From Replacement to Tailoring: Evolving Concepts in the Therapy for Short Stature

Growth hormone (GH) therapy is a cornerstone in managing pediatric growth disorders, yet individual responses vary significantly despite standardized protocols. This variability underscores the need for a comprehensive predictive framework to guide clinical decisions and optimize outcomes. Key determinants of growth response include the underlying diagnosis and degree of GH sensitivity, with conditions like severe GH deficiency typically responding better than disorders such as idiopathic short stature, small for gestational age, or Turner syndrome. Patient-specific factors - including age at treatment initiation, bone age delay, mid-parental height, and auxological parameters - further shape therapeutic outcomes. Definitions of poor response remain debated, typically relying on first-year height gain or height velocity metrics. A suboptimal response should prompt reassessment of the diagnosis and therapeutic strategy. Emerging therapies offer promising alternatives and adjuncts aimed at improving adherence, targeting specific etiologies, and enhancing outcomes. Despite early success, these interventions require further validation regarding long-term efficacy, safety, and cost-effectiveness. Together, these innovations reflect a broader shift toward mechanism-driven, personalized therapy in pediatric endocrinology.Growth hormone (GH) treatment is widely used to help children with growth problems, but not all children respond in the same way. Some conditions, like severe GH deficiency, usually respond better than others, such as idiopathic short stature, being born small for gestational age, or Turner syndrome. A child's age when treatment starts, bone age and family height background, also affect how well the therapy works. Doctors often look at how much height a child gains in the first year to judge whether treatment is effective, but there is no single agreed definition of a "poor response." If the results are disappointing, it's important to recheck the diagnosis and treatment plan. Newer therapies are being developed to improve convenience, target specific causes of poor growth, and achieve better results. While early findings are encouraging, more research is needed to confirm long-term benefits, safety, and costs. Overall, the trend is moving toward more personalized treatments tailored to each child's needs.