Measuring upper limb function in MS: Which existing patient reported outcomes are fit for purpose? (original) (raw)
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Commentary on 'Disability outcome measures in multiple sclerosis clinical trials
Multiple Sclerosis Journal, 2012
In order to fully understand and explore the effectiveness of any intervention for the management of multiple sclerosis (MS), it is important to have robust, valid, reliable, and universally applied measures. The recent article, ‘Disability outcome measures in multiple sclerosis clinical trials’ by Cohen, Reingold, Polman and Wolinsky (2012), explores this issue in regards to the effective measurement of MS-related disability, and the utilisation of patient-reported outcome measures, whilst highlighting the need for collaboration between the academic and clinical communities. Although it is important to examine disability measures, it is also equally important to recognise that physical function is only one aspect of a person’s experience; for example, quality of life and psychological well-being are also important aspects to assess. The application of e-health technologies and patient registers could be a useful method of gaining additional information, using patient-reported outco...
Recommendations from the national multiple sclerosis society clinical outcomes assessment task force
Annals of Neurology, 1997
This article provides recommendations from the National Multiple Sclerosis Society's Clinical Outcomes Assessment Task Force. The Task Force was appointed in 1994 and charged with recommendending improved approaches for clinical outcomes assessment in future controlled clinical trials. The recommendations herein follow extensive deliberation and data analysis during 2.5 years. General principles and desirable measurement attributes were used to assess alternative measurement techniques and clinical scales. On the basis of the analysis of existing multiple sclerosis (MS) data sets, a new measurement approach is proposed. The approach is based on quantitative functional composites that consist of simple quantitative measures from the major clinical dimensions of MS combined into a single score. Quantitative functional composites are likely to provide improved precision and sensitivity in future MS clinical trials. Studies necessary to further refine quantitative functional composites as useful MS clinical trial outcomes are delineated.
PLOS ONE, 2021
Background Multiple Sclerosis (MS) is associated with impaired gait and a growing number of clinical trials have investigated efficacy of various interventions. Choice of outcome measures is crucial in determining efficiency of interventions. However, it remains unclear whether there is consensus on which outcome measures to use in gait intervention studies in MS. Objective We aimed to identify the commonly selected outcome measures in randomized controlled trials (RCTs) on gait rehabilitation interventions in people with MS. Additional aims were to identify which of the domains of the International Classification of Functioning, Disability and Health (ICF) are the most studied and to characterize how outcome measures are combined and adapted to MS severity. Methods Pubmed, Cochrane Central, Embase and Scopus databases were searched for RCT studies on gait interventions in people living with MS according to PRISMA guidelines. Results In 46 RCTs, we identified 69 different outcome me...
Evaluation of multiple sclerosis disability outcome measures using pooled clinical trial data
Neurology
ObjectiveWe report analyses of a pooled database by the Multiple Sclerosis Outcome Assessments Consortium to evaluate 4 proposed components of a multidimensional test battery.MethodsStandardized data on 12,776 participants, comprising demographics, multiple sclerosis disease characteristics, Expanded Disability Status Scale (EDSS) score, performance measures, and Short Form–36 Physical Component Summary (SF-36 PCS), were pooled from control and treatment arms of 14 clinical trials. Analyses of Timed 25-Foot Walk (T25FW), 9-Hole Peg Test (9HPT), Low Contrast Letter Acuity (LCLA), and Symbol Digit Modalities Test (SDMT) included measurement properties; construct, convergent, and known group validity; and longitudinal performance of the measures individually and when combined into a multidimensional test battery relative to the EDSS and SF-36 to determine sensitivity and clinical meaningfulness.ResultsThe performance measures had excellent test–retest reliability and showed expected di...
Methods and measures: what's new for MS?
Multiple Sclerosis Journal, 2013
At no other time in the history of multiple sclerosis (MS) has the accurate measurement of health outcomes been so important. There are now many kinds of interventions of proven or potential efficacy available for people with MS and many other methods are under investigation. Not all outcomes that matter can be measured with a biological parameter. Many important outcomes of treatment can be assessed only by asking the patient directly. For clinical decision making, asking one good question, asking it consistently, and writing down the answer will produce historically accurate data to judge MS progression on life-altering constructs like fatigue, depression and pain. To get a total score from items in a questionnaire, Rasch Measurement Theory provides a way of estimating the extent to which the items form a linear continuum with mathematical properties. Preference-based measures, when the preferences are derived from patients, permit the impact of the multiple health dimensions associated with MS to be valued. The bottom line is, ask a good question and you will likely get a good answer, ask a poor question and assuredly, you will not.
