Miller Bradley S, Blair Jo, Horikawa Reiko, Linglart Agnès, Yuen Kevin C J
Division of Pediatric Endocrinology, University of Minnesota Medical School, MHealth Fairview Masonic Children's Hospital, Minneapolis, MN, USA.
Department of Paediatric Endocrinology, Alder Hey Children's NHS Foundation Trust, Liverpool, UK.
Drug Des Devel Ther. 2024 Feb 3;18:291-306. doi: 10.2147/DDDT.S315172. eCollection 2024.
Growth hormone (GH) replacement therapy for growth hormone deficiency (GHD) in children and adults has for over 25 years, until recently, been administered as daily injections. This daily treatment regimen often incurs a burden to patients and caregivers, leading to high rates of non-adherence and, consequently, decreased treatment efficacy outcomes. To address this shortcoming, long-acting growth hormones (LAGHs) have been developed with the aim of reducing the burden of daily injections, thereby potentially improving treatment adherence and outcomes. Somapacitan (Sogroya) (Novo Nordisk, Bagsværd, Denmark) is a LAGH currently approved for the treatment of adult and childhood GHD (AGHD and CGHD, respectively) in several countries. Other LAGHs, such as somatrogon (Ngenla) (Pfizer, New York, United States) and lonapegsomatropin/TransCon GH (Skytrofa) (Ascendis Pharma, Copenhagen, Denmark), are also currently approved and available for the treatment of CGHD in several countries. In this review, we will consider the method of protraction, pharmacokinetics (PK) and pharmacodynamics (PD), efficacy, and safety results of somapacitan in adult and pediatric trials and how these characteristics differ from those of the other aforementioned LAGHs. Additionally, the administration of somapacitan and timing of measurement of serum insulin-like growth factor-I (IGF-I) levels are summarized. Information on administration, advice on missed doses, and clinical guidelines are discussed, as well as identifying which patients are suitable for somapacitan therapy, and how to monitor and adjust dosing whilst on therapy.
在过去25年多的时间里,直到最近,儿童和成人生长激素缺乏症(GHD)的生长激素(GH)替代疗法一直采用每日注射的方式。这种每日治疗方案常常给患者和护理人员带来负担,导致不依从率很高,进而降低了治疗效果。为了解决这一缺点,长效生长激素(LAGH)已被研发出来,目的是减轻每日注射的负担,从而有可能提高治疗依从性和治疗效果。索马帕西坦(Sogroya)(丹麦巴格斯瓦尔德的诺和诺德公司)是一种长效生长激素,目前已在多个国家被批准用于治疗成人和儿童GHD(分别为成人GHD和儿童GHD)。其他长效生长激素,如索马罗贡(Ngenla)(美国纽约的辉瑞公司)和洛奈培索托品/转化生长激素(Skytrofa)(丹麦哥本哈根的Ascendis Pharma公司),目前也已在多个国家被批准用于治疗儿童GHD。在本综述中,我们将探讨索马帕西坦在成人和儿科试验中的延长方法、药代动力学(PK)和药效学(PD)、疗效及安全性结果,以及这些特性与上述其他长效生长激素有何不同。此外,还总结了索马帕西坦的给药方式以及血清胰岛素样生长因子-I(IGF-I)水平的测量时间。讨论了给药信息、漏服剂量的建议和临床指南,以及确定哪些患者适合索马帕西坦治疗,以及在治疗期间如何监测和调整剂量。