scholarly journals Clinical outcome measures for progressive MS trials

2017 ◽  
Vol 23 (12) ◽  
pp. 1627-1635 ◽  
Author(s):  
Daniel Ontaneda ◽  
Jeffrey A Cohen ◽  
Maria Pia Amato
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Treatment Options ◽  
Unmet Need ◽  
Screening Tests ◽  
Future Research ◽  
Neuroprotective Effects ◽  
Patient Reported

Treatment options for progressive multiple sclerosis remain the main unmet need of the field. As the understanding of multiple sclerosis (MS) pathogenesis improves, new pathways and molecules will be tested for potential reparative, remyelinating, or neuroprotective effects. The clinical outcomes used will determine successful demonstration of beneficial treatment effects to regulatory agencies, clinicians, and persons with MS. This review focuses on clinical outcome measures including the Expanded Disability Status Scale, Multiple Sclerosis Functional Composite, and novel composite measures of disability. The paper also covers cognitive outcomes and screening tests for use in clinical trials. The growing importance of patient-reported outcomes and their suitability for clinical trials is also presented. The review aims to create consensus in regard to these topics and suggestions for future research.

scholarly journals Developing a Core Set of Outcome Measures for Behçet Disease: Report from OMERACT 2016

2017 ◽  
Vol 44 (11) ◽  
pp. 1750-1753 ◽  
Author(s):  
Gulen Hatemi ◽  
Alexa Meara ◽  
Yesim Ozguler ◽  
Haner Direskeneli ◽  
Alfred Mahr ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Working Group ◽  
Organ Involvement ◽  
Future Research ◽  
Behçet Disease ◽  
Behcet Disease ◽  
Core Set ◽  
Patient Reported ◽  
Specific Organ

Objective.The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group has been working toward developing a data-driven core set of outcome measures for use in clinical trials of Behçet’s syndrome [Behçet disease (BD)]. This paper summarizes the group’s work through OMERACT 2016, discussions during the meeting, and the future research agenda.Methods.Qualitative patient interviews were conducted among 20 patients with BD who have different types of organ involvement. A 3-round Delphi among BD experts and patients was initiated to identify domains, subdomains, and outcomes to be assessed in clinical trials of BD. The results of these studies were discussed during OMERACT 2016 and next steps were planned.Results.Patients’ perspectives and priorities were identified through qualitative interviews that identified candidate domains and subdomains for inclusion in the Delphi and characterized some shortcomings of the currently used patient-reported outcomes in BD. The first round of the Delphi was completed and several domains or subdomains were endorsed by the experts and/or the patients. Because many more items were endorsed than would be feasible to assess during a clinical trial, rating and ranking of items by physicians and patients was planned as a next critical step. The challenges of assessing specific organ system involvement was also discussed.Conclusion.The OMERACT Behçet Syndrome Working Group research program will identify core domains for assessment in BD with the goal of developing a core set of outcome measures for use in all trials of BD with the option to incorporate additional outcomes for specific organ involvement.

The Multiple Sclerosis Functional Composite: a new clinical outcome measure for multiple sclerosis trials

2002 ◽  
Vol 8 (5) ◽  
pp. 359-365 ◽  
Author(s):  
R A Rudick ◽  
G Cutter ◽  
S Reingold
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Cognitive Function ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Standard Therapy ◽  
Outcome Measure ◽  
Task Force ◽  
Multiple Sclerosis Functional Composite ◽  
Clinical Outcome Measure

