首页|期刊导航|中国实用儿科杂志|基于暂时连接技术的长效生长激素临床应用专家指导意见

基于暂时连接技术的长效生长激素临床应用专家指导意见OA

Clinical application of long-acting growth hormone based on transient conjugation technology:an expert perspective

中文摘要英文摘要

生长激素缺乏症是由内分泌系统导致、引起儿童及青少年期身材矮小的主要病因之一.外源性补充重组人生长激素是当前主要的治疗手段.重组人生长激素日制剂虽疗效确切,但长期每日注射给患儿及照护者带来了明显的注射负担,以及依从性挑战.近年来,陆续上市的长效生长激素(周制剂)可大幅降低给药频率进而提高依从性,然而,基于不同长效技术的长效生长激素在分子结构、药物特性、作用机制、药代动力学、药效学、临床应用与剂量、安全性监测等方面存在显著差异,临床应用经验尚有限,且缺乏规范指导.暂时连接技术是目前创新的生长激素长效化技术,目前已在欧美等国广泛应用于临床,但中国的应用经验和相关研究仍相对缺乏.为优化生长激素缺乏症的治疗策略并提升患儿治疗体验,特制定基于暂时连接技术的长效生长激素的临床应用专家指导意见.该意见涵盖长效生长激素的研发背景、暂时连接技术及基于该技术开发的隆培生长激素的循证证据解读和具体的临床应用建议.

Growth hormone deficiency(GHD)is one of the leading endocrine causes of short stature in children and adolescents.Exogenous supplementation with recombinant human growth hormone(rhGH)is currently the main treatment option.While daily rhGH is clinically effective,long-term daily injections pose a substantial treatment burden and compliance challenges on both patients and caregivers.In recent years,long-acting growth hormone(LAGH)products(weekly administration)have become available,substantially reducing dosing frequency and thereby improving compliance.However,LAGH products based on different long-acting technologies vary markedly in molecular structure,drug properties,mechanism of action,pharmacokinetics/pharmacodynamics,clinical application and dosing regimens,as well as in safety monitoring requirements.Clinical experience in these products remains limited,and standardized guidance is lacking.Transient conjugation technology is an innovative technology for prolonging the duration of growth hormone;it has been widely used clinically in the United States and Europe,but its application experience and related research in China remain relatively limited.To optimize treatment strategies for GHD and enhance patients' treatment experience,we have developed this expert guidance on the clinical application of LAGH based on transient conjugation technology.This expert guidance addresses the developmental background of LAGH,outlines transient conjugation technology,provides an evidence-based interpretation of lonapegsomatropin(a LAGH developed using this technology),and offers specific recommendations on clinical application.

中国医师协会儿科医师分会内分泌遗传代谢学组;罗小平

医药卫生

生长激素缺乏症长效生长激素暂时连接技术指导意见

growth hormone deficiencylong-acting growth hormonetransient conjugation technologystandardization

《中国实用儿科杂志》 2026 (5)

353-362,10

国家重点研发计划项目(2023YFC2706300)

10.19538/j.ek2026050601

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