Introduction: PATRO Children is an international, observational, postmarketing surveillance study for a biosimilar recombinant human growth hormone (rhGH; somatropin, Omnitrope®; Sandoz), approved by the European Medicines Agency in 2006. We report safety and effectiveness data for patients with Turner syndrome (TS). Methods: The study population included infants, children, and adolescents with TS who received Omnitrope® treatment according to standard clinical practice. Adverse events (AEs) were monitored for safety evaluation, and height velocity (HV), height standard deviation score (HSDS), and HVSDS were calculated to evaluate treatment effectiveness. Results: As of August 2019, 348 TS patients were enrolled from 130 centers. At baseline, 314 patients (90.2%) were prepubertal and 284 patients (81.6%) were rhGH treatment naïve. The mean (range) age at baseline was 9.0 (0.7–18.5) years, and mean (SD) treatment duration in the study was 38.5 (26.8) months. Overall, 170 patients (48.9%) reported AEs, which were considered treatment related in 25 patients (7.2%). One treatment-related serious AE was reported (intracranial hypertension). Mean ΔHSDS after 3 years of therapy was +1.17 in treatment-naïve prepubertal patients and +0.1 in pretreated prepubertal patients. In total, 51 patients (31.1%) reached adult height (AH), 35 of whom were rhGH treatment naïve; in these patients, mean (SD) HSDS was −2.97 (1.03) at the start of Omnitrope® treatment, and they achieved a mean (SD) AHSDS of −2.02 (0.9). Conclusion: These data suggest that biosimilar rhGH is well tolerated and effective in TS patients managed in real-life clinical practice. Optimization of rhGH dose may contribute to a higher AH.
Horm Res Paediatr

中文翻译：

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重组人类生长激素在特纳综合征儿童中的安全性和有效性：来自 PATRO 儿童研究的数据

简介： PATRO Children 是一项针对生物仿制药重组人类生长激素（rhGH；somatropin，Omnitrope®；Sandoz）的国际观察性上市后监测研究，于 2006 年获得欧洲药品管理局批准。我们报告了 Turner 患者的安全性和有效性数据综合征（TS）。方法：研究人群包括根据标准临床实践接受 Omnitrope® 治疗的 TS 婴儿、儿童和青少年。监测不良事件（AEs）以进行安全性评估，并计算身高速度（HV）、身高标准差评分（HSDS）和HVSDS以评估治疗效果。结果：截至 2019 年 8 月，来自 130 个中心的 348 名 TS 患者入组。基线时，314 名患者 (90.2%) 处于青春期前，284 名患者 (81.6%) 未接受过 rhGH 治疗。基线的平均（范围）年龄为 9.0（0.7-18.5）岁，研究中的平均（SD）治疗持续时间为 38.5（26.8）个月。总体而言，170 名患者 (48.9%) 报告了 AE，在 25 名患者 (7.2%) 中被认为与治疗相关。报告了一种与治疗相关的严重 AE（颅内高压）。治疗 3 年后的平均 ΔHSDS 在未接受过治疗的青春期前患者中为 +1.17，在接受过治疗的青春期前患者中为 +0.1。总共有 51 名患者 (31.1%) 达到成人身高 (AH)，其中 35 名患者未接受过 rhGH 治疗；在这些患者中，在 Omnitrope® 治疗开始时，平均 (SD) HSDS 为 -2.97 (1.03)，他们的平均 (SD) AHSDS 为 -2.02 (0.9)。结论：这些数据表明，生物仿制药 rhGH 在实际临床实践中管理的 TS 患者中具有良好的耐受性和有效性。优化 rhGH 剂量可能有助于提高 AH。
儿科荷尔蒙