Gualtierotti Roberta, Ingegnoli Francesca, Scalone Luciana, Cortesi Paolo, Bruschi Eleonora, Gerosa Maria, Meroni Pier Luigi
Division of Rheumatology, G. Pini Institute, Milan, Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy.
Research Centre on Public Health (CESP), University of Milan Bicocca, Milan, Italy; Fondazione CHARTA, Milan, Italy.
Swiss Med Wkly. 2016 Dec 19;146:w14394. doi: 10.4414/smw.2016.14394. eCollection 2016.
Systemic sclerosis is a chronic disabling disease that is often associated with severe physical and psychological impairment. Nonetheless, health-related quality of life (HRQoL) in patients with systemic sclerosis is often left behind in clinical practice and research. One of the reasons for this lack of evaluation is the current use of tools, such as the short form-36 (SF-36) questionnaire, that are complete but complicated to use in everyday routine. Other self-reported outcome measures such as the health assessment questionnaire (HAQ) are simple, but specifically designed for physical disability.
Our aim was to evaluate the feasibility, acceptability and construct validity of EQ-5D, a simple and quick self-assessment tool, and to compare its performance with SF-36 and HAQ. We investigated 119 consecutive patients with systemic sclerosis (94% female; age: median 63 years, interquartile range 53-70 years) at three different rheumatology centres. Acceptability was evaluated from comments made by the patients and feasibility on the basis of the number of patients needing assistance or not answering questions (missing data). Construct validity was based on both convergent and divergent validity between conceptually similar and dissimilar domains, respectively, of the compared instruments.
EQ-5D was well accepted by patients. The percentage of patients missing data in at least one EQ-5D domain was 2.5%. Spearman's correlation coefficients between similar dimensions of EQ-5D vs SF-36 and vs HAQ were moderate (≥0.30) to strong (≥0.50); in contrast, correlation coefficients between less comparable dimensions were weak. As expected, the EQ-5D anxiety/depression domain did not correlate with any of the HAQ domains. The EQ-5D visual analogue scale (VAS) concordance with SF-36 general health domain and HAQ total score was strong (≥0.50 for both). Median value for the EQ-5D index (interquartile range) was 0.81 (0.75-0.86). The EQ-5D index had correlation coefficients >0.40 with all SF-36 domains and with all HAQ domains, HAQ total and HAQ VAS.
Our data demonstrate good acceptability, feasibility and construct validity of EQ-5D in patients with systemic sclerosis. We suggest the use of EQ-5D in systemic sclerosis patients as an HRQoL measure in clinical practice, in randomised clinical trials and/or in pharmacoeconomic evaluations.
系统性硬化症是一种慢性致残性疾病，常伴有严重的身体和心理损害。尽管如此，系统性硬化症患者的健康相关生活质量（HRQoL）在临床实践和研究中常常被忽视。缺乏评估的原因之一是目前使用的工具，如简短健康调查问卷（SF-36），虽然完整但在日常使用中较为复杂。其他自我报告的结局指标，如健康评估问卷（HAQ），则较为简单，但专门针对身体残疾设计。
我们的目的是评估一种简单快速的自我评估工具EQ-5D的可行性、可接受性和结构效度，并将其性能与SF-36和HAQ进行比较。我们在三个不同的风湿病中心对119例连续性系统性硬化症患者进行了调查（94%为女性；年龄：中位数63岁，四分位间距53 - 70岁）。根据患者的反馈评估可接受性，根据需要协助或未回答问题（缺失数据）的患者数量评估可行性。结构效度分别基于所比较工具在概念上相似和不同领域之间的收敛效度和发散效度。
EQ-5D被患者广泛接受。在至少一个EQ-5D领域中缺失数据的患者比例为2.5%。EQ-5D与SF-36以及与HAQ相似维度之间的斯皮尔曼相关系数为中度（≥0.30）至高度（≥0.50）；相比之下，可比性较低维度之间的相关系数较弱。正如预期的那样，EQ-5D焦虑/抑郁领域与HAQ的任何领域均无相关性。EQ-5D视觉模拟量表（VAS）与SF-36总体健康领域以及HAQ总分的一致性很强（两者均≥0.50）。EQ-5D指数的中位数（四分位间距）为0.81（0.75 - 0.86）。EQ-5D指数与所有SF-36领域以及所有HAQ领域、HAQ总分和HAQ VAS的相关系数均>0.40。
我们的数据表明EQ-5D在系统性硬化症患者中具有良好的可接受性、可行性和结构效度。我们建议在临床实践、随机临床试验和/或药物经济学评估中，将EQ-5D用作系统性硬化症患者HRQoL的测量工具。
