Chiara Rodaro, Gianluca Tamaro, Elena Faleschini, Gianluca Tornese
{"title":"长效生长激素:儿童生长激素缺乏症的治疗创新及患者选择指导。","authors":"Chiara Rodaro, Gianluca Tamaro, Elena Faleschini, Gianluca Tornese","doi":"10.6065/apem.2448202.101","DOIUrl":null,"url":null,"abstract":"<p><p>Long-acting growth hormones (LAGHs) represent a significant advancement in the treatment of pediatric GH deficiency, offering an alternative to daily recombinant human GH (rhGH) therapy. Traditional rhGH treatments, although 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 rhGHs in phase 3 clinical trials, showing similar efficacy in terms of growth velocity and safety profiles. Despite these advantages, concerns remain regarding the altered pharmacodynamics of LAGHs such as the lack of pulsatile secretion and potential for antibody formation. Although the overall safety of LAGHs has been confirmed, side effects, such as lipoatrophy at the injection site, may occur, especially with PEGylated formulations. Guidelines for prescribing LAGHs are currently evolving. LAGHs are not yet approved for other conditions traditionally treated with rhGHs, such as Turner or Noonan syndrome. Pediatric endocrinologists should carefully consider which patient groups would benefit most from this therapy, particularly individuals at risk of poor adherence to daily injections such as patients undergoing multidrug 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 such as scouting. LAGHs present an opportunity to enhance therapeutic outcomes and adherence, but careful patient selection remains critical to maximize their potential benefits.</p>","PeriodicalId":44915,"journal":{"name":"Annals of Pediatric Endocrinology & Metabolism","volume":" ","pages":"106-110"},"PeriodicalIF":2.8000,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12061763/pdf/","citationCount":"0","resultStr":"{\"title\":\"Long-acting growth hormones: innovations in treatment and guidance on patient selection in pediatric growth hormone deficiency.\",\"authors\":\"Chiara Rodaro, Gianluca Tamaro, Elena Faleschini, Gianluca Tornese\",\"doi\":\"10.6065/apem.2448202.101\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Long-acting growth hormones (LAGHs) represent a significant advancement in the treatment of pediatric GH deficiency, offering an alternative to daily recombinant human GH (rhGH) therapy. Traditional rhGH treatments, although 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 rhGHs in phase 3 clinical trials, showing similar efficacy in terms of growth velocity and safety profiles. Despite these advantages, concerns remain regarding the altered pharmacodynamics of LAGHs such as the lack of pulsatile secretion and potential for antibody formation. Although the overall safety of LAGHs has been confirmed, side effects, such as lipoatrophy at the injection site, may occur, especially with PEGylated formulations. Guidelines for prescribing LAGHs are currently evolving. LAGHs are not yet approved for other conditions traditionally treated with rhGHs, such as Turner or Noonan syndrome. Pediatric endocrinologists should carefully consider which patient groups would benefit most from this therapy, particularly individuals at risk of poor adherence to daily injections such as patients undergoing multidrug 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 such as scouting. LAGHs present an opportunity to enhance therapeutic outcomes and adherence, but careful patient selection remains critical to maximize their potential benefits.</p>\",\"PeriodicalId\":44915,\"journal\":{\"name\":\"Annals of Pediatric Endocrinology & Metabolism\",\"volume\":\" \",\"pages\":\"106-110\"},\"PeriodicalIF\":2.8000,\"publicationDate\":\"2025-04-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12061763/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Annals of Pediatric Endocrinology & Metabolism\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.6065/apem.2448202.101\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/1/15 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"ENDOCRINOLOGY & METABOLISM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Annals of Pediatric Endocrinology & Metabolism","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.6065/apem.2448202.101","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/15 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}
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 GH deficiency, offering an alternative to daily recombinant human GH (rhGH) therapy. Traditional rhGH treatments, although 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 rhGHs in phase 3 clinical trials, showing similar efficacy in terms of growth velocity and safety profiles. Despite these advantages, concerns remain regarding the altered pharmacodynamics of LAGHs such as the lack of pulsatile secretion and potential for antibody formation. Although the overall safety of LAGHs has been confirmed, side effects, such as lipoatrophy at the injection site, may occur, especially with PEGylated formulations. Guidelines for prescribing LAGHs are currently evolving. LAGHs are not yet approved for other conditions traditionally treated with rhGHs, such as Turner or Noonan syndrome. Pediatric endocrinologists should carefully consider which patient groups would benefit most from this therapy, particularly individuals at risk of poor adherence to daily injections such as patients undergoing multidrug 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 such as scouting. LAGHs present an opportunity to enhance therapeutic outcomes and adherence, but careful patient selection remains critical to maximize their potential benefits.
期刊介绍:
The Annals of Pediatric Endocrinology & Metabolism Journal is the official publication of the Korean Society of Pediatric Endocrinology. Its formal abbreviated title is “Ann Pediatr Endocrinol Metab”. It is a peer-reviewed open access journal of medicine published in English. The journal was launched in 1996 under the title of ‘Journal of Korean Society of Pediatric Endocrinology’ until 2011 (pISSN 1226-2242). Since 2012, the title is now changed to ‘Annals of Pediatric Endocrinology & Metabolism’. The Journal is published four times per year on the last day of March, June, September, and December. It is widely distributed for free to members of the Korean Society of Pediatric Endocrinology, medical schools, libraries, and academic institutions. The journal is indexed/tracked/covered by web sites of PubMed Central, PubMed, Emerging Sources Citation Index (ESCI), Scopus, EBSCO, EMBASE, KoreaMed, KoMCI, KCI, Science Central, DOI/CrossRef, Directory of Open Access Journals(DOAJ), and Google Scholar. The aims of Annals of Pediatric Endocrinology & Metabolism are to contribute to the advancements in the fields of pediatric endocrinology & metabolism through the scientific reviews and interchange of all of pediatric endocrinology and metabolism. It aims to reflect the latest clinical, translational, and basic research trends from worldwide valuable achievements. In addition, genome research, epidemiology, public education and clinical practice guidelines in each country are welcomed for publication. The Journal particularly focuses on research conducted with Asian-Pacific children whose genetic and environmental backgrounds are different from those of the Western. Area of specific interest include the following : Growth, puberty, glucose metabolism including diabetes mellitus, obesity, nutrition, disorders of sexual development, pituitary, thyroid, parathyroid, adrenal cortex, bone or other endocrine and metabolic disorders from infancy through adolescence.