The Huntington's Disease Health Index: Initial Evaluation of a Disease-Specific Patient Reported Outcome Measure.

IF 2.1 Q3 NEUROSCIENCES
Olivia S. Brumfield, Christine Zizzi, N. Dilek, Danae Alexandrou, Alistair Glidden, S. Rosero, Jennifer S. Weinstein, J. Seabury, A. Kaat, Michael P McDermott, E. Dorsey, C. Heatwole
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引用次数: 2

Abstract

BACKGROUND When developed properly, disease-specific patient reported outcome measures have the potential to measure relevant changes in how a patient feels and functions in the context of a therapeutic trial. The Huntington's Disease Health Index (HD-HI) is a multifaceted disease-specific patient reported outcome measure (PROM) designed specifically to satisfy previously published FDA guidance for developing PROMs for product development and labeling claims. OBJECTIVE In preparation for clinical trials, we examine the validity, reliability, clinical relevance, and patient understanding of the Huntington's Disease Health Index (HD-HI). METHODS We partnered with 389 people with Huntington's disease (HD) and caregivers to identify the most relevant questions for the HD-HI. We subsequently utilized two rounds of factor analysis, cognitive interviews with fifteen individuals with HD, and test-retest reliability assessments with 25 individuals with HD to refine, evaluate, and optimize the HD-HI. Lastly, we determined the capability of the HD-HI to differentiate between groups of HD participants with high versus low total functional capacity score, prodromal versus manifest HD, and normal ambulation versus mobility impairment. RESULTS HD participants identified 13 relevant and unique symptomatic domains to be included as subscales in the HD-HI. All HD-HI subscales had a high level of internal consistency and reliability and were found by participants to have acceptable content, relevance, and usability. The total HD-HI score and each subscale score statistically differentiated between groups of HD participants with high versus low disease burden. CONCLUSION Initial evaluation of the HD-HI supports its validity and reliability as a PROM for assessing how individuals with HD feel and function.
亨廷顿舞蹈症健康指数:对特定疾病患者报告结果的初步评估。
背景如果开发得当,特定疾病患者报告的结果指标有可能衡量患者在治疗试验中的感受和功能的相关变化。亨廷顿舞蹈症健康指数(HD-HI)是一种多方面的疾病特异性患者报告结果指标(PROM),专门设计用于满足先前发布的美国食品药品监督管理局关于开发用于产品开发和标签声明的PROM的指导。目的在准备临床试验时,我们检查亨廷顿舞蹈症健康指数(HD-HI)的有效性、可靠性、临床相关性和患者理解。方法我们与389名亨廷顿舞舞蹈症患者和护理人员合作,确定与HD-HI最相关的问题。随后,我们利用两轮因素分析、对15名HD患者的认知访谈以及对25名HD患者进行的重新测试可靠性评估来完善、评估和优化HD-HI。最后,我们确定了HD-HI区分总功能能力得分高与低、前驱期HD与明显HD以及正常行走与行动障碍的HD参与者组的能力。结果参与者确定了13个相关且独特的症状域作为分量表纳入HD-HI。所有HD-HI分量表都具有高度的内部一致性和可靠性,参与者发现它们具有可接受的内容、相关性和可用性。总HD-HI评分和每个分量表评分在高疾病负担和低疾病负担的HD参与者组之间具有统计学差异。结论HD-HI的初步评估支持其作为评估HD患者感觉和功能的PROM的有效性和可靠性。
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来源期刊
CiteScore
4.80
自引率
9.70%
发文量
60
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