Growth hormone treatment in 35 prepubertal children with achondroplasia: a five-year dose-response trial.

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.

• Authors: Niels Thomas Hertel, Ole Eklöf, Sten Ivarsson, Stefan Aronson, Otto Westphal, Ilkka Sipilä, Ilkka Kaitila, Jon Bland, Dag Veimo, Jørn Müller, Klaus Mohnike, Lo Neumeyer, Martin Ritzen, Lars Hagenäs
• Journal: Acta paediatrica (Oslo, Norway : 1992) (Acta Paediatr) Vol. 94 Issue 10 Pg. 1402-10 (Oct 2005) ISSN: 0803-5253 [Print] Norway
• PMID: 16299871 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

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

Topics

• Achondroplasia (diagnosis, 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, 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