Key result
Response to recombinant human growth hormone in children with short stature is largely polygenic, and transcriptomic data combined with network models show promise in predicting treatment response.
Pharmacogenomic and transcriptomic data show promise in predicting the highly variable growth response to recombinant human growth hormone in children with short stature.
Pharmacogenomic prediction of rhGH response in short stature remains investigational; supports polygenic models but leaves open prospective validation.
We present current knowledge concerning the pharmacogenomics of growth hormone therapy in children with short stature. We consider the evidence now emerging for the polygenic nature of response to recombinant human growth hormone (r-hGH). These data are related predominantly to the use of transcriptomic data for prediction. The impact of the complex interactions of developmental phenotype over childhood on response to r-hGH are discussed. Finally, the issues that need to be addressed in order to develop a clinical test are described.
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Stevens et al. (2021) conducted a review in Short stature. Recombinant human growth hormone (r-hGH) was evaluated. Response to recombinant human growth hormone in children with short stature is largely polygenic, and transcriptomic data combined with network models show promise in predicting treatment response.
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