With the advent and widespread use of partially effective disease modifying drug therapies for multiple sclerosis (MS), future clinical trials will undoubtedly test experimental interventions against standard therapy, or will test combinations of drugs against standard therapy. In either case, incremental progress in slowing disability progression in future MS clinical trials will require much larger sample sizes, more sensitive outcome measures, or a combination of the two. Because improved clinical outcome methods would likely accelerate progress in MS therapeutics, the National Multiple Sclerosis Society (NMSS) convened an international task force in 1994 to recommend improved clinical outcome measures. As the result of a two-year process of discussion and data analysis, the task force recommended the Multiple Sclerosis Functional Composite (MSFC) as a new clinical outcome measure for future MS trials. MSFC consists of timed tests of walking, arm function, and cognitive function, expressed as a single score along a continuous scale. The task force recommended that MSFC be included in future MS trials, and recommended a series of validation studies. Subsequent studies have provided evidence that MSFC correlates moderately with Expanded Disability Status Scale (EDSS), and that correlation is driven by strong correlations with the ambulatory function component; arm function and cognitive function correlate at lower levels with EDSS. The MSFC correlates better than EDSS with magnetic resonance imaging (MRI) variables, including brain atrophy, and shows significant correlation with patient-reported disease-related quality of life (QOL). MSFC and short-term change in MSFC correlate with future clinical and MRI status, and the strength of the correlations compares favorably with well-known cardiovascular risk factors. The studies in aggregate indicate that MSFC and MSFC change are clinically meaningful, and that MSFC has substantial advantages over alternative clinical outcome measures for MS clinical trials.

Commentary on ‘Disability outcome measures in multiple sclerosis clinical trials’

2012 ◽  
Vol 18 (12) ◽  
pp. 1718-1720 ◽  
Author(s):  
J Gareth Noble ◽  
Lisa A Osborne ◽  
Kerina H Jones ◽  
Rod M Middleton ◽  
David V Ford
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Well Being ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Health Technologies ◽  
Additional Information ◽  
Patient Reported ◽  
Disability Outcome

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 outcomes. This commentary explores these issues in relation to points raised by the Cohen et al. paper.

Digital Technology in Clinical Trials for Multiple Sclerosis: a systematic review. (Preprint)

2020 ◽  
Author(s):  
Marcello De Angelis ◽  
Luigi Lavorgna ◽  
Antonio Carotenuto ◽  
Martina Petruzzo ◽  
Roberta Lanzillo ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Digital Technology ◽  
Clinical Trial Design ◽  
Motor Rehabilitation ◽  
Robot Assisted ◽  
Future Directions ◽  
Treatment Side Effects

BACKGROUND Clinical trials in multiple sclerosis (MS) have leveraged the use of digital technology to overcome limitations in treatment and disease monitoring. OBJECTIVE To review the use of digital technology in concluded and ongoing MS clinical trials. METHODS In March 2020, we searched for “multiple sclerosis” and “trial” on pubmed.gov and clinicaltrials.gov using “app”, “digital”, “electronic”, “internet” and “mobile” as additional search words, separately. Overall, we included thirty-five studies. RESULTS Digital technology is part of clinical trial interventions to deliver psychotherapy and motor rehabilitation, with exergames, e-training, and robot-assisted exercises. Also, digital technology has become increasingly used to standardise previously existing outcome measures, with automatic acquisitions, reduced inconsistencies, and improved detection of symptoms. Some trials have been developing new patient-centred outcome measures for the detection of symptoms and of treatment side effects and adherence. CONCLUSIONS We will discuss how digital technology has been changing MS clinical trial design, and possible future directions for MS and neurology research.

scholarly journals Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Theresa M. Coles ◽  
Adrian F. Hernandez ◽  
Bryce B. Reeve ◽  
Karon Cook ◽  
Michael C. Edwards ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Contextual Factors ◽  
The United States ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Think Tank ◽  
Level Of Evidence ◽  
Patient Reported ◽  
Evidentiary Standards

