title: Short stature in Noonan syndrome: response to growth hormone therapy.
abstract: BACKGROUND
  Growth hormone (GH) has been used to promote growth in both the short and long term in a number of dysmorphic syndromes, including Turner syndrome. As this condition shares many clinical features with Noonan syndrome, it would seem logical to treat the latter group with GH.
  
  
  OBJECTIVE
  To assess the short and long term response to GH therapy in patients with Noonan syndrome.
  
  
  METHODS
  Analysis of patients with Noonan syndrome in the Pharmacia & Upjohn International Growth Study (this post-marketing database contains data on the majority of patients currently treated with GH in the UK). A questionnaire was also sent to participating clinicians.
  
  
  RESULTS
  Data on 66 patients (54 males) were available for study. At the start of GH therapy children were short, compared with both normal and Noonan children. During the first year of GH therapy height velocity increased from a mean of 4.9 to 7.2 cm per year. For patients treated long term with GH, mean height SDS increased from -2.9 pretreatment to -2.6 after one year and -2.3 after five years. Of the 10 patients at near final height, only one had a height above the 3rd centile for normal adults and above the mean for untreated Noonan patients. The mean increment in final height was 3.1 cm (range -1.1 to 6.5 cm).
  
  
  CONCLUSIONS
  GH therapy in patients with Noonan syndrome will improve height velocity in the short term. Longer-term therapy results in a waning of effect; initial indications are that final height is not improved substantially in most patients.
authors.affiliation: Department of Endocrinology, Birmingham Children's Hospital, Birmingham, UK. Jeremy.Kirk@BhamChildrens.WMids.NHS.UK.
authors.forename: J M
authors.initials: JM
authors.lastname: Kirk
authors.forename: P R
authors.initials: PR
authors.lastname: Betts
authors.forename: G E
authors.initials: GE
authors.lastname: Butler
authors.forename: M D
authors.initials: MD
authors.lastname: Donaldson
authors.forename: D B
authors.initials: DB
authors.lastname: Dunger
authors.forename: D I
authors.initials: DI
authors.lastname: Johnston
authors.forename: C J
authors.initials: CJ
authors.lastname: Kelnar
authors.forename: D A
authors.initials: DA
authors.lastname: Price
authors.forename: P
authors.initials: P
authors.lastname: Wilton
authors.lastname: Group tU
mesh_terms: D000293:Adolescent; D001827:Body Height / Q000187:drug effects; D002648:Child; D005260:Female; D005500:Follow-Up Studies; D006130:Growth Disorders / Q000188:drug therapy / Q000209:etiology*; D013006:Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D008134:Long-Term Care; D008297:Male; D009634:Noonan Syndrome / Q000150:complications*; D016896:Treatment Outcome
chemical_list: D013006:Growth Hormone