RT Journal Article
T1 Gene expression signatures predict response to therapy with growth hormone.
A1 Stevens, Adam
A1 Murray, Philip
A1 De-Leonibus, Chiara
A1 Garner, Terence
A1 Koledova, Ekaterina
A1 Ambler, Geoffrey
A1 Kapelari, Klaus
A1 Binder, Gerhard
A1 Maghnie, Mohamad
A1 Zucchini, Stefano
A1 Bashnina, Elena
A1 Skorodok, Julia
A1 Yeste, Diego
A1 Belgorosky, Alicia
A1 Lopez-Siguero, Juan-Pedro
A1 Coutant, Regis
A1 Vangsoy-Hansen, Eirik
A1 Hagenas, Lars
A1 Dahlgren, Jovanna
A1 Deal, Cheri
A1 Chatelain, Pierre
A1 Clayton, Peter
K1 Gene Expression Profiling
K1 Genetic Markers
K1 Growth Disorders
K1 Human Growth Hormone
AB Recombinant human growth hormone (r-hGH) is used as a therapeutic agent for disorders of growth including growth hormone deficiency (GHD) and Turner syndrome (TS). Treatment is costly and current methods to model response are inexact. GHD (n = 71) and TS patients (n = 43) were recruited to study response to r-hGH over 5 years. Analysis was performed using 1219 genetic markers and baseline (pre-treatment) blood transcriptome. Random forest was used to determine predictive value of transcriptomic data associated with growth response. No genetic marker passed the stringency criteria for prediction. However, we identified an identical set of genes in both GHD and TS whose expression could be used to classify therapeutic response to r-hGH with a high accuracy (AUC > 0.9). Combining transcriptomic markers with clinical phenotype was shown to significantly reduce predictive error. This work could be translated into a single genomic test linked to a prediction algorithm to improve clinical management. 
PB Nature Publishing Group
YR 2021
FD 2021-05-27
LK http://hdl.handle.net/10668/17843
UL http://hdl.handle.net/10668/17843
LA en
NO Stevens A, Murray P, De Leonibus C, Garner T, Koledova E, Ambler G, et al. Gene expression signatures predict response to therapy with growth hormone. Pharmacogenomics J. 2021 Oct;21(5):594-607
DS RISalud
RD Apr 7, 2025