The potential of prediction models based on data from KIGS as tools to measure responsiveness to growth hormone

Various prediction models have been developed, based on data documented wit hin KIGS (Pharmacia International Growth Database), for use in the growth h ormone (GH) treatment of children with short stature resulting from GH defi ciency (GHD) or other causes. In addition to the practical value of such mo dels as part of a 'forward strategy' guiding GH treatment, we now propose t hat prediction models may also be useful for the identification of individu al variance in responsiveness. In a comparison involving 1,800 children wit h idiopathic GHD (IGHD), 151 children who acquired GHD after treatment for medulloblastoma and 192 children with GHD accompanying craniopharyngioma, i t was shown that the responsiveness to GH of patients with craniopharyngiom a equalled that of IGHD patients, whereas patients with medulloblastoma wer e less responsive. These observations and the identification of 'good' and 'poor' responders to GH have practical clinical consequences (e.g. modifica tion of treatment), and will, in the future, lead to the identification of those factors which determine the variability of sensitivity to GH. This wi ll improve the efficacy and safety of GH treatment as well as reducing the costs involved. Copyright (C) 2001 S. Karger AG, Basel.