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

Hertel, N T; Eklof, O; Ivarsson, Sten LU ; Aronson, S; Westphal, O; Sipila, I; Kaitila, I; Bland, J; Veimo, D and Muller, J, et al. (2005) In Acta Pædiatrica 94(10). p.1402-1410
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... (More)
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. (Less)
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organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
skeletal dysplasia, achondroplasia, growth hormone treatment
in
Acta Pædiatrica
volume
94
issue
10
pages
1402 - 1410
publisher
Wiley-Blackwell Publishing Ltd
external identifiers
  • pmid:16299871
  • wos:000232931900010
  • scopus:27144547212
ISSN
1651-2227
DOI
10.1111/j.1651-2227.2005.tb01811.x
language
English
LU publication?
yes
id
40258df6-8379-4b4a-b38c-096b74416106 (old id 898791)
date added to LUP
2008-01-16 11:06:27
date last changed
2017-11-19 04:05:05
@article{40258df6-8379-4b4a-b38c-096b74416106,
  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.},
  author       = {Hertel, N T and Eklof, O and Ivarsson, Sten and Aronson, S and Westphal, O and Sipila, I and Kaitila, I and Bland, J and Veimo, D and Muller, J and Mohnike, K and Neumeyer, L and Ritzen, M and Hagenas, L},
  issn         = {1651-2227},
  keyword      = {skeletal dysplasia,achondroplasia,growth hormone treatment},
  language     = {eng},
  number       = {10},
  pages        = {1402--1410},
  publisher    = {Wiley-Blackwell Publishing Ltd},
  series       = {Acta Pædiatrica},
  title        = {Growth hormone treatment in 35 prepubertal children with achondroplasia: A five-year dose-response trial},
  url          = {http://dx.doi.org/10.1111/j.1651-2227.2005.tb01811.x},
  volume       = {94},
  year         = {2005},
}