Long-acting growth hormones: innovations in treatment and guidance on patient selection in pediatric growth hormone deficiency

Long-acting growth hormones (LAGHs) represent a significant advancement in the treatment of pediatric growth hormone deficiency (GHD), offering an alternative to daily recombinant human growth hormone (rhGH) therapy. Traditional rhGH treatments, while effective, require daily injections, often leading to poor adherence due to the frequency of dosing, injection pain, and difficulties with storage and travel. In contrast, LAGHs, such as somatrogon, somapacitan, and lonapegsomatropin, are designed for once-weekly administration, improving patient compliance and quality of life. LAGHs have demonstrated non-inferiority to daily rhGH in phase 3 clinical trials, showing similar efficacy in terms of growth velocity and safety profiles. Despite these advantages, there remain concerns regarding the altered pharmacodynamics of LAGHs, such as the lack of pulsatile secretion and the potential for antibody formation. While the overall safety of LAGHs has been confirmed, some side effects, like lipoatrophy at the injection site, may occur, especially with PEGylated formulations. Guidelines for prescribing LAGHs are still evolving. They are not yet approved for other conditions traditionally treated with rhGH, such as Turner or Noonan syndrome. Pediatric endocrinologists must carefully consider which patient groups would benefit most from this therapy, particularly those at risk for poor adherence to daily injections, such as patients undergoing multi-drug therapy, patients with needle phobia or behavioral disorders, very young children, adolescents, patients with separated parents, families that travel frequently, or children involved in activities like scouting. LAGHs present an opportunity to enhance therapeutic outcomes and adherence, but careful patient selection remains critical to maximizing their potential benefits.