Growth hormone treatment in 35 prepubertal children with achondroplasia: a five-year dose-response trial.
BACKGROUND
Achondroplasia is a skeletal dysplasia with extreme, disproportionate, short stature.


OBJECTIVE
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.


CONCLUSIONS
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.
Hertel
Niels Thomas
NT
Paediatric Endocrinology Unit, Karolinska Hospital, Stockholm, Sweden.
Eklöf
Ole
O
Ivarsson
Sten
S
Aronson
Stefan
S
Westphal
Otto
O
Sipilä
Ilkka
I
Kaitila
Ilkka
I
Bland
Jon
J
Veimo
Dag
D
Müller
Jørn
J
Mohnike
Klaus
K
Neumeyer
Lo
L
Ritzen
Martin
M
Hagenäs
Lars
L
D000130:Achondroplasia / Q000175:diagnosis* / Q000188:drug therapy*; D000293:Adolescent; D015415:Biomarkers; D001827:Body Height / Q000187:drug effects; D015992:Body Mass Index; D002648:Child; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006130:Growth Disorders / Q000175:diagnosis / Q000188:drug therapy; D013006:Growth Hormone / Q000097:blood; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D018970:Insulin-Like Growth Factor Binding Protein 1 / Q000097:blood; D008297:Male; D020714:Maximum Tolerated Dose; D011336:Probability; D012016:Reference Values; D012720:Severity of Illness Index; D016896:Treatment Outcome
D015415:Biomarkers; D018970:Insulin-Like Growth Factor Binding Protein 1; D019382:Human Growth Hormone; D013006:Growth Hormone