scholarly journals Evaluation of multiple sclerosis disability outcome measures using pooled clinical trial data

Neurology ◽  
2019 ◽  
Vol 93 (21) ◽  
pp. e1921-e1931 ◽  
Author(s):  
Myla D. Goldman ◽  
Nicholas G. LaRocca ◽  
Richard A. Rudick ◽  
Lynn D. Hudson ◽  
Peter S. Chin ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Outcome Measures ◽  
Performance Measures ◽  
Performance Test ◽  
Short Form ◽  
Performance Measure ◽  
Test Battery ◽  
Measurement Properties ◽  
Sf 36 ◽  
Clinical Meaningfulness

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 differences between subgroups based on disease duration and EDSS level. Progression rates in detecting time to 3-month confirmed worsening were lower for T25FW and 9HPT compared to EDSS, while progression rates for LCLA and SDMT were similar to EDSS. When the 4 measures were analyzed as a multidimensional measure rather than as individual measures, progression on any one performance measure was more sensitive than the EDSS. Worsening on the performance measures analyzed individually or as a multidimensional test battery was associated with clinically meaningful SF-36 PCS score worsening, supporting clinical meaningfulness of designated performance test score worsening.ConclusionThese results support the use of the 4 proposed performance measures, individually or combined into a multidimensional test battery as study outcome measures.

Assessment of computer adaptive testing version of the Neuro-QOL for people with multiple sclerosis

2018 ◽  
Vol 25 (13) ◽  
pp. 1791-1799 ◽  
Author(s):  
Brian C Healy ◽  
Jonathan Zurawski ◽  
Cindy T Gonzalez ◽  
Tanuja Chitnis ◽  
Howard L Weiner ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Outcome Measures ◽  
Computerized Adaptive Testing ◽  
Short Form ◽  
Patient Reported Outcome Measures ◽  
Adaptive Testing ◽  
Patient Reported Outcome ◽  
Longitudinal Change ◽  
Sf 36 ◽  
Patient Reported

Background: To date, the computerized adaptive testing (CAT) version of the Neuro-quality of life (QOL) has not been assessed in a large sample of people with multiple sclerosis (MS). Objective: The aim of this study was to assess the associations between the CAT version of Neuro-QOL and other clinical and patient-reported outcome measures. Methods: Subjects ( n = 364) enrolled in SysteMS completed the CAT version of the Neuro-QOL and the 36-Item Short Form Survey (SF-36) within 4 weeks of a clinical exam that included the Multiple Sclerosis Functional Composite-4 (MSFC-4). The correlations between the Neuro-QOL domains and the MSFC-4 subscores and the SF-36 scores were calculated. The changes over time in the Neuro-QOL and other measures were also examined. Results: The lower extremity functioning score of the Neuro-QOL showed the highest correlations with MSFC-4 components including Timed 25-Foot Walk, 9-Hole Peg Test, and cognitive score. The expected domains of the Neuro-QOL showed high correlations with the SF-36 subscores, and some Neuro-QOL domains were associated with many SF-36 subscores. There was limited longitudinal change on the Neuro-QOL domains over 12 months, and the change was not associated with change on other measures. Conclusion: The CAT version of the Neuro-QOL shows many of the expected associations with clinical and patient-reported outcome measures.

PATIENT-REPORTED OUTCOMES AND DISABILITY IN MULTIPLE SCLEROSIS

2015 ◽  
Vol 86 (11) ◽  
pp. e4.32-e4
Author(s):  
Neil Scolding ◽  
Hongwei Wang ◽  
Yan Liu ◽  
Lawrence Steinman
Keyword(s):  
Multiple Sclerosis ◽  
Patient Reported Outcomes ◽  
Short Form ◽  
Minimum Important Difference ◽  
Point Change ◽  
Clinical Difference ◽  
Sf 36 ◽  
Patient Reported ◽  
Edss Score ◽  
Multiple Sclerosis Patients

