Kirsch Susan, Butler Gary, Jensen Lasse de Fries, Okkels Anna, Yssing Cecilie, Håkan-Bloch Jonas
Department of Pediatric Endocrinology, Hospital for Sick Children, Toronto and Oak Valley Health Hospital, Markham, Ontario, Canada.
Department of Paediatric Endocrinology, University College London Hospitals, and UCL Great Ormond Street Institute of Child Health, London, UK.
Patient Relat Outcome Meas. 2025 Jan 10;16:9-21. doi: 10.2147/PROM.S479705. eCollection 2025.
Growth hormone deficiency (GHD) causes decreased growth rate in children, resulting in short stature in childhood and adulthood. Daily subcutaneous injections with growth hormone (GH) have been standard treatment. Newer weekly GH formulations now exist. This study estimates utilities associated with GHD treatment for both people with the disease and caregivers by employing time trade-off (TTO) methodology.
Three online surveys were conducted amongst the general population in the UK and Canada. Based on a pilot, data collection was conducted in two surveys only (Survey A and Survey B). In Survey A, adults aged ≥18 years evaluated health states as if they were receiving injections themselves. In Survey B, adults with a child <15 years evaluated health states as if they were administering injections to a child. The surveys assessed device complexity, injection frequency, injection pain, needle visibility and storage possibilities.
2026 and 2028 respondents completed Survey A and Survey B, respectively. Of these, 1782 respondents and 1678 respondents were valid for inclusion. Avoiding weekly injection pain was associated with a significant utility gain of 0.030 (95% CI 0.026-0.035, p<0.001) in Survey A and 0.044 (95% CI 0.038-0.051, p<0.001) in Survey B. Additionally, less complex injection devices and lower injection frequencies had a significant impact in both Survey A (0.020, 95% CI 0.016-0.025, p<0.001; 0.009, 95% CI 0.005-0.014, p<0.001) and Survey B (0.008, 95% CI 0.002-0.014, p=0.006; 0.009, 95% CI 0.003-0.014, p=0.003).
Several aspects are associated with a significant impact on utilities for people with GHD and potential caregivers. Treatment options without injection pain, a time-consuming and complex injection process and daily injections are expected to result in higher health-related quality of life. These results may inform future economic evaluations and treatment choices.
生长激素缺乏症（GHD）会导致儿童生长速率下降，进而造成儿童期及成年期身材矮小。每日皮下注射生长激素（GH）一直是标准治疗方法。现在有了更新的每周一次的GH制剂。本研究采用时间权衡（TTO）方法，估计了该疾病患者及其护理人员与GHD治疗相关的效用值。
在英国和加拿大的普通人群中进行了三项在线调查。基于一项预试验，仅在两项调查（调查A和调查B）中进行了数据收集。在调查A中，18岁及以上的成年人评估健康状态，就好像他们自己正在接受注射一样。在调查B中，孩子年龄小于15岁的成年人评估健康状态，就好像他们正在给孩子注射一样。这些调查评估了设备复杂性、注射频率、注射疼痛、针头可见性和储存可能性。
分别有2026名和2028名受访者完成了调查A和调查B。其中，1782名受访者和1678名受访者符合纳入标准。在调查A中，避免每周注射疼痛与效用值显著增加0.030相关（95%置信区间0.026 - 0.035，p < 0.001），在调查B中为0.044（95%置信区间0.038 - 0.051，p < 0.001）。此外，注射设备不太复杂和注射频率较低在调查A（0.020，95%置信区间0.016 - 0.025，p < 0.001；0.009，95%置信区间0.005 - 0.014，p < 0.001）和调查B（0.008，95%置信区间0.002 - 0.014，p = 0.006；0.009，95%置信区间0.003 - 0.014，p = 0.003）中均有显著影响。
几个方面对GHD患者及其潜在护理人员的效用值有显著影响。没有注射疼痛、耗时且复杂的注射过程以及每日注射的治疗方案预计会带来更高的健康相关生活质量。这些结果可能为未来的经济评估和治疗选择提供参考。
