Department of Women's and Children's Health, Karolinska Institutet and Pediatric Endocrinology Unit, Karolinska University Hospital, 171 64 Solna, Sweden.
Faculty of Medicine, University of Ljubljana, and University Medical Center Ljubljana, Ljubljana 1000, Slovenia.
J Clin Endocrinol Metab. 2023 Sep 18;108(10):2569-2578. doi: 10.1210/clinem/dgad183.
Growth hormone deficiency (GHD) in children is currently treated with daily injections of GH, which can be burdensome for patients and their parents/guardians. Somapacitan is a GH derivative in development for once-weekly treatment of GHD.
This work aimed to assess the efficacy and safety of somapacitan, and associated disease/treatment burden, after 4 years of treatment and 1 year after switching to somapacitan from daily GH.
This long-term safety extension of a multicenter, controlled phase 2 trial (NCT02616562) took place at 29 sites in 11 countries. Patients were prepubertal, GH-naive children with GHD. Fifty patients completed 4 years of treatment. Patients in the pooled group received somapacitan (0.04, 0.08, 0.16 mg/kg/week) for 1 year, followed by the highest dose (0.16 mg/kg/week) for 3 years. Patients in the switched group received daily GH 0.034 mg/kg/day for 3 years, then somapacitan 0.16 mg/kg/week for 1 year. Main outcome measures were height velocity (HV), change from baseline in HV SD score (SDS), change from baseline in height SDS, disease burden, and treatment burden for patients and parents/guardians.
Changes from baseline in HV and HV SDS were similar and as expected in both groups. Observer-reported outcomes showed that patients and parents/guardians seem to have experienced a reduced treatment burden when switching from daily GH to somapacitan. Most parents/guardians (81.8%) strongly/very strongly preferred somapacitan over daily GH.
Somapacitan showed similar efficacy and safety in patients who continued somapacitan treatment and those who switched from daily GH to somapacitan. Once-weekly injections may lead to a reduced treatment burden relative to once-daily injections. A plain-language summary of this work is available.
目前，儿童生长激素缺乏症（GHD）的治疗方法是每天注射生长激素，这给患者及其父母/监护人带来了负担。Somapacitan 是一种正在开发中的 GH 衍生物，用于每周一次治疗 GHD。
本研究旨在评估 somapacitan 的疗效和安全性，以及在接受 4 年治疗和从每日 GH 转换为 somapacitan 治疗 1 年后与疾病/治疗相关的负担。
这是一项多中心、对照的 2 期临床试验（NCT02616562）的长期安全性扩展研究，在 11 个国家的 29 个地点进行。患者均为青春期前、无 GH 治疗史的 GHD 儿童。50 名患者完成了 4 年的治疗。在汇总组中，患者接受 somapacitan（0.04、0.08、0.16 mg/kg/周）治疗 1 年，然后接受最高剂量（0.16 mg/kg/周）治疗 3 年。在转换组中，患者接受每日 0.034 mg/kg/day 的 GH 治疗 3 年，然后接受 somapacitan 0.16 mg/kg/周治疗 1 年。主要观察指标是身高速度（HV）、HV 标准差评分（SDS）的基线变化、身高 SDS 的基线变化、疾病负担和患者及其父母/监护人的治疗负担。
两组患者的 HV 和 HV SDS 的基线变化均相似且符合预期。观察者报告的结果表明，患者及其父母/监护人在从每日 GH 转换为 somapacitan 治疗时似乎治疗负担有所减轻。大多数父母/监护人（81.8%）强烈/非常强烈地更喜欢 somapacitan 而不是每日 GH。
在继续接受 somapacitan 治疗的患者和从每日 GH 转换为 somapacitan 的患者中，somapacitan 显示出相似的疗效和安全性。每周一次注射可能会减轻与每日注射相关的治疗负担。本研究的通俗易懂摘要可供查阅。