In the 2-year, phase 3 CARE-MS II study (NCT00548405), alemtuzumab demonstrated superior clinical and patient-reported outcomes (PROs) over subcutaneous interferon beta-1a in relapsing-remitting multiple sclerosis patients who had inadequate efficacy response to prior therapy. To further evaluate the relationship between PROs and disability, Short-Form 36-Item (SF-36) survey physical component summary (PCS) and mental component summary (MCS), and Functional Assessment of Multiple Sclerosis (FAMS) scores were analysed against Expanded Disability Status Scale (EDSS) outcomes, adjusted for baseline characteristics and randomisation arm. A 1.0-point difference in baseline EDSS score was associated with 2.0-point PCS, 0.8-point MCS, and 4.0-point FAMS worsening over 12 months (all P<0.001). A 1.0-point annualised EDSS score worsening corresponded to a 2.2-point PCS, 1.6-point MCS, and 6.0-point FAMS worsening (all P<0.001). For baseline EDSS score <4.0, 1.0-point annualised worsening was associated with 7.2-point FAMS and 2.0-point MCS worsening (both P<0.001). For baseline EDSS score ≥4.0, 1.0-point worsening corresponded to worsening on FAMS (2.4 points; P=0.04), but not MCS (P=0.82). Given that a half-point EDSS change is considered the minimum reliably measurable clinical difference, a 1.0-point change in SF-36 PCS and MCS or 3.0-point change in FAMS may represent a minimum important difference in PRO for multiple sclerosis patients.

Improved patient-reported health impact of multiple sclerosis: The ENABLE study of PR-fampridine

2015 ◽  
Vol 22 (7) ◽  
pp. 944-954 ◽  
Author(s):  
Richard Macdonell ◽  
Guy Nagels ◽  
David-Axel Laplaud ◽  
Carlo Pozzilli ◽  
Brigit de Jong ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Psychological Health ◽  
Short Form ◽  
Real Life ◽  
Health Impact ◽  
The United States ◽  
Prolonged Release ◽  
Open Label ◽  
Sf 36 ◽  
Patient Reported

Background: Multiple sclerosis (MS) is a debilitating disease that negatively impacts patients’ lives. Objective: ENABLE assessed the effect of long-term prolonged-release (PR) fampridine (dalfampridine extended release in the United States) treatment on patient-perceived health impact in patients with MS with walking impairment. Methods: ENABLE was a 48-week, open-label, Phase 4 study of PR-fampridine 10 mg twice daily. Patients who showed any improvement in Timed 25-Foot Walk walking speed at weeks 2 and 4 and any improvement in 12-item MS Walking Scale score at week 4 remained on treatment. The primary endpoint was change from baseline in 36-Item Short-Form Health Survey (SF-36) physical component summary (PCS) score. Results: At week 4, 707/901 (78.5%) patients met the criteria to remain on treatment. Patients on treatment demonstrated significant and clinically meaningful improvements in SF-36 PCS scores from baseline (mean change (95% confidence interval)) to week 12 (4.30 (3.83, 4.78); p < 0.0001), week 24 (3.75 (3.23, 4.27); p < 0.0001), week 36 (3.46 (2.95, 3.97); p < 0.0001), and week 48 (3.24 (2.72, 3.77); p < 0.0001). Significant improvements from baseline were also demonstrated in secondary health measures in patients on treatment. Conclusion: PR-fampridine improved patient-perceived physical and psychological health impact of MS measured in a real-life setting.

Comparison of Quality of Life in Clinically Isolated Syndrome Patients Who Convert and Do Not Convert to Clinically Definite Multiple Sclerosis

2009 ◽  
Vol 11 (1) ◽  
pp. 17-24 ◽  
Author(s):  
Deborah M. Miller ◽  
Craig Kollman ◽  
Andrea Kalajian ◽  
Paul W. O'Connor ◽  
R. Philip Kinkel
Keyword(s):  
Quality Of Life ◽  
Multiple Sclerosis ◽  
Short Form ◽  
Clinically Isolated Syndrome ◽  
Sf 36 ◽  
Initial Symptoms ◽  
Patient Reported ◽  
Definite Multiple Sclerosis ◽  
Clinically Definite Multiple Sclerosis

