Background: Growth hormone (GH) treatment in children with short stature homeobox-containing gene (SHOX) deficiency is recognized to increase height velocity (HV) and adult height. Prediction of growth response continues to be a challenge. A comparatively accurate method is the Cologne prediction model developed in children with GH deficiency. The aim was to investigate whether this model also applies to patients with SHOX deficiency. Methods: Included were 48 patients with SHOX deficiency confirmed by DNA analysis and treated with 0.05 mg/kg/ day of somatropin. Prediction by the Cologne model uses the following variables: relative bone age (BA) retardation, baseline insulin-like growth factor-I (IGF-I), urinary deoxypyridinoline (DPD) cross-links at 4 weeks and HV at 3 months. Results: HV and height standard deviation scores (SDS) increased significantly during the first year of treatment. Predicted and observed HV (cm/year) showed a Pearson correlation coefficient of 0.50 (p < 0.001; root-meansquare error = 1.63) and for first-year change in height SDS a Pearson correlation coefficient of 0.751 (p < 0.001; root-mean-square error = 0.32). Poor response could be adequately predicted using SDS change, with sensitivity and specificity both above 70% for certain thresholds.
Conclusions:The results demonstrate that the Cologne model can be used to predict growth response in patients