Kevin C J Yuen1, Bradley S Miller2, Beverly M K Biller3. 1. Department of Neuroendocrinology and Neurosurgery, Barrow Pituitary Center, Barrow Neurological Institute, University of Arizona College of Medicine, Phoenix, Arizona. 2. Division of Pediatric Endocrinology, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota. 3. Neuroendocrine Unit, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Abstract
PURPOSE OF REVIEW: To discuss the rationale of developing long-acting growth hormone (LAGH) preparations, to describe the technologies designed to prolong GH action, and to address key issues regarding efficacy, safety, and monitoring while on treatment. REVIEW FINDINGS: Recombinant human GH is currently approved for daily use and has been shown to restore longitudinal growth, and improve body composition with relatively few side-effects in children and adults with GH deficiency, respectively. However, daily injections can be inconvenient, painful and distressing for some patients, resulting in decreased adherence and efficacy. Over a dozen pharmaceutical companies have designed LAGH preparations that are at various stages of development using a number of different methods to prolong GH action. SUMMARY: LAGH will represent an advancement over daily recombinant human GH injections because of fewer injections that may offer increased acceptance, tolerability, and therapeutic flexibility to patients that potentially can improve treatment outcomes. However, given the unphysiological profile of LAGH preparations, long-term surveillance of efficacy and safety are needed. This review summarizes recent developments of LAGH preparations, and highlights the importance of long-term surveillance registries to assess for efficacy and safety that will be essential for understanding the impact of prolonged exposure to these compounds.
PURPOSE OF REVIEW: To discuss the rationale of developing long-acting growth hormone (LAGH) preparations, to describe the technologies designed to prolong GH action, and to address key issues regarding efficacy, safety, and monitoring while on treatment. REVIEW FINDINGS: Recombinant human GH is currently approved for daily use and has been shown to restore longitudinal growth, and improve body composition with relatively few side-effects in children and adults with GH deficiency, respectively. However, daily injections can be inconvenient, painful and distressing for some patients, resulting in decreased adherence and efficacy. Over a dozen pharmaceutical companies have designed LAGH preparations that are at various stages of development using a number of different methods to prolong GH action. SUMMARY: LAGH will represent an advancement over daily recombinant human GH injections because of fewer injections that may offer increased acceptance, tolerability, and therapeutic flexibility to patients that potentially can improve treatment outcomes. However, given the unphysiological profile of LAGH preparations, long-term surveillance of efficacy and safety are needed. This review summarizes recent developments of LAGH preparations, and highlights the importance of long-term surveillance registries to assess for efficacy and safety that will be essential for understanding the impact of prolonged exposure to these compounds.
Authors: Meryl Brod; Michael Højby Rasmussen; Suzanne Alolga; Jane F Beck; Donald M Bushnell; Kai Wai Lee; Aristides Maniatis Journal: Pharmacoecon Open Date: 2022-10-18
Authors: Margaret C S Boguszewski; Adriane A Cardoso-Demartini; Cesar Luiz Boguszewski; Wassim Chemaitilly; Claire E Higham; Gudmundur Johannsson; Kevin C J Yuen Journal: Pituitary Date: 2021-07-25 Impact factor: 4.107