Koledova Ekaterina, Stoyanov George, Ovbude Leroy, Davies Peter S W
Biopharma Global Medical Affairs, General Medicine and Endocrinology, Merck KGaA, Darmstadt, Germany.
Business & Decision Life Sciences, Brussels, Woluwe-Saint-Lambert, Belgium.
Endocr Connect. 2018 Aug;7(8):914-923. doi: 10.1530/EC-18-0172. Epub 2018 Jul 5.
The easypod connect observational study (ECOS) assessed treatment adherence among paediatric patients receiving growth hormone (GH) via the easypod electronic injection device.
ECOS was an open-label, observational, longitudinal study conducted in 24 countries between 2010 and 2016, enrolling children treated with GH.
The primary endpoint was the rate of treatment adherence during 5 years of follow-up. Impact of adherence on growth outcomes was assessed using Spearman's product-moment correlations.
Overall, 1190 patients had easypod data available for ≥3 months; most patients had GH deficiency (75%); 606 of these patients were GH naïve at baseline. Over the first year of monitoring, the median rate of adherence was 93.7% among patients overall and >93.0% in GH-naïve patients, irrespective of the treatment indication. Clinically meaningful improvements in growth rates were observed after 1 year of treatment across all GH indications. Adherence decreased with increasing treatment duration, but the overall median adherence rate remained high after 3 years of follow-up: 87.2% ( = 409), 75.5% after 4 years ( = 143) and 70.2% after 5 years ( = 43). Statistically significant correlations between adherence and 1-year change in height standard deviation score ( < 0.001 for patients overall) and height velocity ( < 0.001) were observed.
ECOS produced accurate, real-time adherence data in a large population of GH-treated children over 5 years of follow-up. Using the easypod connect system, physicians can potentially identify patients with inadequate adherence and poor response to treatment, enabling them to take appropriate action to help them maximise the benefits of GH treatment.
easypod connect观察性研究(ECOS)评估了通过easypod电子注射装置接受生长激素(GH)治疗的儿科患者的治疗依从性。
ECOS是一项2010年至2016年间在24个国家开展的开放标签、观察性纵向研究,纳入接受GH治疗的儿童。
主要终点是5年随访期间的治疗依从率。使用Spearman积矩相关分析依从性对生长结局的影响。
总体而言,1190例患者有≥3个月的easypod数据;大多数患者患有生长激素缺乏症(75%);其中606例患者基线时未接受过GH治疗。在监测的第一年,总体患者的依从率中位数为93.7%,未接受过GH治疗的患者中这一比例>93.0%,与治疗适应证无关。在所有GH适应证的治疗1年后,观察到生长速率有具有临床意义的改善。依从性随治疗持续时间的增加而降低,但随访3年后总体依从率中位数仍较高:409例患者为87.2%,4年后为75.5%(n = 143),5年后为70.2%(n = 43)。观察到依从性与身高标准差评分1年变化(总体患者P < 0.001)和身高增长速度(P < 0.001)之间存在统计学显著相关性。
ECOS在5年随访期间为大量接受GH治疗的儿童提供了准确的实时依从性数据。使用easypod connect系统,医生有可能识别出依从性不足和对治疗反应不佳的患者,使他们能够采取适当行动帮助这些患者最大限度地从GH治疗中获益。