Treatment of growth hormone deficiency with long-acting growth hormones in children, adolescents and adults: Clinical evidence and practical considerations.

IF 2.9
Agnès Linglart, Régis Coutant, Véronique Kerlan, Marc Nicolino, Michel Polak, Gérald Raverot, Patrice Rodien, Thierry Brue
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Abstract

Growth hormone deficiency (GHD) is a rare endocrine disorder affecting both children and adults that may occur in isolation or as part of multihormonal hypopituitarism. Recombinant human growth hormone (GH) administered as daily injections has long been the standard of care and improves growth, body composition and quality of life. However, daily administration is associated with a substantial treatment burden and adherence is frequently suboptimal, particularly in children and adolescents. Long-acting growth hormone (LAGH) formulations have been developed to reduce injection frequency and potentially support long-term adherence. Three once-weekly LAGH products are currently available in the EU: lonapegsomatropin, somatrogon and somapacitan. Clinical trials have demonstrated non-inferiority of LAGHs compared with daily GH in children in terms of annualised height velocity. Safety profiles were consistent with those of daily GH. This review summarises the clinical evidence for LAGH formulations, including patient preference and the impact on metabolism, bone health and muscle strength. With a focus on somapacitan, currently the only LAGH approved in France for both children and adults, the review discusses practical aspects of dosing and monitoring across age groups and addresses use in special populations and during the transition period from paediatric to adult care. Ongoing follow-up and real-world studies will further clarify long-term safety, adherence and metabolic outcomes.

儿童、青少年和成人用长效生长激素治疗生长激素缺乏症:临床证据和实际考虑。
生长激素缺乏症(GHD)是一种影响儿童和成人的罕见内分泌疾病,可单独发生或作为多激素垂体功能减退症的一部分。重组人生长激素(GH)作为每日注射长期以来一直是护理的标准,并改善生长,身体成分和生活质量。然而,每天给药会带来巨大的治疗负担,而且依从性往往不是最佳的,特别是在儿童和青少年中。长效生长激素(LAGH)制剂已开发,以减少注射频率和潜在的支持长期依从性。目前在欧盟有三种每周一次的LAGH产品:lonapegsomatropin, somatroon和somapacitan。临床试验表明,在年化身高速度方面,LAGHs与儿童每日生长激素相比无劣效性。安全概况与每日GH一致。本文综述了LAGH制剂的临床证据,包括患者偏好和对代谢、骨骼健康和肌肉力量的影响。somapacitan是目前法国批准的唯一一种用于儿童和成人的LAGH,本综述讨论了跨年龄组给药和监测的实际方面,并讨论了特殊人群和从儿科到成人护理过渡时期的使用情况。正在进行的随访和现实世界的研究将进一步阐明长期安全性、依从性和代谢结果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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