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长效生长激素治疗的原理及未来展望。

Rationale for Long-acting Growth Hormone Therapy and Future Aspects.

作者信息

Çetinkaya Semra, Eren Erdal, Erdoğan Furkan, Darendeliler Feyza

机构信息

University of Health Sciences Türkiye, Ankara Dr. Sami Ulus Obstetrics and Gynecology, Child Health and Diseases Training and Research Hospital, Clinic of Child Health and Diseases, Ankara, Türkiye

Bursa Uludağ University Faculty of Medicine, Department of Child Health and Diseases, Bursa, Türkiye

出版信息

J Clin Res Pediatr Endocrinol. 2025 Mar 19;17(1):1-8. doi: 10.4274/jcrpe.galenos.2024.2023-11-8. Epub 2024 Mar 15.

DOI:10.4274/jcrpe.galenos.2024.2023-11-8
PMID:38488050
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11923522/
Abstract

Recombinant growth hormone (GH) is administered as daily subcutaneous injections. Daily treatment can be challenging for children/adolescents, as well as for parents and/or caregivers, such as legal representatives or guardians of children in institutional care. Challenges associated with daily treatment may result in missing several doses but non-adherence with treatment leads to inadequate growth response. As an inadequate growth response does not meet criteria for continuing treatment, payers (commercial or public) may decide to end reimbursement. Novel long-acting GH (LAGH) formulations with extended half-life may be administered less frequently and aim to improve patient convenience and consequently to improve adherence and responses to treatment. LAGH formulations can restore growth velocity and body composition as effectively as daily treatment, without unexpected adverse effects, as reported in randomized clinical trials.

摘要

重组生长激素(GH)通过每日皮下注射给药。每日治疗对于儿童/青少年以及父母和/或护理人员(如机构照料中儿童的法定代表或监护人)来说可能具有挑战性。与每日治疗相关的挑战可能导致错过几剂,但不坚持治疗会导致生长反应不足。由于生长反应不足不符合继续治疗的标准,支付方(商业或公共)可能会决定终止报销。半衰期延长的新型长效生长激素(LAGH)制剂给药频率可能较低,旨在提高患者便利性,从而改善依从性和治疗反应。随机临床试验报告显示,LAGH制剂恢复生长速度和身体成分的效果与每日治疗一样有效,且无意外不良影响。

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2
Physician experience with once-weekly somatrogon versus once-daily rhGH regimen in pediatric patients with growth hormone deficiency: a cross-sectional survey of physicians from the global phase 3 study.医生对每周一次索马鲁肽与每日一次 rhGH 方案治疗儿童生长激素缺乏症的经验:来自全球 3 期研究的医生的横断面调查。
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Model-Based Analysis of IGF-I Response, Dosing, and Monitoring for Once-Weekly Somapacitan in Children With GH Deficiency.基于模型的生长激素缺乏症儿童每周一次索马帕西坦的IGF-I反应、剂量和监测分析
J Endocr Soc. 2023 Sep 11;7(11):bvad115. doi: 10.1210/jendso/bvad115. eCollection 2023 Oct 9.
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Development of a Minimum Dataset for the Monitoring of Recombinant Human Growth Hormone Therapy in Children with Growth Hormone Deficiency: A GloBE-Reg Initiative.生长激素缺乏症儿童重组人生长激素治疗监测最小数据集的制定:GloBE-Reg 倡议。
Horm Res Paediatr. 2024;97(4):365-373. doi: 10.1159/000533763. Epub 2023 Sep 13.
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