Tai G, Corben L A, Yiu E M, Delatycki M B
Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Parkville, VIC, Australia.
School of Psychological Science, Faculty of Medicine, Nursing and Health Sciences, Monash University, Clayton, VIC, Australia.
Acta Neurol Scand. 2017 Jul;136(1):41-46. doi: 10.1111/ane.12693. Epub 2016 Sep 28.
The Medical Outcomes Study 36 item Short-Form Health Survey (SF-36) is one of the most commonly used patient reported outcome measure. This study aimed to examine the relationship between SF-36 version 2 (SF-36V2) summary scores and Friedreich ataxia (FRDA) clinical characteristics, and to investigate the responsiveness of the scale, in comparison with the Friedreich Ataxia Rating Scale (FARS), over 1, 2 and 3 years.
Descriptive statistics were used to examine the characteristics of the cohort at baseline and years 1, 2 and 3. Correlations between FRDA clinical characteristics and SF-36V2 summary scores were reported. Responsiveness was measured using paired t tests.
We found significant correlations between the physical component summary (PCS) of the SF-36V2 and various FRDA clinical parameters but none for the mental component summary. No significant changes in the SF-36V2 were seen over 1 or 2 years; however, PCS scores at Year 3 were significantly lower than at baseline (-3.3, SD [7.6], P=.01). FARS scores were found to be significantly greater at Years 1, 2 and 3 when compared to baseline.
Our findings suggest that despite physical decline, individuals with FRDA have relatively stable mental well-being. This study demonstrates that the SF-36V2 is unlikely to be a useful tool for identifying clinical change in FRDA therapeutic trials.
医学结局研究36项简短健康调查（SF - 36）是最常用的患者报告结局指标之一。本研究旨在探讨SF - 36第2版（SF - 36V2）的汇总得分与弗里德赖希共济失调（FRDA）临床特征之间的关系，并与弗里德赖希共济失调评定量表（FARS）相比，研究该量表在1年、2年和3年期间的反应性。
采用描述性统计分析来研究队列在基线以及第1、2和3年时的特征。报告FRDA临床特征与SF - 36V2汇总得分之间的相关性。使用配对t检验来测量反应性。
我们发现SF - 36V2的身体成分汇总（PCS）与各种FRDA临床参数之间存在显著相关性，但心理成分汇总与这些参数之间无显著相关性。在1年或2年内，SF - 36V2未见显著变化；然而，第3年的PCS得分显著低于基线（-3.3，标准差[7.6]，P = 0.01）。与基线相比，发现FARS得分在第1、2和3年时显著更高。
我们的研究结果表明，尽管身体机能下降，但FRDA患者的心理健康状况相对稳定。本研究表明，SF - 36V2不太可能成为识别FRDA治疗试验中临床变化的有用工具。
