Biogen

Author Of 3 Presentations

Clinical Outcome Measures Poster Presentation

P0022 - A systematic literature review and meta-analysis of the efficacy and effectiveness of PR-fampridine in patients with multiple sclerosis (ID 1316)

Speakers
Presentation Number
P0022
Presentation Topic
Clinical Outcome Measures

Abstract

Background

Prolonged release (PR) fampridine is indicated for the improvement of walking in adult multiple sclerosis (MS) patients. Several studies have supported that PR-fampridine improves walking ability in this population; however, reported effects have varied widely across studies and study design.

Objectives

To conduct a systematic literature review (SLR) and meta-analysis (MA) to summarize the evidence on the efficacy and effectiveness of PR-fampridine in MS patients.

Methods

Following PRISMA guidelines, a systematic search of PubMed, EMBASE, YORK and Cochrane Library was conducted from January 1, 2006-April 1, 2019 to identify publications comparing the efficacy and effectiveness of PR-fampridine from Randomized Controlled Trials (RCTs) and observational studies (OBS). For RCTs, outcome measures include Timed 25-Foot Walk (T25FW) and 12-item Multiple Sclerosis Walking Scale (MSWS-12) responders and MSWS-12 scores; for OBS, T25FW walking time and MSWS-12 scores. In the MA, pooled estimates were derived using odds ratios (OR) and standardized mean differences (SMD) of both endpoints. Results from RCTs and OBS were reported separately using a random effects model.

Results

Of a total of 897 unique citations, 27 studies met all criteria for inclusion in the MA, 9 RCTs and 18 single-arm OBS. A pooled estimate based on T25FW responder data from 4 RCTs showed statistically significant improvements in walking ability in the PR-fampridine vs. placebo group; OR (95% CI): 4.8 (2.9-7.9), p<0.0005. Also, findings based on MSWS-12 responder data from 2 RCTs showed significant improvements in walking ability in the PR-fampridine group vs. placebo; OR (95% CI): 1.7 (1.1-2.5); p=0.011. A pooled estimate of mean MSWS-12 scores from 4 RCTs also showed significant improvements in the PR-fampridine vs. placebo group; OR (95% CI): 1.5 (1.2-1.9); p<0.0005. Summary estimates from OBS suggest a significant improvement vs. baseline on T25FW time derived from 9 studies showing that walking time was significantly improved vs. baseline; with SMD (95% CI): –0.31 (-0.479 to -0.146); p<0.0005. Also, the pooled estimate from MSWS-12 endpoint in 6 studies showed that walking ability was statistically improved vs. baseline; SMD (95% CI): -0.98 (-0.892 to -1.058); p<0.0005.

Conclusions

Across randomized and observational data, the use of PR-fampridine is consistently associated with significantly improved walking ability in MS patients measured by MSWS-12 or T25FW endpoints.

This study is funded by Biogen. Biogen funded the analyses for this abstract.

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Epidemiology Poster Presentation

P0494 - Socio-demographic and clinical characteristics of patients with multiple sclerosis by race and ethnicity (NARCRMS registry) (ID 808)

Speakers
Presentation Number
P0494
Presentation Topic
Epidemiology

Abstract

Background

The North American Registry for Care and Research in Multiple Sclerosis (NARCRMS) registry is a physician-based registry/longitudinal database for patients with multiple sclerosis (MS). NARCRMS may elucidate the patient characteristics of underserved populations such as Blacks/African Americans (AA) and Hispanics/Latinos.

Objectives

To describe the socio-demographic and clinical characteristics of patients presenting with relapsing-remitting MS (RRMS) within the NARCRMS registry by race and ethnicity.

Methods

The NARCRMS registry contains data of MS patients aged 18-50 years across 24 sites from the US and Canada. This exploratory analysis describes characteristics of patients who enrolled between December 2016 and May 2020 (N=722), including age, gender, education, income level and occupation, Expanded Disability Status Scale (EDSS) categories, and disease-modifying therapy (DMT) categories/DMT use. Patient characteristics were summarized by frequencies and proportions for categorical variables and by means, standard deviations (SDs) and medians for continuous variables.

