Pediatric Endocrinology Unit, Hospital Miguel Servet, Zaragoza University, Zaragoza, Spain.
Department of Child Health, The Netherlands Organization for Applied Scientific Research (TNO), Leiden, Netherlands.
Front Endocrinol (Lausanne). 2024 Oct 4;15:1450573. doi: 10.3389/fendo.2024.1450573. eCollection 2024.
Human growth hormone (hGH) therapy in children can be administered by subcutaneous injection using either a manual non-connected device, which is a portable injection pen loaded with a pre-filled cartridge, or an electronic connected device. The electronic device is connected to a platform where adherence data is recorded and available for health care professionals (HCPs) and patient support programs. Real-world data used in the clinic, includes regular monitoring of adherence data which are shared with families during patients' visits and aim to determine the root causes of poor adherence. This study aimed to identify whether there are differences in growth during the first four years of treatment depending on the device, i.e. non-connected versus connected devices.
This retrospective study reports treatment of either GH deficiency or short stature secondary to birth size small for gestational age (SGA) in 174 pediatric patients attending Miguel Servet Hospital, Zaragoza, Spain. hGH treatment was administered with manual non-connected devices in 87 patients and 87 patients used connected devices. Height was followed for 4 years after start of hGH therapy.
In total, 57% of subjects had GHD and 43% were SGA. Height standard deviation score (HSDS) at treatment start was higher (p<0.001) in the non-connected device group compared to the connected device group. Change of HSDS in the connected device group was significantly higher in the second (+0.13), third (+0.20) and fourth (+0.23) year of treatment compared to the non-connected group after adjustment for age and HSDS at treatment start, sex, indication, dose and Tanner stages during treatment, and timing of measurements.
These results support the use of the connected device for hGH treatment of pediatric growth disorders.
在儿童中,人类生长激素(hGH)治疗可以通过皮下注射进行,使用的设备可以是手动非连接装置,即装有预填充药筒的便携式注射笔,也可以是电子连接装置。电子设备连接到一个平台,在该平台上记录依从性数据,供医疗保健专业人员(HCP)和患者支持计划使用。在临床中使用的真实世界数据包括定期监测依从性数据,这些数据在患者就诊期间与家属共享,并旨在确定依从性差的根本原因。本研究旨在确定治疗的前四年,根据设备(即非连接装置与连接装置)的不同,生长是否存在差异。
本回顾性研究报告了在西班牙萨拉戈萨 Miguel Servet 医院就诊的 174 名儿科患者中,使用手动非连接装置治疗的生长激素缺乏症或因出生体重小于胎龄(SGA)而导致的身材矮小症。87 名患者使用手动非连接装置,87 名患者使用连接装置进行 hGH 治疗。在开始 hGH 治疗后,对身高进行了 4 年的随访。
在所有患者中,57%患有 GHD,43%为 SGA。在非连接装置组中,治疗开始时的身高标准差评分(HSDS)更高(p<0.001)。在连接装置组中,调整年龄和治疗开始时的 HSDS、性别、适应证、剂量和治疗期间的 Tanner 阶段以及测量时间后,HSDS 在第二年(+0.13)、第三年(+0.20)和第四年(+0.23)的变化明显更高。
这些结果支持使用连接装置进行儿科生长障碍的 hGH 治疗。