We found a match
Your institution may have rights to this item. Sign in to continue.
- Title
Growth hormone treatment in 35 prepubertal children with achondroplasia: a five-year dose-response trial.
- Authors
Hertel, Niels Thomas; Eklöf, Ole; Ivarsson, Sten; Aronson, Stefan; Westphal, Otto; Sipilä, Ilkka; Kaitila, Ilkka; Bland, Jon; Veimo, Dag; Müller, Jørn; Mohnike, Klaus; Neumeyer, Lo; Ritzen, Martin; Hagenäs, Lars; Eklöf, Ole; Sipilä, Ilkka; Müller, Jørn; Hagenäs, Lars
- Abstract
<bold>Background: </bold>Achondroplasia is a skeletal dysplasia with extreme, disproportionate, short stature.<bold>Aim: </bold>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.<bold>Methods: </bold>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.<bold>Results: </bold>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.<bold>Conclusion: </bold>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.
- Subjects
ACHONDROPLASIA; SOMATOTROPIN; BONE diseases; DWARFISM; FETAL diseases; INBORN errors of metabolism; PEDIATRIC endocrinology; CHILD development deviations; PEDIATRICS; CARRIER proteins; COMPARATIVE studies; DRUG administration; DRUG dosage; DOSE-effect relationship in pharmacology; DRUG toxicity; GROWTH disorders; LONGITUDINAL method; RESEARCH methodology; MEDICAL cooperation; PROBABILITY theory; REFERENCE values; RESEARCH; STATURE; EVALUATION research; BODY mass index; HUMAN growth hormone; RANDOMIZED controlled trials; TREATMENT effectiveness; SEVERITY of illness index; THERAPEUTICS
- Publication
Acta Paediatrica, 2005, Vol 94, Issue 10, p1402
- ISSN
0803-5253
- Publication type
journal article
- DOI
10.1080/08035250510039982