scholarly journals Serum neurofilament levels and patient‐reported outcomes in multiple sclerosis

2021 ◽  
Author(s):  
Kristin Galetta ◽  
Chinmay Deshpande ◽  
Brian C. Healy ◽  
Bonnie Glanz ◽  
Marina Ziehn ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Patient Reported Outcomes ◽  
Patient Reported

The impact of a recent relapse on patient-reported outcomes in subjects with multiple sclerosis

2012 ◽  
Vol 21 (10) ◽  
pp. 1677-1684 ◽  
Author(s):  
Brian C. Healy ◽  
Irene R. Degano ◽  
Ana Schreck ◽  
David Rintell ◽  
Howard Weiner ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Patient Reported Outcomes ◽  
Patient Reported ◽  
The Impact

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.

scholarly journals Effect of intermittent vs. daily calorie restriction on changes in weight and patient-reported outcomes in people with multiple sclerosis

2018 ◽  
Vol 23 ◽  
pp. 33-39 ◽  
Author(s):  
Kathryn C. Fitzgerald ◽  
Diane Vizthum ◽  
Bobbie Henry-Barron ◽  
Amy Schweitzer ◽  
Sandra D. Cassard ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Calorie Restriction ◽  
Patient Reported Outcomes ◽  
Patient Reported

scholarly journals Suggestions for improving the design of clinical trials in multiple sclerosis—results of a systematic analysis of completed phase III trials

2019 ◽  
Vol 10 (4) ◽  
pp. 425-436 ◽  
Author(s):  
Sinje Gehr ◽  
Thomas Kaiser ◽  
Reinhold Kreutz ◽  
Wolf-Dieter Ludwig ◽  
Friedemann Paul
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Patient Reported Outcomes ◽  
Progressive Multiple Sclerosis ◽  
Phase Iii ◽  
Primary Progressive Multiple Sclerosis ◽  
Systematic Overview ◽  
Patient Reported ◽  
Phase Iii Trials ◽  
Secondary Endpoints

AbstractThis manuscript reviews the primary and secondary endpoints of pivotal phase III trials with immunomodulatory drugs in multiple sclerosis (MS). Considering the limitations of previous trial designs, we propose new standards for the planning of clinical trials, taking into account latest insights into MS pathophysiology and patient-relevant aspects. Using a systematic overview of published phase III (pivotal) trials performed as part of application for drug market approval, we evaluate the following characteristics: trial duration, number of trial participants, comparators, and endpoints (primary, secondary, magnetic resonance imaging outcome, and patient-reported outcomes). From a patient perspective, the primary and secondary endpoints of clinical trials are only partially relevant. High-quality trial data pertaining to efficacy and safety that stretch beyond the time frame of pivotal trials are almost non-existent. Understanding of long-term benefits and risks of disease-modifying MS therapy is largely lacking. Concrete proposals for the trial designs of relapsing (remitting) multiple sclerosis/clinically isolated syndrome, primary progressive multiple sclerosis, and secondary progressive multiple sclerosis (e.g., study duration, mechanism of action, and choice of endpoints) are presented based on the results of the systematic overview. Given the increasing number of available immunotherapies, the therapeutic strategy in MS has shifted from a mere “relapse-prevention” approach to a personalized provision of medical care as to the choice of the appropriate drugs and their sequential application over the course of the disease. This personalized provision takes patient preferences as well as disease-related factors into consideration such as objective clinical and radiographic findings but also very burdensome symptoms such as fatigue, depression, and cognitive impairment. Future trial designs in MS will have to assign higher relevance to these patient-reported outcomes and will also have to implement surrogate measures that can serve as predictive markers for individual treatment response to new and investigational immunotherapies. This is an indispensable prerequisite to maximize the benefit of individual patients when participating in clinical trials. Moreover, such appropriate trial designs and suitable enrolment criteria that correspond to the mode of action of the study drug will facilitate targeted prevention of adverse events, thus mitigating risks for individual study participants.

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