Childhood growth hormone treatment: challenges, opportunities, and considerations

With long standing demand and popularity, growth hormone treatments continue to be a topic of interest for paediatric endocrinologists and general paediatricians due to ongoing issues regarding their long-term effects, the safety of childhood treatment, and the introduction of long-acting growth hormone preparations in the past decade. Moreover, uncertainty regarding how to approach individual patients and their treatment indications remains, particularly concerning tailored treatment goals and objectives; this uncertainty is further complicated by the multitude of approved indications that surpass substitution therapy. The paediatric endocrinologist thus grapples with pertinent questions, such as what defines reasonable treatment goals for each individual given their indications, and when (and how) to initiate the necessary discussions about risks and benefits with patients and their families. The aim of this Review is to offer advanced physiological concepts of growth hormone function, map out approved paediatric indications for treatment along with evidence on their effects and safety, highlight controversies and complexities surrounding childhood growth hormone treatment, and discuss the potential of long-acting growth hormone and future directions in the realm of childhood growth hormone treatment.