Galen Research Ltd, Manchester, UK.
Health Qual Life Outcomes. 2010 Oct 11;8:117. doi: 10.1186/1477-7525-8-117.
The PRIMUS is a Multiple Sclerosis (MS)-specific suite of outcome measures including assessments of QoL (PRIMUS QoL, scored 0-22) and activity limitations (PRIMUS Activities, scored 0-30). The U-FIS is a measure of fatigue impact (scored 0-66). These measures have been fully validated previously using an MS sample with mixed diagnoses. The aim of the present study was to validate the measures further in a specifically Relapse Remitting MS (RRMS) sample and to provide preliminary evidence of the responder definitions (RD; also known as minimal important difference) for these instruments.
Data were derived from a multi-country efficacy trial of MS patients with assessments at baseline and 12 months. Baseline data were used to assess the internal reliability and validity of the measures. Both anchor-based and distribution-based approaches were employed for estimating RD. Anchor-based estimates were based on published RD values for the EQ-5D and were assessed for those improving and deteriorating separately. Distribution-based estimates were based on standard error of measurement (SEM), change score equivalent to 0.30, and change score equivalent to 0.50, effect sizes (ES).
The sample included 911 RRMS patients (67.3% female, age mean (SD) 36.2 (8.4) years, duration of MS mean (SD) 4.8 (5.2) years). Results showed that the PRIMUS and U-FIS had good internal consistency. Appropriate correlations were observed with comparator instruments and both measures were able to distinguish between participants based on Expanded Disability Status Scale scores and time since diagnosis. The anchor-based and distribution-based RD estimates were: PRIMUS Activities range = 1.2-2.3, PRIMUS QoL range = 1.0-2.2, and U-FIS range = 2.4-7.0.
The results show that the PRIMUS and U-FIS are valid instruments for use with RRMS patients. The analyses provide preliminary information on how to interpret scores on the scales. These data will be useful for assessing treatment efficacy and for powering clinical studies. TRIAL REFERENCE NUMBER: ClinicalTrials.gov Identifier NCT00340834.
PRIMUS 是一套多发性硬化症（MS）专用的评估工具，包括生活质量评估（PRIMUS QoL，评分范围 0-22）和活动能力受限评估（PRIMUS Activities，评分范围 0-30）。U-FIS 是衡量疲劳影响的指标（评分范围 0-66）。这些工具之前已经在混合诊断的 MS 样本中进行了充分验证。本研究的目的是进一步在特定的复发缓解型多发性硬化症（RRMS）样本中验证这些工具，并为这些工具的响应定义（RD；也称为最小有意义差异）提供初步证据。
数据来自一项多国多发性硬化症患者疗效试验，在基线和 12 个月时进行评估。使用基线数据评估这些工具的内部可靠性和有效性。采用基于锚定和基于分布的方法来估计 RD。基于已发表的 EQ-5D RD 值来进行基于锚定的估计，并分别评估那些改善和恶化的患者。基于分布的估计是基于测量误差的标准误差（SEM）、变化评分等于 0.30 和变化评分等于 0.50、效应量（ES）。
样本包括 911 名 RRMS 患者（67.3%为女性，年龄平均（SD）为 36.2（8.4）岁，多发性硬化症平均（SD）病程为 4.8（5.2）年）。结果表明，PRIMUS 和 U-FIS 具有良好的内部一致性。与对照工具观察到适当的相关性，并且这两种工具都能够根据扩展残疾状况量表评分和诊断后时间来区分参与者。基于锚定和基于分布的 RD 估计值为：PRIMUS Activities 范围=1.2-2.3，PRIMUS QoL 范围=1.0-2.2，U-FIS 范围=2.4-7.0。
结果表明，PRIMUS 和 U-FIS 是用于 RRMS 患者的有效工具。这些分析提供了有关如何解释量表得分的初步信息。这些数据将有助于评估治疗效果和为临床试验提供动力。试验参考编号：ClinicalTrials.gov 标识符 NCT00340834。
