Growth Hormone Therapy in Pediatric Disorders: Long-term Outcomes, Treatment Optimization, and Real-world Clinical Evidence

Growth Hormone Therapy is widely used in pediatric endocrinology for conditions including growth hormone deficiency, Turner syndrome, Prader–Willi syndrome, small for gestational age, chronic renal disease, and idiopathic short stature. While short-term efficacy in promoting linear growth is well established, long-term outcomes extending into adulthood remain an area of active investigation. Questions persist regarding metabolic health, cardiovascular risk, bone quality, neurocognitive development, safety, adherence, and cost-effectiveness. In parallel, advances in long-acting formulations, precision dosing strategies, and real-world registry data are reshaping clinical practice and offering opportunities to optimize individualized treatment approaches.This Research Topic aims to provide a comprehensive and clinically relevant overview of growth hormone therapy in pediatric disorders, with a particular focus on long-term safety, metabolic and skeletal outcomes, and treatment optimization strategies. We welcome original research, systematic reviews, meta-analyses, real-world registry studies, and translational investigations addressing predictors of response, adherence, pharmacogenomics, and evolving therapeutic formulations. By integrating mechanistic insights with real-world clinical evidence, this collection seeks to inform precision endocrinology approaches and support evidence-based decision-making in pediatric growth disorders, ultimately improving lifelong health trajectories for affected children and adolescents. Themes of interest include, but are not limited to:• Long-term efficacy and safety of growth hormone therapy across pediatric indications• Treatment optimization including dosing strategies, IGF-1 monitoring, and individualized response prediction• Comparative evaluation of daily versus long-acting growth hormone formulations• Metabolic, skeletal, and cardiovascular outcomes during and after GHT• Real-world clinical evidence, registry data, and transition to adult care following pediatric GHT• Diagnostic strategies and biomarkers for growth hormone deficiency that inform treatment selection and optimization Topic editors: Reiko Horikawa, Hae Sang Lee Source: https://www.frontiersin.org/research-topics/80122/growth-hormone-therapy-in-pediatric-disorders-long-term-outcomes-treatment-optimization-and-real-world-clinical-evidence