Skeletal dysplasias: response to growth hormone therapy

This study was conducted to determine the response of children with skeletal dysplasia and short stature to growth hormone therapy and whether such therapy is appropriate in skeletal dysplasia.

A retrospective analysis of the Australia-wide group of children with skeletal dysplasia registered on the Ozgrow database, using growth data at commencement and at 12 monthly intervals. Eighteen females and 35 males received growth hormone (GH) therapy for at least 12 months. Patients were aged 10.3 +/- 3.3 (mean +/- s.d.) years and had severe short stature with height standard deviation score (SDS) of -3.5 +/- 1.0 (mean +/- s.d.).

Hypochondroplasia (N = 7), metaphyseal dysplasia (N = 5) and dyschondrosteosis (N = 5) were the most frequently represented specific skeletal dysplasia subgroups. These groups and the remaining heterogeneous group did not differ significantly at baseline or subsequent growth response. Duration of GH therapy was mean (range) 36 mo (12-60 mo). Sex, age or degree of short stature did not correlate with response to GH therapy. delta height SDS was 0.3 (mean) after 12 months of GH therapy, which corresponded to an increase in mean growth velocity from 4.3 cm/yr to 6.9 cm/yr. Mean GV declined after the first year, and by 36 months was only 1.1 cm/year above baseline, while delta height SDS was 0.8

The results suggest that growth hormone therapy is not generally effective for treatment of short stature in skeletal dysplasia.