Abstract
BackgroundThere 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.MethodsWe 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.ResultsFor 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).ConclusionsRecognizing 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.
Highlights
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 investigated to what extent the psychometric properties of the two most important instruments – Expanded Disability Status Scale (EDSS) and Multiple Sclerosis Functional Composite (MSFC) – meet the methodological standards and what value they have in clinical trials
3680 results were identified (4272 of the initially generated 8132 hits were excluded as duplicates); the abstracts were reviewed by two independent scientists and structured according to the following criteria: 1) Psychometric studies: validity, reliability, sensitivity of change, head-to-head comparisons, further developments of EDSS or MSFC and reviews
Summary
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. Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system that mainly affects young adults. The disease is characterized by the occurrence of relapsing neurological deficits that affect different functional systems of the central nervous system. A number of instruments have been developed that describe the clinical severity and the functional deficits in multiple sclerosis. These instruments are increasingly used as an endpoint in clinical trials to assess the effectiveness of therapeutic interventions
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
