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Growth hormone treatment in 35 prepubertal children with achondroplasia: a five-year dose-response trial
Niels Thomas Hertel1, Ole Eklöf, Sten Ivarsson
1Paediatric Endocrinology Unit, Karolinska Hospital, Stockholm, Sweden.
Insights
Growth hormone (GH) treatment in children with achondroplasia significantly improves height over 4 years. This treatment for achondroplasia does not negatively impact body proportions, offering a promising therapeutic avenue.
Area of Science:
- Pediatric Endocrinology
- Skeletal Dysplasias
- Growth Hormone Therapy
Background:
- Achondroplasia is a genetic disorder causing extreme, disproportionate short stature.
- Understanding growth patterns and body proportions is crucial for managing achondroplasia.
Purpose of the Study:
- To evaluate the efficacy of growth hormone (GH) treatment in children with achondroplasia over a 5-year period.
- To assess the impact of GH therapy on growth velocity, height standard deviation score (HSDS), and body proportions.
- To investigate catch-down growth following a temporary interruption of GH treatment.
Main Methods:
- A 5-year randomized controlled study involving 35 children with achondroplasia.
- Two dosage groups: 0.1 IU/kg/day (n=18) and 0.2 IU/kg/day (n=17).
- Prepubertal patients had GH treatment interrupted for 12 months after 2 years to assess catch-down growth.
Main Results:
- Significant increases in growth velocity and HSDS were observed during the first two years of GH treatment.
- A temporary decrease in growth velocity below baseline occurred during the third year after treatment interruption.
- Overall, HSDS improved significantly over 5 years, and sitting height SDS showed improvement without adverse changes in trunk-leg proportion.
Conclusions:
- Growth hormone therapy effectively improves height in children with achondroplasia over 4 years.
- GH treatment does not adversely affect the disproportionate body structure characteristic of achondroplasia.
- The short-term growth response is comparable to that seen in other conditions like Turner syndrome, Noonan syndrome, and idiopathic short stature.
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.
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