title: Long-term results of growth hormone therapy in children with short stature, subnormal growth rate and normal growth hormone response to secretagogues. Dutch Growth Hormone Working Group.
abstract: OBJECTIVE
  Growth hormone treatment in children with idiopathic short stature (ISS) leads to growth acceleration in the first years, but the effect on final height is still poorly documented. We therefore studied the long-term effect of GH therapy in children with idiopathic short stature.
  
  
  METHODS
  We have treated 27 prepubertal children with ISS with recombinant human GH (rhGH) in an initial dosage of 2 IU/m2 body surface/day subcutaneously, which was doubled either after the first year if the height velocity increment was less than 2 cm/year, or thereafter if height velocity fell below the P50 for bone age. Growth and bone maturation of the treatment group (ISS group, n = 21) were compared to those of an untreated control group with ISS (ISS controls, n = 27) and of a group of rhGH treated children with isolated GH deficiency (GHD group, n = 7).
  
  
  RESULTS
  In 9 patients of the ISS group still on treatment, height standard deviation score (HSDS) for chronological age increased from -3.8 +/- 0.7 to -2.3 +/- 0.9 (mean +/- standard deviation) over 6 years, while in matched ISS controls HSDS for age did not change. HSDS for age in the GHD group increased from -3.9 +/- 0.6 to -1.8 +/- 0.7 after 4 years, significantly more than the ISS group. Bone maturation was accelerated in the ISS and GHD groups. HSDS for bone age and predicted adult height did not change in either group. Final height in 12 children of the ISS group was -2.6 +/- 1.0 SDS. In the untreated controls final height was similar. A low integrated GH concentration over 24 hours, a low GH peak to provocative stimuli, and minimal initial BA delay predicted a favourable outcome.
  
  
  CONCLUSIONS
  rhGH treatment in this group of children with idiopathic short stature did not increase average final height. Part of the heterogeneity of the response can be attributed to the variation in endogenous GH secretion and initial bone age delay.
authors.affiliation: Department of Paediatrics, University of Leiden, The Netherlands.
authors.forename: J M
authors.initials: JM
authors.lastname: Wit
authors.forename: B
authors.initials: B
authors.lastname: Boersma
authors.forename: S M
authors.initials: SM
authors.lastname: de Muinck Keizer-Schrama
authors.forename: H E
authors.initials: HE
authors.lastname: Nienhuis
authors.forename: W
authors.initials: W
authors.lastname: Oostdijk
authors.forename: B J
authors.initials: BJ
authors.lastname: Otten
authors.forename: H A
authors.initials: HA
authors.lastname: Delemarre-Van de Waal
authors.forename: M
authors.initials: M
authors.lastname: Reeser
authors.forename: J J
authors.initials: JJ
authors.lastname: Waelkens
authors.forename: B
authors.initials: B
authors.lastname: Rikken
mesh_terms: D001827:Body Height / Q000187:drug effects; D001846:Bone Development / Q000187:drug effects; D002648:Child; D005260:Female; D005500:Follow-Up Studies; D006130:Growth Disorders / Q000097:blood / Q000188:drug therapy*; D013006:Growth Hormone / Q000097:blood / Q000627:therapeutic use*; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000032:analysis; D008297:Male; D011237:Predictive Value of Tests; D011446:Prospective Studies; D011627:Puberty; D011994:Recombinant Proteins / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D007334:Insulin-Like Growth Factor I; D013006:Growth Hormone