Human growth hormone treatment in prepubertal children with achondroplasia

Am J Med Genet. 1996 Feb 2;61(4):396-400. doi: 10.1002/(SICI)1096-8628(19960202)61:4<396::AID-AJMG17>3.0.CO;2-N.

Abstract

We studied the effects of recombinant human growth hormone (GH) treatment in 6 prepubertal children with achondroplasia. The patients' age ranged from 2 11/12 to 8 5/12 years and the GH dose was of 0.1 IU/kg/day subcutaneously. Auxological assessments and bone age determinations were performed 6 months before, at the beginning, and after 6 and 12 months of therapy. The growth velocity increase during the whole year of treatment ranged from 1.1 to 2.6 cm/year in 3 patients while in the others no variation was detected. No side effects were observed during the trial apart from a slight advancement of bone age in two patients. MRI at the cervicomedullary junction and CT scan of the base of the skull did not show any variation of the dimensions of the foramen magnum at the end of the trial compared to baseline. Our study shows that r-hGH can safely increase short-term growth velocity in some but not all prepubertal children with achondroplasia. Our data confirm the individual variability in the response to the GH treatment.

MeSH terms

  • Achondroplasia / drug therapy*
  • Achondroplasia / metabolism
  • Child
  • Child, Preschool
  • Female
  • Growth
  • Growth Hormone / therapeutic use*
  • Humans
  • Insulin-Like Growth Factor I / metabolism
  • Male
  • Peptide Fragments / blood
  • Procollagen / blood
  • Puberty
  • Thyroid Gland / metabolism

Substances

  • Peptide Fragments
  • Procollagen
  • procollagen type I carboxy terminal peptide
  • Insulin-Like Growth Factor I
  • Growth Hormone