title: Safety Outcomes and Near-Adult Height Gain of Growth Hormone-Treated Children with SHOX Deficiency: Data from an Observational Study and a Clinical Trial.
abstract: OBJECTIVE
  To assess auxological and safety data for growth hormone (GH)-treated children with SHOX deficiency.
  
  
  METHODS
  Data were examined for GH-treated SHOX-deficient children (n = 521) from the observational Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS). For patients with near-adult height information, GeNeSIS results (n = 90) were compared with a clinical trial (n = 28) of SHOX-deficient patients. Near-adult height was expressed as standard deviation score (SDS) for chronological age, potentially increasing the observed effect of treatment.
  
  
  RESULTS
  Most SHOX-deficient patients in GeNeSIS had diagnoses of Leri-Weill syndrome (n = 292) or non-syndromic short stature (n = 228). For GeNeSIS patients with near-adult height data, mean age at GH treatment start was 11.0 years, treatment duration 4.4 years, and height SDS gain 0.83 (95% confidence interval 0.49-1.17). Respective ages, GH treatment durations and height SDS gains for GeNeSIS patients prepubertal at baseline (n = 42) were 9.2 years, 6.0 years and 1.19 (0.76-1.62), and for the clinical trial cohort they were 9.2 years, 6.0 years and 1.25 (0.92-1.58). No new GH-related safety concerns were identified.
  
  
  CONCLUSIONS
  Patients with SHOX deficiency who had started GH treatment before puberty in routine clinical practice had a similar height gain to that of patients in the clinical trial on which approval for the indication was based, with no new safety concerns.
authors.affiliation: Endocrinology and Diabetes Unit, Eli Lilly, Neuilly-sur-Seine, France.
authors.forename: Imane
authors.initials: I
authors.lastname: Benabbad
authors.forename: Myriam
authors.initials: M
authors.lastname: Rosilio
authors.forename: Christopher J
authors.initials: CJ
authors.lastname: Child
authors.forename: Jean-Claude
authors.initials: JC
authors.lastname: Carel
authors.forename: Judith L
authors.initials: JL
authors.lastname: Ross
authors.forename: Cheri L
authors.initials: CL
authors.lastname: Deal
authors.forename: Stenvert L S
authors.initials: SL
authors.lastname: Drop
authors.forename: Alan G
authors.initials: AG
authors.lastname: Zimmermann
authors.forename: Nan
authors.initials: N
authors.lastname: Jia
authors.forename: Charmian A
authors.initials: CA
authors.lastname: Quigley
authors.forename: Werner F
authors.initials: WF
authors.lastname: Blum
mesh_terms: D002648:Child; D005260:Female; D006801:Humans; D008297:Male; D001827:Body Height / Q000187:drug effects / Q000235:genetics; D002657:Child Development / Q000187:drug effects; D005500:Follow-Up Studies; D006130:Growth Disorders / Q000188:drug therapy / Q000235:genetics / Q000503:physiopathology; D018398:Homeodomain Proteins / Q000235:genetics; D019382:Human Growth Hormone / Q000008:administration & dosage; D010009:Osteochondrodysplasias / Q000188:drug therapy / Q000235:genetics / Q000503:physiopathology; D000074122:Short Stature Homeobox Protein; D002986:Clinical Trials as Topic; D064887:Observational Studies as Topic
chemical_list: D018398:Homeodomain Proteins; D019382:Human Growth Hormone; D000074122:Short Stature Homeobox Protein; C106071:SHOX protein, human