title: Growth hormone treatment before the age of 4 years prevents short stature in young girls with Turner syndrome.
abstract: OBJECTIVE
  Adult height deficit seen in Turner syndrome (TS) originates, in part, from growth retardation in utero and throughout the first 3 years of life. Earlier diagnosis enables earlier therapeutic intervention, such as with recombinant human GH (r-hGH), which may help to prevent growth retardation. In this open-label, multicentre phase III study, we investigated efficacy and safety in r-hGH treatment in young girls with TS.
  
  
  METHODS
  Girls (n=61) aged <4 years with TS receiving 0.035-0.05 mg/kg per day r-hGH for 4 years were compared with an historical control group (n=51) comprising untreated, age- and height-matched girls with TS. The main outcome measure was change in height SDS (H-SDS). Other measures included changes in height velocity SDS, IGF1 levels and glucose metabolism.
  
  
  RESULTS
  After 4 years, a gain in mean H-SDS of 1.0 SDS (from -2.33±0.73 to -1.35±0.86 SDS) was observed with r-hGH treatment, in contrast to the decrease in mean H-SDS of 0.3 SDS in the control group (from -2.09±0.81 to -2.44±0.73 SDS; P<0.0001). r-hGH treatment was the main predictor of H-SDS gain and accounted for 52% of variability (multivariate analysis). r-hGH was well tolerated. As expected, IGF1 levels rose with treatment. A case of transient glucose intolerance resolved after dietary adaptation.
  
  
  CONCLUSIONS
  Early treatment with r-hGH helps to prevent natural evolution towards short stature in most girls with TS. IGF1 levels and glucose metabolism should be monitored routinely during r-hGH therapy.
authors.affiliation: Groupe Hospitalier Cochin/Saint-Vincent-de-Paul, INSERM U561, 82 Avenue Denfert Rochereau, 75674 Paris Cedex 14, France. agnes.linglart@inserm.fr
authors.forename: A
authors.initials: A
authors.lastname: Linglart
authors.forename: S
authors.initials: S
authors.lastname: Cabrol
authors.forename: P
authors.initials: P
authors.lastname: Berlier
authors.forename: C
authors.initials: C
authors.lastname: Stuckens
authors.forename: K
authors.initials: K
authors.lastname: Wagner
authors.forename: M
authors.initials: M
authors.lastname: de Kerdanet
authors.forename: C
authors.initials: C
authors.lastname: Limoni
authors.forename: J-C
authors.initials: JC
authors.lastname: Carel
authors.forename: J-L
authors.initials: JL
authors.lastname: Chaussain
mesh_terms: D000365:Age Determination by Skeleton; D001772:Blood Cell Count; D001786:Blood Glucose / Q000378:metabolism; D001827:Body Height / Q000187:drug effects*; D050260:Carbohydrate Metabolism / Q000187:drug effects; D002675:Child, Preschool; D005260:Female; D006442:Glycated Hemoglobin / Q000032:analysis; D006128:Growth / Q000187:drug effects; D013006:Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D007334:Insulin-Like Growth Factor I / Q000032:analysis; D007621:Karyotyping; D008111:Liver Function Tests; D008137:Longitudinal Studies; D016896:Treatment Outcome; D014424:Turner Syndrome / Q000188:drug therapy* / Q000473:pathology*
chemical_list: D001786:Blood Glucose; D006442:Glycated Hemoglobin; D013006:Growth Hormone; D007334:Insulin-Like Growth Factor I