A secondary analysis was undertaken to compare patient-reported outcomes (PROs) of individuals who did and did not convert to clinically definite multiple sclerosis (CDMS) approximately 5 years after their first clinically isolated syndrome (CIS). Patients included in the analysis were participating in a long-term extension (called CHAMPIONS) of the Controlled High-Risk Avonex® Multiple Sclerosis Prevention Study (CHAMPS). The Multiple Sclerosis Quality of Life Inventory (MSQLI), a battery including the Short Form Health Status Survey (SF-36) and nine disease-specific scales, was administered to participants 5 years after their initial symptoms suggestive of MS (randomization into the CHAMPS study). Of 203 CHAMPIONS patients, 188 (93%) completed the MSQLI at enrollment into this extension study. Of these, 79 (42%) converted to CDMS. Statistically significant differences (P &lt; .001) between those who did and did not convert to CDMS were found for 4 of the 11 MSQLI scales: the SF-36 Physical Component Summary, the Modified Fatigue Impact Scale, the Pain Effects Scale, and the Bladder Control Scale. Trends not meeting our criteria for statistical significance (P &gt; .001 but &lt; .01) were observed for the SF-36 Mental Component Summary, the Perceived Deficits Questionnaire, and the Mental Health Inventory. SF-36 scores for patients not converting to CDMS over 5 years were similar to those reported for age-matched normal controls. No other demographic or disease-related factors were associated with these PROs. When stratified by Expanded Disability Status Scale score, patients who converted to CDMS demonstrated statistically significant differences on the same four scales defined above that differentiated those who did and did not convert to CDMS. These data show that individuals who have CDMS but limited disability demonstrate clear evidence of diminished health-related quality of life.

Patient characteristics and determinants of quality of life in an international population with multiple sclerosis: Assessment using the MusiQoL and SF-36 questionnaires

2011 ◽  
Vol 17 (10) ◽  
pp. 1238-1249 ◽  
Author(s):  
Oscar Fernández ◽  
Karine Baumstarck-Barrau ◽  
Marie-Claude Simeoni ◽  
Pascal Auquier ◽  
Keyword(s):  
Quality Of Life ◽  
Multiple Sclerosis ◽  
Short Form ◽  
Occupational Status ◽  
Mental Component Summary Score ◽  
International Study ◽  
Summary Score ◽  
Sf 36 ◽  
Edss Score

Background: Multiple sclerosis (MS) can have psychological and socioeconomic consequences that affect patients’ health-related quality of life (HRQoL) as much as physical disability. Objective: To determine the clinical and sociodemographic factors affecting HRQoL in a large international study using the MS International QoL (MusiQoL) questionnaire. Methods: Patients aged >18 years with a diagnosis of MS for >6 months or clinically isolated syndrome (CIS) were enrolled. Sociodemographic and clinical data were recorded, and patients completed the MusiQoL and 36-item short form (SF-36) health survey questionnaires. Results: In total, 1992 patients from 15 countries were enrolled (mean [standard deviation] age: 42.3 [12.5] years; 70.5% women; 70.4% with relapsing–remitting MS). Multivariate multiple regression analyses identified lower educational level, higher Expanded Disability Status Scale (EDSS) score, cognitive impairment, being single and shorter time since last relapse as significant predictors of lower MusiQoL global index scores ( p < 0.05). Older age, female sex, higher EDSS score, shorter time since last relapse and receiving current MS treatment were significant predictors of lower SF-36 physical component summary scores ( p < 0.05). The SF-36 mental component summary score was linked to occupational status, inpatient/outpatient status, time since last relapse, and whether the patient was receiving MS treatment ( p < 0.05). Conclusion: Sociodemographic and clinical factors are linked to HRQoL in patients with MS. Interventions that affect these factors might be expected to influence HRQoL.

Validation of the Multiple Sclerosis International Quality of Life questionnaire

2007 ◽  
Vol 14 (2) ◽  
pp. 219-230 ◽  
Author(s):  
MC Simeoni ◽  
P. Auquier ◽  
O. Fernandez ◽  
P. Flachenecker ◽  
S. Stecchi ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Multiple Sclerosis ◽  
Internal Consistency ◽  
Discriminant Validity ◽  
Short Form ◽  
Life Questionnaire ◽  
Validity And Reliability ◽  
Sf 36 ◽  
Quality Of Life Scale

