BACKGROUND In 2007, Omnitrope® was the first biosimilar recombinant human growth hormone (rhGH) to be approved in Sweden for treatment in adults and children. Over 10 years' safety and effectiveness data for biosimilar rhGH can now be presented. METHODS PATRO Children and PATRO Adults are multicenter, longitudinal, observational, post-marketing surveillance studies. Eligible patients include children 0-18 years and adults receiving biosimilar rhGH treatment. Adverse events (AEs) are monitored for safety evaluation. Growth variables in children and metabolic data in adults are recorded for effectiveness evaluation. RESULTS As of January 2019, data from 136 children (48% male) were reported from Swedish centers. Mean age in rhGH treatment-naïve patients at study entry (n = 114) was 7.5 years, with mean 3.6 years treatment duration. No severe AEs of diabetes, impaired glucose tolerance, or malignancy were reported. The most frequently reported AE was nasopharyngitis (n = 16 patients). No clinically relevant anti-hGH or neutralizing antibodies were observed. The mean change from baseline in height standard deviation score (SDS) in naïve prepubertal GH deficiency patients was + 0.79 at 1 year, + 1.27 at 2 years, and + 1.55 at 3 years. Data from 293 adults (44% rhGH-naïve, 51% male) were included. Fatigue was the most frequently reported AE (n = 26 patients). The incidence of new neoplasms or existing neoplasm progression was 23.8 patients per 1000 patient-years. Type 2 diabetes mellitus was reported in four patients. At baseline in rhGH-naïve adults, mean (SD) body mass index (BMI) was 29.1 (5.6) kg/m2 and mean (SD) insulin-like growth factor (IGF)-I SDS was - 3.0 (1.4). Mean daily dose increased from 0.1 mg at baseline to 0.3 mg after 4 years. IGF-I SDS normalized during the first year of treatment. Mean BMI and glucose were unchanged over 4 years, while low-/high-density lipoprotein cholesterol ratio decreased. CONCLUSIONS For the first time, Swedish data from the PATRO Children and Adults studies are presented. The 10-year data suggest that biosimilar rhGH is well tolerated across pediatric and adult indications. Safety and effectiveness were similar to previous reports for other rhGH preparations. These results need to be confirmed in larger cohorts, highlighting the importance of long-term post-marketing studies.

中文翻译：

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在瑞典的临床实践中使用生物仿制药rhGH有十年的经验-来自未来的PATRO儿童和成人研究的经验。

背景技术2007年，Omnitrope®是第一个在瑞典获准用于成人和儿童治疗的生物仿制药重组人类生长激素（rhGH）。现在可以提供生物仿制rhGH超过10年的安全性和有效性数据。方法PATRO儿童和PATRO成人是多中心，纵向，观察性，售后监测研究。符合条件的患者包括接受生物仿制rhGH治疗的0-18岁儿童和成人。监视不良事件（AE）以进行安全性评估。记录儿童的生长变量和成人的代谢数据以进行有效性评估。结果截至2019年1月，瑞典中心报告了136名儿童（男48％）的数据。初次接受rhGH治疗的患者的平均年龄（n = 114）为7.5岁，平均治疗时间为3.6年。没有糖尿病的严重不良事件，葡萄糖耐量降低或恶性肿瘤的报道。报告最频繁的AE是鼻咽炎（n = 16例）。没有观察到临床相关的抗hGH或中和抗体。青春期前GH缺乏症患者身高标准偏差评分（SDS）与基线的平均变化在1年时为+ 0.79，在2年时为+ 1.27，在3年时为+ 1.55。包括来自293位成年人（未接受过rhGH的44％，男性的51％）的数据。疲劳是最常报告的不良事件（n = 26例）。每1000病人-年的新肿瘤或现有肿瘤进展的发生率为23.8例。据报道有4名患者患有2型糖尿病。初次使用rhGH的成年人在基线时，平均（SD）体重指数（BMI）为29.1（5.6）kg / m2，而平均（SD）胰岛素样生长因子（IGF）-I SDS为-3.0（1.4）。平均日剂量从基线时的0.1 mg增加到4年后的0.3 mg。在治疗的第一年，IGF-I SDS恢复正常。平均BMI和葡萄糖在4年内没有变化，而低/高密度脂蛋白胆固醇比率下降。结论首次展示了PATRO儿童和成人研究的瑞典数据。十年的数据表明，小儿和成人适应症对生物仿制药rhGH的耐受性良好。安全性和有效性与其他rhGH制剂的先前报道相似。这些结果需要在更大的队列中得到证实，突出了长期售后研究的重要性。低/高密度脂蛋白胆固醇比率降低。结论首次展示了PATRO儿童和成人研究的瑞典数据。十年的数据表明，小儿和成人适应症对生物仿制药rhGH的耐受性良好。安全性和有效性与其他rhGH制剂的先前报道相似。这些结果需要在更大的队列中得到证实，突出了长期售后研究的重要性。低/高密度脂蛋白胆固醇比率降低。结论首次展示了PATRO儿童和成人研究的瑞典数据。十年的数据表明，小儿和成人适应症对生物仿制药rhGH的耐受性良好。安全性和有效性与其他rhGH制剂的先前报道相似。这些结果需要在更大的队列中得到证实，突出了长期售后研究的重要性。