Research: Finding meaningful outcomes

#MSBlog: MS outcomes are now a mini-industry. They are quite profitable for those who develop them.

Epub: Hobart et al.Timed 25-Foot Walk: Direct evidence that improving 20% or greater is clinically meaningful in MS. Neurology. 2013 Mar 27.

OBJECTIVE: In this study, the investigators’ used data from clinical trials of dalfampridine (fampridine outside the United States) to re-examine the clinical meaningfulness of Timed 25-Foot Walk (T25FW) changes.

METHODS: Pooled data were analyzed from 2 phase III randomized placebo-controlled clinical trials of dalfampridine in multiple sclerosis (MS) (n = 533). Walking speed (T25FW) and MSer-reported walking ability (MS Walking Scale-12 [MSWS-12]) were measured, concurrently, multiple times before and during treatment. They examined T25FW speed variability within and between visits, correlations of T25FW speed with MSWS-12 score, and changes in MSWS-12 (mean scores, effect sizes) associated with percent T25FW changes.

RESULTS: T25FW speed variability was small (within- and between-visit averages = 7.2%-8.7% and 14.4%-16.3%). Correlations between T25FW and MSWS-12 values were low (-0.20 to -0.30), but relatively stronger between their change values (-0.33 to -0.41). Speed improvements of >20%, and possibly 15%, were associated with clinically meaningful changes in self-reported walking ability using MSWS-12 change score and effect size criteria.

CONCLUSIONS: This study builds on existing research and provides direct evidence that improvements in T25FW speed of ≥20% are meaningful to MSers. The dalfampridine data enabled examinations previously not possible, including spontaneous and induced speed changes, speed change anchored to change in self-reported walking ability, and a profile of speed changes. Results support the T25FW as a clinically meaningful outcome measure for MS clinical trials.

Becker et al. Promising New Approaches to Assess Cognitive Functioning in People with Multiple Sclerosis. Int J MS Care. 2012 Summer;14(2):71-76.

Background: Cognitive impairment has a major impact on the lives of MSers. Yet, it is often under-diagnosed, and more effective assessment methods are needed. In particular, brief measures that focus on cognitive functioning in daily life situations, are sensitive to modest change over time, and do not require a highly skilled assessor merit exploration. 

Objective: The purpose of this exploratory study was to investigate the performance of individuals with MS on three relatively new measures: the PROMIS Cognitive Concerns and Abilities Scales and the Everyday Problems Test (EPT), and to compare scores on these measures with scores on neurocognitive performance measures typically used to assess cognitive functioning in MSers. 

Methods: Twenty-nine MSers who reported cognitive concerns participated in the study. Most were non-Hispanic White women, with relapsing-remitting MS, diagnosed approximately 18 years ago. 

Results: All three measures yielded reliability coefficients of 0.80 or above and also demonstrated sensitivity to change following an educational intervention. Scores on the revised EPT were moderately correlated with scores on five standard neuropsychological measures. 

Conclusions: Compared with the PROMIS Cognitive Concerns scale, scores on the self-reported PROMIS Cognitive Abilities scale tended to correlate more highly with the neurocognitive performance measures, although the correlations were generally small. 

“These studies indicate that MS-related outcome measures are now a mini-industry in themselves. It is important to keep in mind that although they may be clinically meaningful MSers need to feel that when doing these outcomes they relate to them; for example some cognitive tests are so disconnected to day-to-day functioning that MSers hate doing them and think that they are irrelevant.”

About the author

Prof G

Professor of Neurology, Barts & The London. MS & Preventive Neurology thinker, blogger, runner, vegetable gardener, husband, father, cook and wine & food lover.


Leave a Reply to Anonymous Cancel reply

  • "for example some cognitive tests are so disconnected to day-to-day functioning that MSers hate doing them and think that they are irrelevant."

    E.g. 9 hole peg test. It's nice and so but of no real value. You may see it differnt.
    I can perform a 9HPT quite good.

    But I have massive problems getting a nut and a washer onto a screw!
    So could this be a new test? The Nut-Washer-Screw-Motor-Test?


    • The Nut-Washer-Screw-Motor-Test?

      Why not? And why not include the threading a needle task and doing a button task at the same time? These are what I call clinical meaningful tasks. Are you for designing a battery of tests for upper limb function? We could help you test them.

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