This study aims to validate the Multiple Sclerosis (MS) International Quality of Life (MusiQoL) questionnaire, a multi-dimensional, self-administered questionnaire, available in 14 languages, as a disease-specific quality of life scale that can be applied internationally. A total of 1992 patients with different types and severities of MS from 15 countries were recruited. At baseline and day 21 ± 7, each patient completed the MusiQoL, a symptom checklist and the short-form (SF)-36 QoL questionnaire. Neurologists also collected socio-demographic, MS history and outcome data. The database was randomly divided into two subgroups and analysed according to different patient characteristics. For each model, psychometric properties were tested and the number of items was reduced by various statistical methods. Construct validity, internal consistency, reproducibility and external consistency were also tested. Nine dimensions, explaining 71% of the total variance, were isolated. Internal consistency and reproducibility were satisfactory for all the dimensions. External validity testing revealed that dimension scores correlated significantly with all SF-36 scores, but showed discriminant validity by gender, socio-economic and health status. Significant correlations were found between activity in daily life scores and clinical indices. These results demonstrate the validity and reliability of the MusiQoL as an international scale to evaluate QoL in patients with MS. Multiple Sclerosis 2008; 14: 219—230. http://msj.sagepub.com

scholarly journals Evaluation of Epidural Analgesic Paste Components in Lumbar Decompressive Surgery

Neurosurgery ◽  
2011 ◽  
Vol 70 (2) ◽  
pp. 414-424 ◽  
Author(s):  
Roberto Jose Diaz ◽  
S. Terry Myles ◽  
R. John Hurlbert
Keyword(s):  
Postoperative Pain ◽  
Outcome Measures ◽  
Short Form ◽  
Decompressive Surgery ◽  
Secondary Outcome ◽  
Mcgill Pain Questionnaire ◽  
Analgesic Consumption ◽  
Sf 36 ◽  
Cord Injury ◽  
Discharge From Hospital

Abstract BACKGROUND: Adjuncts for pain management in lumbar decompressive surgery are needed to reduce narcotic consumption and promote early mobility. OBJECTIVE: To evaluate the efficacy and active components of a previously described epidural analgesic paste in controlling postoperative pain and facilitating early discharge from hospital after lumbar decompressive surgery. METHODS: A randomized double-blind controlled trial was conducted. Two-hundred and one patients were randomized to 1 of 4 analgesic epidural pastes at the time of lumbar spinal surgery: combination paste (morphine + methylprednisolone), steroid paste (methylprednisolone alone), morphine paste (morphine alone), or placebo. The primary outcome measures used were analgesic consumption and the McGill Pain Questionnaire (MPQ). Secondary outcome measures were: modified American Spinal Cord Injury Association (ASIA) score, Short Form 36 General Health Survey (SF-36), Aberdeen Pain Index (ABPI), time to ambulation and time to discharge from hospital. RESULTS: Administration of combination and steroid paste, but not morphine paste, resulted in a statistically significant reduction in mean pain rating index (PRI) and present pain intensity (PPI) components of the MPQ in the first 3 days after surgery. Likewise, postoperative in-patient narcotic analgesic consumption was reduced in the combination paste and steroid paste group, but not in the morphine paste group. No difference in time to ambulation or discharge, SF-36 scores, ABPI scores, or neurologic recovery was observed. CONCLUSION: An analgesic paste containing methylprednisolone acetate is effective at reducing postoperative pain after lumbar decompressive surgery. Mixing effective doses of morphine sulfate in the paste abrogates the expected analgesic effects of epidural morphine.

scholarly journals A Prospective Multicenter Study for Assessing MusiQoL Validity among Arabic-Speaking MS Patients Treated with Subcutaneous Interferon β-1a

2021 ◽  
Vol 2021 ◽  
pp. 1-7
Author(s):  
Mohammed Al Jumah ◽  
Suleiman Kojan ◽  
Raed Alroughani ◽  
Edward Cupler ◽  
Saeed Bohlega ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Short Form ◽  
Well Being ◽  
Primary Objective ◽  
Multicenter Cohort Study ◽  
Sf 36 ◽  
The Mean ◽  
Edss Score ◽  
Arabic Speaking ◽  
Over Time