Journal of Neurology, Neurosurgery & Psychiatry, 2000
Objectives-Understanding the properties of an outcome measure is essential in choosing the appropriate instrument and interpreting the information it generates. The MOS 36 item short form health survey questionnaire (SF-36) is widely acknowledged as the gold standard generic measure of health status; few studies however have evaluated its use for clinical trials in multiple sclerosis. Its clinical appropriateness, internal consistency reliability, validity, and responsiveness was investigated across a broad range of patients with multiple sclerosis. Methods-A prospective study in which 150 adults with clinically definite multiple sclerosis completed a battery of questionnaires evaluating generic health status, disability, handicap, and emotional wellbeing. Of these, 44 patients undergoing inpatient rehabilitation completed the questionnaires before and after intervention to evaluate responsiveness. Results-Score distributions demonstrated significant floor and ceiling eVects in four of the eight dimensions which were particularly marked when patient selection was restricted to a narrow band of disease severity (as is the case in most clinical trials). Internal consistency exceeded the standard for group comparisons for all dimensions. Convergent and discriminant construct validity was supported by the direction, magnitude, and pattern of correlations with other health measures. In comparison with instruments measuring associated constructs, the responsiveness of the SF-36 was poor in evaluating change in moderate to severely disabled patients participating in a programme of inpatient rehabilitation. Conclusions-The SF-36 has some limitations as an outcome measure in multiple sclerosis. The results highlight the need for all instruments to be examined in the specific sample population under question and for the specific research question being investigated. In multiple sclerosis clinical trials, the SF-36 should be supplemented with other relevant measures.
Disability and Rehabilitation, 2015
Impaired manual dexterity is frequent in multiple sclerosis (MS) interfering with activities of daily living (ADL) and quality of life (QoL). 1,2 Therefore, manual dexterity should be routinely evaluated in the daily care of patients with MS as well as in clinical trials, which implies the need for valid, reliable, and convenient measurement methods. The Arm Function in Multiple Sclerosis Questionnaire (AMSQ) is the first patient-reported outcome (PRO) measure specifically developed to evaluate manual dexterity in MS. It was developed in Dutch showing good validity and reliability. 3,4 The aim of this study was to develop the German version of the AMSQ and to evaluate its psychometric properties in assessing manual dexterity and its impact on ADL and QoL. To do so, the AMSQ was correlated to performance-based tests and PROs evaluating similar constructs. Test-retest reliability was assessed as well. We hypothesized that the measurement properties of the German version are similar to the Dutch version of the AMSQ. 3,4
Validity of the TEMPA for the measurement of upper limb function in multiple sclerosis
Clinical Rehabilitation, 2002
To investigate concurrent and construct validity of the TEMPA (Test d'Evaluation de la performance des Membres Supérieurs des Personnes Âgées) in patients with upper limb dysfunction due to multiple sclerosis. Subjects: Forty-three patients with upper limb dysfunction due to multiple sclerosis. Design: Patients performed upper limb tests and were assessed on measures of functional independence. Setting: National Multiple Sclerosis Centre, Melsbroek, Belgium. Main outcome measures: TEMPA, Jebsen Handfunction Test, Nine Hole Peg Test, Functional Independence Measure (FIM) and activities of daily living (ADL) self-questionnaire. Results: The correlation between TEMPA and Jebsen Handfunction and the Nine Hole Peg test respectively is good (0.56-0.87) and high (0.79-0.9). The correlation between TEMPA and the FIM and ADL self-questionnaire was moderate (0.44-0.61) although mostly higher than the correlation between the Jebsen Handfunction and the Nine Hole Peg test respectively and the measures of functionality (0.22-0.55/0.1-0.47). Conclusions: This study provides data supporting the concurrent validity of the TEMPA with the Jebsen Handfunction and the Nine Hole Peg Test in patients with multiple sclerosis. The results suggest construct validity of the TEMPA for the measurement of functionality during activities of daily life in patients with upper limb dysfunction due to multiple sclerosis.
Evolution of Patient-Reported Outcomes and Their Role in Multiple Sclerosis Clinical Trials
Neurotherapeutics
Patient-reported outcomes (PROs) are playing an increasing role in multiple sclerosis (MS) research and practice, and are essential for understanding the effects that MS and MS treatments have on patients' lives. PROs are captured directly from patients and include symptoms, function, health status, and health-related quality of life. In this article, we review different categories (e.g., generic, targeted, preference-based) of PRO measures and considerations in selecting a measure. The PROs included in MS clinical research have evolved over time, as have the measures used to assess them. We describe findings from recent MS clinical trials that included PROs when evaluating Food and Drug Administration-approved disease-modifying therapies (e.g., daclizumab, teriflunomide). Variation in the measures used in these trials makes it difficult to draw any conclusions from the data. We therefore suggest a standardized approach to PRO assessment in MS research and describe 2 generic, National Institutes of Health-supported measurement systems [Neuro-QoL and the Patient-Reported Outcomes Measurement Information System (PROMIS)] that would facilitate such an approach. The use of PROs in MS care and research is expanding beyond clinical trials, as is demonstrated by examples from comparative effectiveness and other patientcentered research. The importance of PRO assessment is expected to continue to grow in the future.
Multiple sclerosis (Houndmills, Basingstoke, England), 2017
The Multiple Sclerosis Outcome Assessments Consortium (MSOAC) was formed by the National MS Society to develop improved measures of multiple sclerosis (MS)-related disability. (1) To assess the current literature and available data on functional performance outcome measures (PerfOs) and (2) to determine suitability of using PerfOs to quantify MS disability in MS clinical trials. (1) Identify disability dimensions common in MS; (2) conduct a comprehensive literature review of measures for those dimensions; (3) develop an MS Clinical Data Interchange Standards Consortium (CDISC) data standard; (4) create a database of standardized, pooled clinical trial data; (5) analyze the pooled data to assess psychometric properties of candidate measures; and (6) work with regulatory agencies to use the measures as primary or secondary outcomes in MS clinical trials. Considerable data exist supporting measures of the functional domains ambulation, manual dexterity, vision, and cognition. A CDISC s...