Background:The Multiple Sclerosis Outcome Assessments Consortium (MSOAC) was formed by the National MS Society to develop improved measures of multiple sclerosis (MS)-related disability.Objectives:(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.Methods:(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.Conclusion:Considerable data exist supporting measures of the functional domains ambulation, manual dexterity, vision, and cognition. A CDISC standard for MS (http://www.cdisc.org/therapeutic#MS) was published, allowing pooling of clinical trial data. MSOAC member organizations contributed clinical data from 16 trials, including 14,370 subjects. Data from placebo-arm subjects are available to qualified researchers. This integrated, standardized dataset is being analyzed to support qualification of disability endpoints by regulatory agencies.
Upper extremity (UE) dysfunction may be present in up to ~80% of individuals with multiple sclerosis (MS), although its importance may be under-recognized relative to walking impairment, which is the hallmark symptom of MS. Upper extremity dysfunction affects independence and can impact the ability to use walking aids. Under-recognition of UE dysfunction may result in part from limited availability of performance-based and patient self-report measures that are validated for use in MS and that can be readily incorporated into clinical practice for screening and regularly scheduled assessments. In addition to the 9-Hole Peg Test, which is part of the Multiple Sclerosis Functional Composite, there are several performance-based measures that are generally used in the rehabilitation setting. These measures include the Box and Block Test, the Action Research Arm Test, the Test d'Evaluation de la performance des Membres Supérieurs des Personnes Agées, and the Jebsen-Taylor Test of Hand Function. Several of these measures were developed for use in stroke, although in contrast to stroke, which is characterized by unilateral dysfunction, UE impairment in MS is generally bilateral, and should be assessed as such. Similarly, patient-reported UE measures are available, including Disabilities of the Arm, Shoulder, and Hand (DASH) and its shorter version, QuickDASH, the Manual Ability Measure, and ABILHAND, although none has been psychometrically validated for MS. Recently, item response theory was used to develop a Neuro-QOL (Quality of Life) UE measure and a Patient-Reported Outcomes Measurement Information System UE measure; neither of these have demonstrated sensitivity to change, limiting their use for longitudinal assessment. Consequently, although work is still needed to develop and validate performance-based and patient-reported measures of UE function that are suitable for use in daily MS clinical practice, currently available UE measures can be recommended for incorporation into MS management, albeit with an understanding of their limitations.
Incremental decrements in HRQoL were observed as patients perceived greater levels of reduction in their walking speed.
This study was conducted to estimate the indirect costs and health-related quality of life (HRQoL) (utilities) of multiple sclerosis (MS) patients in the United States (US), and to determine the impact of worsening mobility on these parameters. In collaboration with the North American Research Committee on Multiple Sclerosis (NARCOMS) registry we conducted a cross-sectional study of participants who completed the biannual update and supplemental spring 2010 survey. Demographic, employment status, income, mobility impairment, and health utility data were collected from a sample of registry participants who met the study criteria and agreed to participate in the supplemental Mobility Study. Mean annual indirect costs per participant in 2011US$ and mean utilities for the population and for cohorts reporting different levels of mobility impairment were estimated. Analyses included 3,484 to 3,611 participants, based on survey completeness. Thirty-seven percent of registrants were not working or attending school and 46.7% of these reported retiring early. Indirect costs per participant per year, not including informal caregiver cost, were estimated at $30,601±31,184. The largest relative increase in indirect costs occurred at earlier mobility impairment stages, regardless of the measure used. Participants’ mean utility score (0.73±0.18) was lower than that of a similarly aged sample from the general US population (0.87). As with indirect costs, larger decrements in utility were seen at earlier mobility impairment stages. These results suggest that mobility impairment may contribute to increases in indirect costs and declines in HRQoL in MS patients.
BackgroundLimited data define what constitutes a minimal clinically important difference (MCID) on the EuroQol 5-Dimension (EQ-5D) health status index in persons with multiple sclerosis (PwMS). We sought to estimate the MCID for the EQ-5D health index in North American PwMS.MethodsPwMS completing the Patient Determined Disease Steps (PDDS) scale, 12-Item Multiple Sclerosis Walking Scale (MSWS-12) and EQ-5D as part of the North American Research Committee on Multiple Sclerosis (NARCOMS) registry’s spring 2011 update and supplemental survey were included in this retrospective, cross-sectional study. Distribution-based (standard error of measurement [SEM], 0.50 standard deviation [SD] and 0.33 SD unit) approaches were used to estimate a range of MCIDs for the EQ-5D based upon disease severity groups determined by the PDDS and MSWS-12 tertiles.ResultsA total of 3,044 participants were included. Moderately strong correlations between the EQ-5D and the PDDS and MSWS-12 were observed (Spearman’s r = -0.56 and -0.59, respectively, p < 0.0001 for both). MCID estimates based on PDDS score categories ranged from 0.065-0.158 (SEMs), 0.059-0.142 (0.50 SDs) and 0.039-0.095 (0.33 SDs). MCID estimates as measured by MSWS-12 tertile categories ranged from 0.068-0.098 (SEMs), 0.061-0.088 (0.50 SDs), and 0.041-0.059 (0.33 SDs). Across both the PDDS and tertiles of MSWS-12, MCID estimates tended to be larger as disease severity worsened. Mean weighted MCID estimates ranged from 0.05-0.084 for both the PDDS and MSWS-12 tertiles.ConclusionMCID estimates for the EQ-5D in PwMS were within the range of estimates seen for other disease states and appeared to be larger in those reporting more severe disease.
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