Results

The mean age (SD) of patients in this study was 40.1 (10.4) years; 71% were female. Majority (85%, n=587/695) were White; Black/AA patients comprised 11% (n=74/695). Educational attainment was comparable between races ― 22-24% with a high school degree, and 47-49% with an undergraduate degree. However, more Black/AA than White patients were unemployed (8%, n=6/72 vs 3%, n=15/565) or had an annual income <$15K (16%, n=12/73 vs 6%, n=35/573). Overall, 72% of patients had mild MS (EDSS scores 0‒2.5). However, twice as many Blacks/AAs had substantial disability (EDSS score ≥4.0) vs Whites (20%, n=15/74 vs 9.7%, n=57/587, respectively). Over half of all patients (57%, n=370/646) were treated with DMTs, with 50% (n=198) using injectables and 37% (n=147) using oral DMTs. Hispanics comprised 24% (n=152/646) of the patients, including Black/AA-Hispanic (3%, n=19/646) and White-Hispanic (21%, n=133/646). Hispanic patients were less likely than non-Hispanics to use DMTs, 43% (n=65/152) vs 62% (n=305/494). Of the subgroups, Black/AA-Hispanics were least likely to use DMTs (26%, n=5/19).

Conclusions

Blacks/AAs present with more severe disability than White patients. More Hispanics than non-Hispanics are not treated with DMTs. Real-world data show disparities in socio-demographic and clinical characteristics of patients with MS.

Supported by: Biogen

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Patient-Reported Outcomes and Quality of Life Poster Presentation

P1035 - Impact of Natalizumab and Other High Efficacy Disease-Modifying Therapies on Productivity Loss in Multiple Sclerosis: A Systematic Literature Review. (ID 1623)

Speakers
Presentation Number
P1035
Presentation Topic
Patient-Reported Outcomes and Quality of Life

Abstract

Background

Despite availability of high-efficacy disease-modifying therapies (hDMTs), there is no cure for multiple sclerosis (MS). Productivity loss associated with MS can be substantial, yet whether a specific hDMT performs better than another in reducing the resultant indirect costs is not well understood.

Objectives

To conduct a systematic literature review evaluating the impact of natalizumab and other hDMTs on productivity loss among patients with MS.

Methods

Embase, MEDLINE, Cochrane, EconLit, and NHS EED were searched (Jan 1, 2005 to Dec 4, 2019) using key terms; additional materials were identified from grey literature. Data from studies meeting all pre-defined inclusion and no exclusion criteria were collected.

Results

Of 649 unique citations, 20 reported on productivity loss among relapsing remitting MS (RRMS) or unspecified/mixed MS patients receiving hDMTs. Work productivity loss was the most commonly reported outcome (k=14), followed by disability/activity impairment (6), sick leave (k=6), household productivity loss (k=2), and retirement (k=1). Most studies were on natalizumab (k=13), followed by fingolimod (k=5), alemtuzumab (k=4), cladribine (k=1), and other DMTs (k=2). Only two studies provided a direct comparison between natalizumab and other DMTs. A cohort study showed natalizumab led to significantly greater reduction in productivity loss than fingolimod, beta-interferons, and glatiramer acetate (k=1 cohort study). A separate cost-minimization analysis, modeling indirect costs, estimated fingolimod led to greater cost savings than natalizumab, though significance was not reported. Across non-comparative studies, natalizumab (k=7 cohort studies, 1 cost analysis, 1 single arm trial) and other DMTs (k=3 cohort studies) consistently reduced patients’ productivity loss burden.

Conclusions

Natalizumab was consistently reported to improve productivity. However, a paucity of evidence and heterogeneity in study design limit comparisons to other hDMTs. Future research should evaluate the impact of specific hDMTs on attenuating productivity loss using methods reflecting real-world evidence, rather than modeling techniques, and productivity loss definitions already commonly used in the literature.

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