Preliminary validation of a prediction model for the short-term growth response to growth hormone therapy in children with idiopathic short stature

A discriminant scoring system, using multivariate analysis, has been developed for pretreatment prediction of responsiveness to a 6-month trial of growth hormone (GH) treatment in short children with subnormal growth velocity, but without GH deficiency. Inclusion criteria included a birth weight above 2.5 kg, height below the 3rd centile for chronological age, height velocity below the 25th centile for bone age, no signs of puberty, a maximal GH response to pharmacological stimulation of above 10 μg/l and treatment with GH at a dose of 12–16 IU/m²/week. Children with an increase in height velocity greater than 2.5 cm/year after therapy were considered to be responders. Pretreatment clinical data from 67 patients were employed in a discriminant analysis in order to establish the model. The scoring system developed was as follows: score = -0.4 + 0.92X₁– 0.87X₂, where X₁ is the height velocity SD score (SDS) for chronological age, and X₂ is the bone age SDS for chronological age. This model had a specificity of 96.3% and a sensitivity of 92.5% in predicting the responsiveness to GH. The model has subsequently been applied to a group of 14 patients in order to establish its validity; in this group its sensitivity was 83.3% and its specificity 100%. These preliminary data suggest that the model can be used as a guideline for selecting short, slowly growing, non-GH-deficient children who will respond to short-term GH therapy.