Systematic literature review and validity evaluation of the Expanded Disability Status Scale (EDSS) and the Multiple Sclerosis Functional Composite (MSFC) in patients with multiple sclerosis.
Systematic literature review and validity evaluation of the Expanded Disability Status Scale (EDSS) and the Multiple Sclerosis Functional Composite (MSFC) in patients with multiple sclerosis.
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DOI：
10.1186/1471-2377-14-58
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发表时间：
2014-03-25
期刊：
影响因子：
2.6
通讯作者：
Kohlmann T
中科院分区：
文献类型：
--
作者：
Meyer-Moock S;Feng YS;Maeurer M;Dippel FW;Kohlmann T
有许多工具可以描述多发性硬化的严重程度和进展，它们越来越多地用作评估治疗干预有效性的终点。我们研究了两种公认的工具- EDSS和MSFC -的心理测量学特性在多大程度上符合方法学标准以及它们在临床试验中的价值。我们在相关数据库[MEDLINE（PubMed），ISI Web of Science，EMBASE，PsycINFO & PSYNDEX，CINAHL]中进行了系统的文献检索，获得了3，860个结果。首先使用摘要识别相关全文出版物，然后进行全文综述，并对文献进行综述。为了评价EDSS和MSFC的心理测量学特性（效度、可靠性、变化敏感性），确定了120篇相关全文出版物，其中54篇评估了EDSS，26篇评估了MSFC，40篇包括两种工具。EDSS在可靠性和对变化的敏感性方面有一些记录在案的弱点。MSFC的主要局限性是学习效果和用于计算总分的z分数方法。然而，有效性的方法标准充分适用于这两种工具。在临床研究中，我们发现EDSS在最近的研究中（50例EDSS，9例MSFC）被首选为主要和次要结局指标。认识到它们的优点和缺点，EDSS和MSFC都适合检测临床干预措施的有效性和监测疾病进展。几乎所有出版物都将EDSS确定为临床试验中测量疾病结局的最广泛使用的工具。尽管存在一些局限性，但两种工具均被接受为终点，并且在已识别的出版物中均未作为替代参数进行讨论。EDSS的一大优势是其作为临床试验主要终点的国际认可（例如EMA）及其在试验中的广泛使用，从而能够进行跨研究比较。
There are a number of instruments that describe severity and progression of multiple sclerosis and they are increasingly used as endpoints to assess the effectiveness of therapeutic interventions. We examined to what extent the psychometric properties of two accepted instruments – EDSS and MSFC – meet methodological standards and the value they have in clinical trials. We conducted a systematic literature search in relevant databases [MEDLINE (PubMed), ISI Web of Science, EMBASE, PsycINFO & PSYNDEX, CINAHL] yielding 3,860 results. Relevant full-text publications were identified using abstract and then full-text reviews, and the literature was reviewed. For evaluation of psychometric properties (validity, reliability, sensitivity of change) of EDSS and MSFC, 120 relevant full-text publications were identified, 54 of them assessed the EDSS, 26 the MSFC and 40 included both instruments. The EDSS has some documented weaknesses in reliability and sensitivity to change. The main limitations of the MSFC are learning effects and the z-scores method used to calculate the total score. However, the methodological criterion of validity applies sufficiently for both instruments. For use in clinical studies, we found the EDSS to be preferred as a primary and secondary outcome measure in recent studies (50 EDSS, 9 MSFC). Recognizing their strengths and weaknesses, both EDSS and MSFC are suitable to detect the effectiveness of clinical interventions and to monitor disease progression. Almost all publications identify the EDSS as the most widely used tool to measure disease outcomes in clinical trials. Despite some limitations, both instruments are accepted as endpoints and neither are discussed as surrogate parameters in identified publications. A great advantage of the EDSS is its international acceptance (e.g. by EMA) as a primary endpoint in clinical trials and its broad use in trials, enabling cross-study comparisons.
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影响因子：
--
作者：
Cohen, JA;Cutter, GR;Whitaker, JN
通讯作者：
Whitaker, JN
影响因子：
5.8
作者：
Bosma, L. V. A. E.;Kragt, J. J.;Uitdehaag, B. M. J.
通讯作者：
Uitdehaag, B. M. J.
影响因子：
--
作者：
AMATO, MP;FRATIGLIONI, L;AMADUCCI, L
通讯作者：
AMADUCCI, L
影响因子：
2.6
作者：
Barker-Collo, SL
通讯作者：
Barker-Collo, SL
DOI：
10.1191/135245899678846113
发表时间：
1999-08-01
期刊：
MULTIPLE SCLEROSIS
影响因子：
--
作者：
Amato, MP;Ponziani, G
通讯作者：
Ponziani, G