Predictors of response to growth hormone therapy in children with idiopathic growth hormone deficiency: a narrative review

Background: Growth hormone deficiency (GHD) in childhood impairs linear growth and may affect body composition, metabolism, and quality of life; recombinant human growth hormone (rhGH) therapy improves outcomes, but response is highly variable, especially in idiopathic GHD (IGHD). Objective: To summarize current evidence on predictors of growth response to rhGH therapy in children with IGHD, focusing on clinical, biochemical, and treatment-related determinants. Methods: This is a narrative review dealing with studies assessing clinical, auxological, biochemical and treatment-associated factors that may influence response to rhGH in IGHD. Results: Early treatment initiation, baseline short stature, prepubertal status, and higher early height growth velocity are strong clinical predictors; biochemical markers, including GH peak, IGF-1, and IGFBP-3, provide complementary information. Modifiable factors such as GH dose, adherence to therapy, and therapy duration also influence outcomes. Integrated predictive models improve accuracy, but require further validation. Conclusions: Growth response to rhGH in IGHD is multifactorial and could be individualized: early identification of suboptimal responders and personalized treatment strategies that integrate clinical, biochemical, and treatment-related data may optimize the final outcome. Future research studies should focus on validated predictive models incorporating genetic and molecular markers.