Department of Paediatrics, Saarland University Medical Centre, Homburg/Saar, Germany.
Medical Department, Ferring Arzneimittel GmbH, Kiel, Germany.
Horm Res Paediatr. 2018;90(6):393-406. doi: 10.1159/000496614. Epub 2019 Mar 5.
Treatment with growth hormone (GH) is standard clinical practice in children with GH deficiency (GHD) or Turner syndrome (TS). Hitherto, no long-term data on auxological outcome and safety of Zomacton® have been published. Data comparing needle-free administration (NF) and needle injection (NI) of GH are very sparse.
To analyse longitudinal auxological outcome and safety data of GH treatment-naïve patients diagnosed with GHD or TS and to compare NF and NI in a real-life setting.
Pooled auxological data and safety information from three consecutive prospective observational Zomacton® studies covering 22 years of treatment were analysed and NF was compared to NI.
The safety cohort comprised 1,595 patients who received at least one GH dose. The auxological outcome cohort comprised 856 treatment-naïve patients with follow-up data ≥12 months. Height-SDS and height velocity improved significantly during the first 3 years of treatment. Documented choice of device was available for 658 patients (NF 69.1%, NI 30.9%). NF administration was non-inferior to NI. No previously unknown safety signals occurred.
Real-life data show that treatment with Zomacton® improves auxological outcome parameters without new safety concerns. NF administration of GH represents a useful alternative to NI in children with growth disorders.
生长激素（GH）治疗是生长激素缺乏症（GHD）或特纳综合征（TS）患儿的标准临床实践。迄今为止，尚无关于 Zomacton®的长期生长结果和安全性数据。关于无针给药（NF）和 GH 针注射（NI）的比较数据非常稀少。
分析 GH 治疗初治的 GHD 或 TS 患者的纵向生长结果和安全性数据，并在真实环境中比较 NF 和 NI。
对三项连续前瞻性 Zomacton®研究的汇总生长数据和安全性信息进行分析，这些研究涵盖了 22 年的治疗时间，并对 NF 与 NI 进行了比较。
安全性队列包括接受至少一剂 GH 的 1595 名患者。生长结果队列包括 856 名治疗初治且随访时间≥12 个月的患者。在治疗的前 3 年内，身高 SDS 和身高增长速度显著改善。有 658 名患者（NF 占 69.1%，NI 占 30.9%）记录了选择设备的情况。NF 给药与 NI 相比非劣效。未出现新的未知安全性信号。
真实数据表明，Zomacton®治疗可改善生长参数，且无新的安全性担忧。对于生长障碍的儿童，GH 的 NF 给药是 NI 的一种有用替代方法。
