Growth Hormone Treatment in 35 Prepubertal Children With Achondroplasia: A Five-Year Dose-Response Trial

Acta Paediatr. 2005 Oct;94(10):1402-10. doi: 10.1111/j.1651-2227.2005.tb01811.x.

Abstract

Background: Achondroplasia is a skeletal dysplasia with extreme, disproportionate, short stature.

Aim: In a 5-y growth hormone (GH) treatment study including 1 y without treatment, we investigated growth and body proportion response in 35 children with achondroplasia.

Methods: Patients were randomized to either 0.1 IU/kg (n = 18) or 0.2 IU/kg (n = 17) per day. GH treatment was interrupted for 12 mo after 2 y of treatment in prepubertal patients to study catch-down growth. Mean height SDS (HSDS) at start was -5.6 and -5.2 for the low- and high-dose groups, respectively, and mean age 7.3 and 6.6 y.

Results: Mean growth velocity (baseline 4.5/4.6 cm/y for the groups) increased significantly by 1.9/3.6 cm/y during the first year and by 0.5/1.5 cm/y during the second year. During the third year, a decrease of growth velocity was observed at 1.9/1.3 cm/y below baseline values. HSDS increased significantly by 0.6/0.8 during the first year of treatment and in total by 1.3/1.6 during the 5 y of study. Sitting height SDS improved significantly from -2.1/-1.7 to -0.8/0.2 during the study. Body proportion (sitting height/total height) or arm span did not show any significant change.

Conclusion: GH treatment of children with achondroplasia improves height during 4 y of therapy without adverse effect on trunk-leg disproportion. The short-term effect is comparable to that reported in Turner and Noonan syndrome and in idiopathic short stature.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Achondroplasia / diagnosis*
  • Achondroplasia / drug therapy*
  • Adolescent
  • Biomarkers
  • Body Height / drug effects
  • Body Mass Index
  • Child
  • Dose-Response Relationship, Drug
  • Drug Administration Schedule
  • Female
  • Follow-Up Studies
  • Growth Disorders / diagnosis
  • Growth Disorders / drug therapy
  • Growth Hormone / blood
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Insulin-Like Growth Factor Binding Protein 1 / blood
  • Male
  • Maximum Tolerated Dose
  • Probability
  • Reference Values
  • Severity of Illness Index
  • Treatment Outcome

Substances

  • Biomarkers
  • Insulin-Like Growth Factor Binding Protein 1
  • Human Growth Hormone
  • Growth Hormone