生长激素缺乏症儿童每周使用somapacitan的疗效、安全性和胰岛素样生长因子I: REAL4的3年结果

IF 5.3 1区 医学 Q1 ENDOCRINOLOGY & METABOLISM
Bradley S Miller, Joanne C Blair, Michael Højby Rasmussen, Jan Frystyk, Anders Krogh Lemminger, Aristides Maniatis, Jun Mori, Volker Böttcher, Ho-Seong Kim, Michel Polak, Reiko Horikawa
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引用次数: 0

摘要

目的:Somapacitan是一种长效生长激素,被批准用于每周一次治疗生长激素缺乏症(GHD)。本研究旨在评估儿童GHD患者在治疗3年后和从每日生长激素转换后2年使用somapacitan的疗效和耐受性。设计:随机,多国,开放标签,主动对照的平行组3期试验,52周主期和3年安全性延长(NCT03811535)。方法:Treatment-naïve儿童GHD随机(2:1)连续使用somapacitan (0.16 mg/kg/周;“soma/soma”组)或每日GH (Norditropin®;0.034 mg/kg/天),其次是somapacitan (0.16 mg/kg/周;“开关”集团)。结果:在200名参与者中,188人完成了3年的治疗。两组均观察到持续生长。在第156周,第104周至156周期间,soma/soma组的平均(SD)高度速度(HV)为7.4 (1.5)cm/年,而开关组为7.8 (1.4)cm/年。第156周,体细胞/体细胞组和转换组的平均高度SD评分(HSDS)分别为-0.95(0.98)和-1.08(0.93),接近双亲中位平均HSDS(-0.74)。第3年各组间的平均总胰岛素样生长因子I (IGF-I) SDS相似,均在正常范围内(-2.0至+2.0)。各组间生物活性IGF-I和生物活性IGF-I / IGF-I比值相似。Somapacitan耐受性良好,报告注射部位反应的比例较低。结论:持续的somapacitan治疗3年,以及从每日GH治疗转换后2年的持续疗效和耐受性观察。两组HSDS均接近中亲代HSDS平均值。临床试验注册:NCT03811535。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Efficacy, safety, and insulin-like growth factor I of weekly somapacitan in children with growth hormone deficiency: 3-year results from REAL4.

Objective: Somapacitan is a long-acting GH approved for once-weekly treatment of GH deficiency (GHD). This study aims to evaluate the efficacy and tolerability of somapacitan after 3 years of treatment and 2 years after switch from daily GH in children with GHD.

Design: Randomized, multi-national, open-labelled, active-controlled parallel-group phase 3 trial, with a 52-week main phase and 3-year safety extension (NCT03811535).

Methods: Treatment-naïve children with GHD were randomized (2:1) to continuous somapacitan (0.16 mg/kg/week; "soma/soma" group) or daily GH (Norditropin®; 0.034 mg/kg/day) followed by somapacitan (0.16 mg/kg/week; "switch" group).

Results: Of 200 participants, 188 completed 3 years of treatment. Sustained growth was observed in both groups. At week 156, mean (SD) height velocity (HV) between weeks 104 and 156 was 7.4 (1.5) cm/year in the soma/soma group and 7.8 (1.4) cm/year in the switch group. At week 156, the soma/soma and switch groups had reached a mean (SD) height SD score (HSDS) of -0.95 (0.98) and -1.08 (0.93), respectively, and were approaching the mean mid-parental HSDS of -0.74 (for both groups). Mean total insulin-like growth factor I (IGF-I) SDS during year 3 was similar between groups and within normal range (-2.0 to +2.0). Bioactive IGF-I and bioactive IGF-I to IGF-I ratio were similar between groups. Somapacitan was well tolerated, with low proportions reporting injection-site reactions.

Conclusions: Sustained efficacy and tolerability were observed for continuous somapacitan treatment for 3 years, and for 2 years after the switching from daily GH treatment. HSDS in both groups was approaching mean mid-parental HSDS.

Clinical trial registration: NCT03811535.

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来源期刊
European Journal of Endocrinology
European Journal of Endocrinology 医学-内分泌学与代谢
CiteScore
9.80
自引率
3.40%
发文量
354
审稿时长
1 months
期刊介绍: European Journal of Endocrinology is the official journal of the European Society of Endocrinology. Its predecessor journal is Acta Endocrinologica. The journal publishes high-quality original clinical and translational research papers and reviews in paediatric and adult endocrinology, as well as clinical practice guidelines, position statements and debates. Case reports will only be considered if they represent exceptional insights or advances in clinical endocrinology. Topics covered include, but are not limited to, Adrenal and Steroid, Bone and Mineral Metabolism, Hormones and Cancer, Pituitary and Hypothalamus, Thyroid and Reproduction. In the field of Diabetes, Obesity and Metabolism we welcome manuscripts addressing endocrine mechanisms of disease and its complications, management of obesity/diabetes in the context of other endocrine conditions, or aspects of complex disease management. Reports may encompass natural history studies, mechanistic studies, or clinical trials. Equal consideration is given to all manuscripts in English from any country.
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