Few studies examine health-related quality of life (HRQoL) in Arabic-speaking multiple sclerosis (MS) patients. However, HRQoL tools such as the Short Form-36 QoL instrument (SF-36) and the Multiple Sclerosis International QoL (MusiQoL) questionnaire have been validated in other languages. The primary objective of this study was to prospectively assess HRQoL using the MusiQoL questionnaire among Arabic-speaking MS patients treated with subcutaneous interferon (sc IFN β-1a) over 12 months, as part of a prospective, multinational, multicenter cohort study. Patients’ clinical parameters and HRQoL were assessed at baseline, 6 months, and 12 months. Changes in MusiQoL total and subdomain scores were compared using a Friedman test. Correlation between MusiQoL total score and Expanded Disability Status Score (EDSS) was also evaluated. In total, 439 patients from four Arabic-speaking countries were included. The mean age was 32.44 (±0.34) years, 71.5% were female, and 63.1% had an education level of university or above. The mean MS duration was 4.13 (±0.12) years, mean age at first attack was 27.35 (±0.26) years, and mean baseline EDSS score was 2.05 (±0.04). MusiQoL total score significantly improved at 6 months; however, this diminished at 12 months ( 65.67 ± 0.8 at baseline vs. 67.21 ± 0.79 at 6 months and 65.75 ± 0.8 at 12 months; p = 0.0015 ). Several aspects of patients’ HRQoL including activity of daily living, physical well-being, symptoms, and coping improved. Overall HRQoL measured using SF-36 remained generally unchanged over time ( p = 0.215 ). There was a statistically significant inverse relationship between change in EDSS score over time and change in overall MusiQoL score over time. In summary, findings confirm the utility of using MusiQoL for assessing changes in HRQoL during treatment with sc IFN β-1a in Arabic-speaking patients with MS.

Efficacy of Balance and Eye-Movement Exercises for Persons With Multiple Sclerosis (BEEMS)

Neurology ◽  
2018 ◽  
Vol 90 (9) ◽  
pp. e797-e807 ◽  
Author(s):  
Jeffrey R. Hebert ◽  
John R. Corboy ◽  
Timothy Vollmer ◽  
Jeri E. Forster ◽  
Margaret Schenkman
Keyword(s):  
Multiple Sclerosis ◽  
Eye Movement ◽  
Balance Control ◽  
Short Form ◽  
Controlled Trial ◽  
Rehabilitation Program ◽  
Cerebellar Lesion ◽  
Dynamic Posturography ◽  
Impact Scale ◽  
Sf 36

ObjectiveTo determine whether a multifaceted vestibular-related rehabilitation program (Balance and Eye-Movement Exercises for Persons with Multiple Sclerosis; BEEMS) improves balance in persons with MS and whether there are differences in outcomes based on brainstem/cerebellar lesion involvement.MethodsA 2-arm, examiner-blinded, stratified (involvement vs no involvement of brainstem/cerebellar structures), randomized controlled trial was implemented. Eighty-eight participants were allocated to BEEMS or no treatment control. Computerized Dynamic Posturography-Sensory Organization Test (CDP-SOT) measured balance control. The Dizziness Handicap Inventory (DHI), Modified Fatigue Impact Scale (MFIS), and Short Form-36 Health Status Questionnaire (SF-36) were also administered. Linear mixed models were used to investigate the primary and secondary aims.ResultsFrom baseline to 6 weeks, BEEMS participants experienced greater improvements compared to control participants in CDP-SOT composite (model-estimated difference in change 4.9, 95% confidence interval 1.39–8.38, p = 0.006), DHI total (−13.5, −17.7 to −7.25, p < 0.0001), MFIS total (−11.4, −15.7 to −7.0, p < 0.0001), SF-36 Mental (5.6, 2.43–8.71, p = 0.0006), and SF-36 Physical (3.5, 1.12–5.81, p = 0.004) scores and from baseline to 14 weeks in CDP-SOT composite (8.3, 4.73–11.9, p < 0.0001), DHI total (−13.9, −19.3 to −8.62, p < 0.0001), MFIS total (−12.3, −16.7 to −7.79, p < 0.0001), SF-36 Mental (3.9, 0.70–7.16, p = 0.02), and SF-36 Physical (3.2, 0.79–5.62, p = 0.01) scores. From baseline to 6 weeks, BEEMS participants with brainstem/cerebellar lesion involvement experienced greater improvements compared to those without in CDP-SOT composite (5.26, 0.34–10.2, p = 0.04) and MFIS total (−7.6, −14.0 to −1.33, p = 0.02) scores.ConclusionBEEMS improved multiple outcomes regardless of whether brainstem/cerebellar lesions were present, supporting the generalizability of BEEMS for ambulatory people with MS who have at least minimally impaired balance and fatigue.Clinical trials.gov identifierNCT01698086.Classification of evidenceThis study provides Class I evidence that BEEMS training improves dynamic posturography-based balance, dizziness, fatigue, and quality of life in persons with MS.

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