Department of Psychosomatic Medicine and Psychotherapy, Hannover Medical School, Hanover, Germany.
Division of Pediatric Endocrinology and Diabetes, Department of Paediatrics and Adolescent Medicine, University Medical Center, Ulm, Germany.
Front Endocrinol (Lausanne). 2020 Feb 25;11:84. doi: 10.3389/fendo.2020.00084. eCollection 2020.
This paper presents development and validation of a new patient reported outcome measure (PRO), the Barriers to Growth Hormone Therapy (BAR-GHT) in a patient (child/adolescent) and a parent version. The BAR-GHT was developed to measure problems and potential barriers to GHT. The development and validation of the BAR-GHT was conducted according to the Food and Drug Administration (FDA) Guidance on the development of PROs. Concept elicitation included a literature review and open-ended interviews with young patients, parents, and clinical experts. Qualitative data were analyzed based on grounded theory principles and draft items were rated in terms of their importance and clarity. The instruments underwent psychometric validation in a German clinic-based patient population of children and adolescents who inject themselves and in a parent sample who inject their child. The statistical analysis plan included exploratory factor analysis, reliability, and validity. 29 patients, 22 parents, and 4 clinical experts participated in the concept elicitation, 156 children and adolescents aged 8-18 years and 146 parents completed the validation study. Exploratory factor analysis resulted in six domains: Fear, Public Embarrassment, Annoyance, Daily Routine, Supplies, and Travel. Internal consistencies and test-retest reliabilities of the total score of both the patient version and the parent version were >0.8. Convergent and discriminant validity was demonstrated. The final 19-item BAR-GHT for patients aged 8-18 years and the 16-item version for parents can be considered reliable and valid PROs of barriers to GHT. www.ClinicalTrials.gov, identifier: NCT03672617. Universal Trial Number (UTN) of the International Clinical Trials Registry Platform (ICTRP, www.who.int): U1111-1210-1036.
本文介绍了一种新的患者报告结局测量工具（PRO）——生长激素治疗障碍量表（BAR-GHT）的开发和验证，包括患者（儿童/青少年）和家长版本。BAR-GHT 旨在测量生长激素治疗的问题和潜在障碍。BAR-GHT 的开发和验证遵循了美国食品和药物管理局（FDA）关于 PRO 开发的指南。概念启发包括对文献的回顾和对年轻患者、家长和临床专家的开放式访谈。定性数据基于扎根理论原则进行分析，并根据重要性和清晰度对草案项目进行评分。该工具在一家德国基于诊所的儿童和青少年患者群体以及为其孩子注射的家长样本中进行了心理测量学验证。统计分析计划包括探索性因素分析、信度和效度。29 名患者、22 名家长和 4 名临床专家参与了概念启发，156 名 8-18 岁的儿童和青少年以及 146 名家长完成了验证研究。探索性因素分析得出了六个领域：恐惧、公开尴尬、烦恼、日常例行、用品和旅行。患者版本和家长版本的总分的内部一致性和重测信度均>0.8。显示了收敛和判别效度。最终的 19 项 8-18 岁患者版本和 16 项家长版本的 BAR-GHT 可被视为生长激素治疗障碍的可靠和有效的 PRO。ClinicalTrials.gov，标识符：NCT03672617。国际临床试验注册平台（ICTRP，www.who.int）的通用试验编号（UTN）：U1111-1210-1036。
