane
authors.initials: D
authors.lastname: Samara-Boustani
authors.forename: Geneviève
authors.initials: G
authors.lastname: Baujat
authors.forename: Laurence
authors.initials: L
authors.lastname: Fresneau
authors.forename: Magali
authors.initials: M
authors.lastname: Viaud
authors.forename: Jean Claude
authors.initials: JC
authors.lastname: Souberbielle
authors.forename: Jean Claude
authors.initials: JC
authors.lastname: Pineau
authors.forename: Michel
authors.initials: M
authors.lastname: Polak
mesh_terms: D000293:Adolescent; D001827:Body Height / Q000187:drug effects; D001842:Bone and Bones / Q000002:abnormalities*; D002648:Child; D002657:Child Development / Q000187:drug effects; D002675:Child, Preschool; D015331:Cohort Studies; D004392:Dwarfism / Q000188:drug therapy* / Q000235:genetics; D005260:Female; D020249:Hormone Replacement Therapy / Q000009:adverse effects; D019382:Human Growth Hormone / Q000008:administration & dosage / Q000627:therapeutic use*; D006801:Humans; D007279:Injections, Subcutaneous; D017880:Limb Deformities, Congenital / Q000188:drug therapy* / Q000235:genetics; D008141:Lordosis / Q000188:drug therapy* / Q000235:genetics; D008297:Male; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D019382:Human Growth Hormone