Background

The easypod™ injection device allows automatic recording and transmission of adherence data from patients receiving recombinant human growth hormone (rhGH [Saizen®]) to treat growth disorders. This analysis aimed to evaluate the adherence of Saizen® administered via easypod™ in a cohort of Greek patients from the easypod™ connect observational study (ECOS).

Methods

The phase IV, open-label, multicentre, observational, and longitudinal ECOS study (EMR200104–520, NCT01363674) enrolled patients treated for a minimum of 6 months and up to 3 years. The primary endpoint was to assess the mean rate of adherence to treatment at different time points, where good adherence was defined as ≥85%. Change in height, height standard deviation score (SDS), height velocity and height velocity SDS were evaluated after 1 year of treatment as secondary endpoints, together with the impact of adherence on growth outcomes using the Spearman's product moment.

Results

Of the 180 patients enrolled, 86 were included in the analysis. The mean adherence to Saizen®, as recorded via easypod™, was high at each individual time point, and was maintained at 95.5% after 1 year of treatment. Clinically meaningful positive changes were also noted for all of the secondary endpoints (median increase in height = 7.25 cm, height SDS = 0.32, median height velocity = 7.62 cm/year and height velocity SDS = 1.65). However, no significant correlation was noted between adherence and growth outcomes.

Conclusions

rhGH replacement therapy using Saizen® with easypod™ led to full compliance to the treatment in a representative Greek population from ECOS, and provided additional insights on how the easypod™ device can assist physicians in monitoring adherence and help to optimise linear growth in paediatric patients with growth disorders.

中文翻译：

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使用 easypod™ 机电设备进行生长激素治疗的希腊儿科患者的治疗依从性和长期结果：IV 期多中心 easypod™ 连接观察性研究 (ECOS)。

背景

easypod™ 注射装置允许自动记录和传输接受重组人生长激素 (rhGH [Saizen®]) 治疗生长障碍的患者的依从性数据。该分析旨在评估来自 easypod™ 连接观察性研究 (ECOS) 的一组希腊患者中通过 easypod™ 给药的 Saizen® 的依从性。

方法

IV 期、开放标签、多中心、观察性和纵向 ECOS 研究（EMR200104-520，NCT01363674）招募了至少 6 个月至 3 年的患者。主要终点是评估不同时间点对治疗的平均依从率，其中良好的依从性定义为≥85%。身高变化、身高标准偏差评分 (SDS)、身高速度和身高速度 SDS 在治疗 1 年后作为次要终点进行评估，同时使用 Spearman 乘积矩评估依从性对生长结果的影响。

结果

在登记的 180 名患者中，86 名被纳入分析。通过 easypod™ 记录的对 Saizen® 的平均依从性在每个单独的时间点都很高，并且在治疗 1 年后保持在 95.5%。还注意到所有次要终点（身高中位数增加 = 7.25 厘米，身高 SDS = 0.32，身高中值速度 = 7.62 厘米/年和身高速度 SDS = 1.65）具有临床意义的积极变化。然而，在依从性和生长结果之间没有发现显着的相关性。

结论

使用 Saizen® 和 easypod™ 的 rhGH 替代疗法使来自 ECOS 的代表性希腊人群完全符合治疗，并提供了关于 easypod™ 设备如何帮助医生监测依从性并帮助优化儿童患者的线性生长的更多见解生长障碍。