The MS Center for Innovations in Care, Missouri Baptist Medical Center

Author Of 2 Presentations

Clinical Trials Poster Presentation

P0205 - Effects of Diroximel Fumarate on Brain Volume Change and Disability Progression in Adults With Relapsing-Remitting Multiple Sclerosis From EVOLVE-MS-1 (ID 434)

Speakers
Presentation Number
P0205
Presentation Topic
Clinical Trials

Abstract

Background

Diroximel fumarate (DRF) is a novel oral fumarate approved in the United States for relapsing forms of multiple sclerosis (MS). DRF undergoes pre-systemic hydrolysis to monomethyl fumarate (MMF), the same pharmacologically active metabolite as dimethyl fumarate (DMF). DRF 462 mg and DMF 240 mg produce bioequivalent exposure of MMF and are expected to have similar efficacy and safety profiles. In DMF-treated patients, annual rates of brain volume loss over 6 years ranged from -0.19 to -0.37, approaching rates observed in healthy adults (-0.1% to -0.3%).

Objectives

To report percent brain volume change (PBVC) and impact on disability in patients from EVOLVE-MS-1 who have received DRF treatment for up to 2 years.

Methods

EVOLVE-MS-1 (NCT02634307) is an ongoing, open-label, phase 3 study to assess the long-term safety, tolerability, and efficacy of DRF 462 mg over 96 weeks in adults with relapsing-remitting MS. Normalized brain volume was assessed at baseline and used to calculate PBVC at Weeks 48 and 96. Confirmed Disability progression (CDP) was measured using the Expanded Disability Status Scale (≥1.5-, ≥1.0-, or ≥0.5-point increase from a baseline score of 0, 1.0-5.5, or 6.0, respectively), with changes sustained for 12 weeks. Estimated proportion of patients with CDP was calculated by the Kaplan-Meier method. No evidence of disease activity (NEDA)-3 was defined as no relapses, no 12-week CDP, and no new/enlarging T2 or new gadolinium-enhancing lesions. This post hoc analysis was conducted in a subgroup of patients who had brain volume scan measurements at baseline, Week 48, and Week 96. The Week 48 and Week 96 visits occurred within an analysis window of ±12 weeks.

Results

As of 2 July 2019, a total of 1051 patients were enrolled in EVOLVE-MS-1 and 365 patients were included in this analysis. Median (range) exposure was 96 (75-100) weeks. Mean (SD) PBVC was -0.36 (0.60) from baseline to Week 48 and -0.35 (0.55) from Week 48 to Week 96. Estimated proportion of patients who were free of CDP was 94.3% at Week 48 and 90.7% at Week 96. The proportion of patients with NEDA-3 at Week 48 and Week 96 was 44.7% (163/365) and 25.2% (91/361), respectively.

Conclusions

Interim findings from the ongoing EVOLVE-MS-1 study demonstrate that yearly PBVC in DRF-treated patients approached the rate observed in healthy adults and was consistent with previous studies of DMF. Most patients remained free of CDP at 2 years.

Supported by: Biogen

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Patient-Reported Outcomes and Quality of Life Poster Presentation

P1067 - Understanding the patient and clinician perspective of ideal treatments for multiple sclerosis via group concept mapping (ID 698)

Speakers
Presentation Number
P1067
Presentation Topic
Patient-Reported Outcomes and Quality of Life

Abstract

Background

Patients with multiple sclerosis (MS) have an array of treatment options targeting various aspects of their disease. Clinicians should consider patient preferences when selecting from these options. The medical literature has shown that MS patients often focus on safety as the most important attribute of a medication; however, preference instruments often include preselected attributes chosen by the investigator.

Objectives

A novel online method to elicit patient-centered preferences, Group Concept Mapping (GCM), was used to generate statements defining the patient experience in relapsing-remitting MS to identify the most important, relevant, and patient-focused outcomes from patient and clinician perspectives.

Methods

Twenty patients and 14 MS specialists in the US provided statements describing an ideal treatment that would affect daily function in MS. The statements were harmonized by the research team, sorted into domains that were meaningful to participants, and rated on importance on an 11-point scale.

Results

Sixty-four unique concepts were generated by patients and clinicians. GCM analysis identified 6 domains of clustered concepts. Patient and clinician ratings were highly correlated (r=0.82); however, patients rated items within the domains of Activities of Daily Living, Prevent & Cure, and Address Symptoms as highest in importance, while clinicians rated items within Prevent & Cure, Safe & Effective, and Activities of Daily Living as highest in importance. Three of the 11 statements in the Activities of Daily Living domain were rated above the mean in importance by both patients and clinicians; these included “improve cognitive function” and “improve motor function.” The 5 statements in the Address Symptoms domain were all rated above the mean in importance by both patients and clinicians; these included “help with memory issues” and “help preserve cognition.” The statement “improve short term memory” was 1 of 3 statements rated above the mean by patients that was rated below the mean by clinicians.

Conclusions

A high level of agreement of concept importance was found between patients and MS specialists. Both tended to rate concepts relating to cognitive function more highly than the other statements. Concepts related to safety or administration of medication were not rated as highly as concepts related to activities of daily living that have more of a functional impact on quality of life.

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Presenter Of 1 Presentation

Patient-Reported Outcomes and Quality of Life Poster Presentation

P1067 - Understanding the patient and clinician perspective of ideal treatments for multiple sclerosis via group concept mapping (ID 698)

Speakers
Presentation Number
P1067
Presentation Topic
Patient-Reported Outcomes and Quality of Life

Abstract

Background

Patients with multiple sclerosis (MS) have an array of treatment options targeting various aspects of their disease. Clinicians should consider patient preferences when selecting from these options. The medical literature has shown that MS patients often focus on safety as the most important attribute of a medication; however, preference instruments often include preselected attributes chosen by the investigator.

Objectives

A novel online method to elicit patient-centered preferences, Group Concept Mapping (GCM), was used to generate statements defining the patient experience in relapsing-remitting MS to identify the most important, relevant, and patient-focused outcomes from patient and clinician perspectives.

Methods

Twenty patients and 14 MS specialists in the US provided statements describing an ideal treatment that would affect daily function in MS. The statements were harmonized by the research team, sorted into domains that were meaningful to participants, and rated on importance on an 11-point scale.

Results

Sixty-four unique concepts were generated by patients and clinicians. GCM analysis identified 6 domains of clustered concepts. Patient and clinician ratings were highly correlated (r=0.82); however, patients rated items within the domains of Activities of Daily Living, Prevent & Cure, and Address Symptoms as highest in importance, while clinicians rated items within Prevent & Cure, Safe & Effective, and Activities of Daily Living as highest in importance. Three of the 11 statements in the Activities of Daily Living domain were rated above the mean in importance by both patients and clinicians; these included “improve cognitive function” and “improve motor function.” The 5 statements in the Address Symptoms domain were all rated above the mean in importance by both patients and clinicians; these included “help with memory issues” and “help preserve cognition.” The statement “improve short term memory” was 1 of 3 statements rated above the mean by patients that was rated below the mean by clinicians.

Conclusions

A high level of agreement of concept importance was found between patients and MS specialists. Both tended to rate concepts relating to cognitive function more highly than the other statements. Concepts related to safety or administration of medication were not rated as highly as concepts related to activities of daily living that have more of a functional impact on quality of life.

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