Treatment of growth hormone deficiency with long-acting growth hormones in children, adolescents and adults: Clinical evidence and practical considerations.
TL;DR
The review discusses practical aspects of dosing and monitoring across age groups and addresses use in special populations and during the transition period from paediatric to adult care, including patient preference and the impact on metabolism, bone health and muscle strength.
Abstract
Growth hormone deficiency (GHD) is a rare endocrine disorder affecting both children and adults that may occur in isolation or as part of multihormonal hypopituitarism. Recombinant human growth hormone (GH) administered as daily injections has long been the standard of care and improves growth, body composition and quality of life. However, daily administration is associated with a substantial treatment burden and adherence is frequently suboptimal, particularly in children and adolescents. Long-acting growth hormone (LAGH) formulations have been developed to reduce injection frequency and potentially support long-term adherence. Three once-weekly LAGH products are currently available in the EU: lonapegsomatropin, somatrogon and somapacitan. Clinical trials have demonstrated non-inferiority of LAGHs compared with daily GH in children in terms of annualised height velocity. Safety profiles were consistent with those of daily GH. This review summarises the clinical evidence for LAGH formulations, including patient preference and the impact on metabolism, bone health and muscle strength. With a focus on somapacitan, currently the only LAGH approved in France for both children and adults, the review discusses practical aspects of dosing and monitoring across age groups and addresses use in special populations and during the transition period from paediatric to adult care. Ongoing follow-up and real-world studies will further clarify long-term safety, adherence and metabolic outcomes.