Effectiveness of Recombinant Human Growth Hormone Therapy in Small-for-Gestational-Age Children With Short Stature: A Stratified Analysis Based on Genetic Variant Status.

As one of the first longitudinal analyses utilizing repeated-measures modelling in a genetically characterized patient population, this study demonstrates that while rhGH therapy effectively promoted linear growth in SGA-SS children, patients with a molecular diagnosis presented with lower baseline stature and an attenuated incremental growth response over time, highlighting the importance of genetic evaluation for personalized treatment strategies.