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Growth hormone treatment in 35 prepubertal children with achondroplasia: A five‐year dose‐response trial
Author(s) -
Thomas Hertel Niels,
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
Publication year - 2005
Publication title -
acta pædiatrica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.772
H-Index - 115
eISSN - 1651-2227
pISSN - 0803-5253
DOI - 10.1111/j.1651-2227.2005.tb01811.x
Subject(s) - medicine , achondroplasia , short stature , growth hormone treatment , sitting , growth velocity , arm span , growth hormone , endocrinology , pediatrics , hormone , anthropometry , pathology
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.