Abstract Objectives There has been limited success in achieving integration of patient-reported outcomes (PROs) in clinical trials. We describe how stakeholders envision a solution to this challenge. Methods Stakeholders from academia, industry, non-profits, insurers, clinicians, and the Food and Drug Administration convened at a Think Tank meeting funded by the Duke Clinical Research Institute to discuss the challenges of incorporating PROs into clinical trials and how to address those challenges. Using examples from cardiovascular trials, this article describes a potential path forward with a focus on applications in the United States. Results Think Tank members identified one key challenge: a common understanding of the level of evidence that is necessary to support patient-reported outcome measures (PROMs) in trials. Think Tank participants discussed the possibility of creating general evidentiary standards depending upon contextual factors, but such guidelines could not be feasibly developed because many contextual factors are at play. The attendees posited that a more informative approach to PROM evidentiary standards would be to develop validity arguments akin to courtroom briefs, which would emphasize a compelling rationale (interpretation/use argument) to support a PROM within a specific context. Participants envisioned a future in which validity arguments would be publicly available via a repository, which would be indexed by contextual factors, clinical populations, and types of claims. Conclusions A publicly available repository would help stakeholders better understand what a community believes constitutes compelling support for a specific PROM in a trial. Our proposed strategy is expected to facilitate the incorporation of PROMs into cardiovascular clinical trials and trials in general.

Extended dosing of monoclonal antibodies in multiple sclerosis

2021 ◽  
pp. 135245852110657
Author(s):  
Zoé LE van Kempen ◽  
Alyssa A Toorop ◽  
Finn Sellebjerg ◽  
Gavin Giovannoni ◽  
Joep Killestein
Keyword(s):  
Multiple Sclerosis ◽  
Monoclonal Antibodies ◽  
Treatment Options ◽  
Future Research ◽  
Therapeutic Drug ◽  
Therapeutic Landscape ◽  
Anti Cd20 ◽  
Dosing Intervals ◽  
Care Costs ◽  
Review Current

Over the past two decades, treatment options for patients with multiple sclerosis (MS) have increased exponentially. In the current therapeutic landscape, “no evidence of MS disease activity” is within reach in many of our patients. Minimizing risks of complications, improving treatment convenience, and decreasing health care costs are goals that are yet to be reached. One way to optimize MS therapy is to implement personalized or extended interval dosing. Monoclonal antibodies are suitable candidates for personalized dosing (by therapeutic drug monitoring) or extended interval dosing. An increasing number of studies are performed and underway reporting on altered dosing intervals of anti-α4β1-integrin treatment (natalizumab) and anti-CD20 treatment (ocrelizumab, rituximab, and ofatumumab) in MS. In this review, current available evidence regarding personalized and extended interval dosing of monoclonal antibodies in MS is discussed with recommendations for future research and clinical practice.

scholarly journals Multidisciplinary data infrastructures in multiple sclerosis: Why they are needed and can be done!

2018 ◽  
Vol 25 (4) ◽  
pp. 500-509 ◽  
Author(s):  
Liesbet M Peeters ◽  
Caspar EP van Munster ◽  
Bart Van Wijmeersch ◽  
Robin Bruyndonckx ◽  
Ilse Lamers ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Personalized Treatment ◽  
Multidisciplinary Data ◽  
Patient Reported ◽  
Longitudinal Data Collection ◽  
Patient Profiling ◽  
Data Infrastructures

Personalized treatment is highly desirable in multiple sclerosis (MS). We believe that multidisciplinary measurements including clinical, functional and patient-reported outcome measures in combination with extensive patient profiling can enhance personalized treatment and rehabilitation strategies. We elaborate on four reasons behind this statement: (1) MS disease activity and progression are complex and multidimensional concepts in nature and thereby defy a one-size-fits-all description, (2) functioning, progression, treatment, and rehabilitation effects are interdependent and should be investigated together, (3) personalized healthcare is based on the dynamics of system biology and on technology that confirms a patient’s fundamental biology and (4) inclusion of patient-reported outcome measures can facilitate patient-relevant healthcare. We discuss currently available multidisciplinary MS data initiatives and introduce joint actions to further increase the overall success. With this topical review, we hope to drive the MS community to invest in expanding towards more multidisciplinary and longitudinal data collection.

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