The Brod Group (M.B.), Mill Valley, California 94941; and Department of Global Development (L.H., J.E.A., M.H.R.), Novo Nordisk A/S, DK-2860 Søborg, Denmark.
J Clin Endocrinol Metab. 2014 Apr;99(4):1204-12. doi: 10.1210/jc.2013-3438. Epub 2014 Jan 17.
Approximately 50 000 adults in the United States are diagnosed with GH deficiency, which has negative impacts on cognitive functioning, psychological well-being, and quality of life.
This paper presents development and validation of a patient-reported outcome measure (PRO), the Treatment-Related Impact Measure-Adult Growth Hormone Deficiency (TRIM-AGHD). The TRIM-AGHD was developed to measure the impact of GH deficiency and its treatment.
The development and validation of the TRIM-AGHD was conducted according to the Food and Drug Administration guidance on the development of PROs. Concept elicitation, conducted in three countries included interviews with patients, clinical experts, and literature review. Qualitative data were analyzed based on grounded theory principles, and draft items were cognitively debriefed. The measure underwent psychometric validation in a US clinic-based population. An a priori statistical analysis plan included assessment of the measurement model, reliability, and validity. Item functioning was reviewed using item response theory analyses.
Forty-eight patients and six clinical experts participated in concept elicitation and 169 patients completed the validation study.
TRIM-AGHD was measured.
Factor analysis resulted in four domains: energy level, physical health, emotional health, and cognitive ability. The item response theory confirmed adequate item fit and placement within their domain. Internal consistency ranged from 0.82 to 0.95 and test-retest ranged from 0.80 to 0.92. All prespecified hypotheses for convergent validity and all but two for discriminant validity were met.
The final 26-item TRIM-AGHD can be considered a reliable and valid PRO of the impact of disease and treatment for adult GH deficiency.
大约有 50000 名美国成年人被诊断患有生长激素缺乏症，这对认知功能、心理健康和生活质量都有负面影响。
本文介绍了一种患者报告的结局测量工具（PRO），即治疗相关影响测量成人生长激素缺乏症（TRIM-AGHD）的开发和验证。TRIM-AGHD 旨在测量生长激素缺乏症及其治疗的影响。
根据食品和药物管理局关于 PRO 开发的指导原则，进行了 TRIM-AGHD 的开发和验证。概念发掘在三个国家进行，包括对患者、临床专家和文献的访谈。基于扎根理论原则对定性数据进行了分析，并对草案项目进行了认知审查。该测量工具在美国基于诊所的人群中进行了心理测量学验证。一个预先设定的统计分析计划包括评估测量模型、可靠性和有效性。使用项目反应理论分析审查项目功能。
48 名患者和 6 名临床专家参与了概念发掘，169 名患者完成了验证研究。
测量了 TRIM-AGHD。
因子分析产生了四个领域：能量水平、身体健康、心理健康和认知能力。项目反应理论证实了项目在其领域内的适当拟合和位置。内部一致性范围为 0.82 至 0.95，测试-重测范围为 0.80 至 0.92。所有预设的关于收敛有效性的假设和除两个关于判别有效性的假设外，其他均得到满足。
最终的 26 项 TRIM-AGHD 可被视为成人生长激素缺乏症疾病和治疗影响的可靠和有效的 PRO。
