Boileau Nicholas R, Stout Julie C, Paulsen Janes S, Cella David, McCormack Michael K, Nance Martha A, Frank Samuel, Lai Jin-Shei, Carlozzi Noelle E
Department of Physical Medicine and Rehabilitation, University of Michigan, Ann Arbor, MI, USA.
School of Psychological Sciences, Monash University, Clayton, Victoria, Australia.
J Huntingtons Dis. 2017;6(3):201-215. doi: 10.3233/JHD-170238.
Huntington's disease (HD), is a neurodegenerative disorder that is associated with cognitive, behavioral, and motor impairments that diminish health related quality of life (HRQOL). The HD-PRO-TRIADTM is a quality of life measure that assesses health concerns specific to individuals with HD. Preliminary psychometric characterization was limited to a convenience sample of HD participants who completed measures at home so clinician-ratings were unavailable.
The current study evaluates the reliability and validity of the HD-PRO-TRIADTM in a well-characterized sample of individuals with HD.
Four-hundred and eighty-two individuals with HD (n = 192 prodromal, n = 193 early, and n = 97 late) completed the HD-PRO-TRIADTM questionnaire. Clinician-rated assessments from the Unified Huntington Disease Rating Scales, the short Problem Behaviors Assessment, and three generic measures of HRQOL (WHODAS 2.0, RAND-12, and EQ-5D) were also examined.
Internal reliability for all domains and the total HD-PRO-TRIADTM was excellent (all Cronbach's α >0.93). Convergent and discriminant validity were supported by significant associations between the HD-PRO-TRIADTM domains, and other patient reported outcome measures as well as clinician-rated measures. Known groups validity was supported as the HD-PRO-TRIADTM differentiated between stages of the disease. Floor and ceiling effects were generally within acceptable limits. There were small effect sizes for 12-month change over time and moderate effect sizes for 24-month change over time.
Findings support excellent internal reliability, convergent and discriminant validity, known groups validity, and responsiveness to change over time. The current study supports the clinical efficacy of the HD-PRO-TRIADTM. Future research is needed to assess the test-retest reliability of this measure.
亨廷顿舞蹈症（HD）是一种神经退行性疾病，与认知、行为和运动障碍相关，这些障碍会降低健康相关生活质量（HRQOL）。HD-PRO-TRIADTM是一种生活质量测量工具，用于评估HD患者特有的健康问题。初步的心理测量特征仅限于在家中完成测量的HD参与者的便利样本，因此无法获得临床医生评分。
本研究评估HD-PRO-TRIADTM在特征明确的HD个体样本中的信度和效度。
482名HD患者（n = 192名前驱期患者，n = 193名早期患者，n = 97名晚期患者）完成了HD-PRO-TRIADTM问卷。还检查了来自统一亨廷顿病评定量表、简短问题行为评估以及三种HRQOL通用测量工具（世界卫生组织残疾评定量表2.0、兰德12项健康调查和欧洲五维度健康量表）的临床医生评分评估。
所有领域以及整个HD-PRO-TRIADTM的内部信度都非常好（所有克隆巴赫α系数>0.93）。HD-PRO-TRIADTM各领域与其他患者报告结局测量工具以及临床医生评分测量工具之间的显著关联支持了收敛效度和区分效度。已知组效度得到支持，因为HD-PRO-TRIADTM能够区分疾病阶段。地板效应和天花板效应通常在可接受范围内。12个月随时间变化的效应量较小，24个月随时间变化的效应量中等。
研究结果支持了良好的内部信度、收敛效度和区分效度、已知组效度以及对随时间变化的反应性。本研究支持HD-PRO-TRIADTM的临床疗效。未来需要进行研究以评估该测量工具的重测信度。
