Contributions
Abstract: P351
Type: Poster
Abstract Category: Clinical aspects of MS - 5 Epidemiology
Background: MS PATHS (Multiple Sclerosis Partners Advancing Technology and Health Solutions) plans to enrol approximately 10,000 MS patients from 10 healthcare institutions in the US and Europe. The large population, broad inclusion criteria (MS or CIS, no exclusions) and high participation rates (>90%) raise the potential for more representative populations and more generalizable research findings compared to randomized control trials (RCTs) or prospective cohort studies.
Objectives: To compare socio-demographic and clinical characteristics of MS PATHS patients with MS patients from six Biogen-sponsored RCTs and the EPIC (Expression, Proteomics, Imaging, Clinical) prospective cohort study.
Methods: Descriptive statistics were used to describe MS PATHS (n=1353), the pooled RCTs (n=6574) and EPIC (n=579) at baseline. The characteristics within each cohort were: age (yrs), sex, MS duration (yrs), relapses in last 12 months, 9-Hole Peg Test (9HPT) (sec) and Timed 25-Foot Walk (T25FW) (sec). Pairwise comparisons of MS PATHS patients vs. RCTs and EPIC were made with Wilcoxon rank sum tests and chi-squared tests. Standardized differences were also assessed. Pairwise c-statistics were estimated from multivariate logistic regression models to address overall similarity.
Results: MS PATHS mean (SD) age was 48.9 (12.0), higher than the RCTs [37.4 (8.9)] and EPIC [43.0 (9.8)]. Mean disease duration was 12.2 (9.4) in MS PATHS, compared with 7.7 (6.5) and 9.0 (8.8) in the RCTs and EPIC, respectively. The percent of patients with zero relapses in the last 12 months was higher in MS PATHS (47.3) and EPIC (58.0) vs. RCTs (1.4). For MS PATHS, RCTs and EPIC, the 9HPT means (SD) were 28.8 (7.1), 22.7 (11.2) and 21.9 (5.3), whereas T25FW means (SD) were 8.1 (5.4), 6.7 (6.9) and 6.0 (6.2). All standardized differences were >10% and all p-values were < 0.05, except for sex. MS PATHS multivariate c-statistics for membership was 0.88 vs. RCTs and 0.82 vs. EPIC, showing the overall uniqueness of the MS PATHS cohort.
Conclusions: MS PATHS patients were older with longer disease duration, less active disease and worse functional performance compared to patients enrolled in RCTs and EPIC. Given the high participation rate, the MS PATHS cohort is likely representative of MS patients seeking care at MS centers. MS PATHS provides an opportunity to answer key clinical questions that might be generalizable beyond findings from RCTs and other selected cohorts.
Disclosure: Project funded by Biogen, Inc.
Author Disclosures:
Fabio Pellegrini, Carl de Moor, Ulrich Freudensprung and Richard Rudick are employees of, and stockholders in, Biogen.
Ellen M. Mowry has received research funding from Biogen and free medication for a clinical trial from Teva Neuroscience. She is a site PI for trials funded by Biogen and Sun Pharma, and receives royalties for editorial duties with UpToDate.
Massimiliano Copetti received consulting fees from Biogen, Teva and Eisai.
Francesca Bovis has nothing to disclose.
Maria Pia Sormani received consulting fees from Biogen, Novartis, Genzyme, Roche, Teva, GeNeuro, Merck Serono and Medday.
Abstract: P351
Type: Poster
Abstract Category: Clinical aspects of MS - 5 Epidemiology
Background: MS PATHS (Multiple Sclerosis Partners Advancing Technology and Health Solutions) plans to enrol approximately 10,000 MS patients from 10 healthcare institutions in the US and Europe. The large population, broad inclusion criteria (MS or CIS, no exclusions) and high participation rates (>90%) raise the potential for more representative populations and more generalizable research findings compared to randomized control trials (RCTs) or prospective cohort studies.
Objectives: To compare socio-demographic and clinical characteristics of MS PATHS patients with MS patients from six Biogen-sponsored RCTs and the EPIC (Expression, Proteomics, Imaging, Clinical) prospective cohort study.
Methods: Descriptive statistics were used to describe MS PATHS (n=1353), the pooled RCTs (n=6574) and EPIC (n=579) at baseline. The characteristics within each cohort were: age (yrs), sex, MS duration (yrs), relapses in last 12 months, 9-Hole Peg Test (9HPT) (sec) and Timed 25-Foot Walk (T25FW) (sec). Pairwise comparisons of MS PATHS patients vs. RCTs and EPIC were made with Wilcoxon rank sum tests and chi-squared tests. Standardized differences were also assessed. Pairwise c-statistics were estimated from multivariate logistic regression models to address overall similarity.
Results: MS PATHS mean (SD) age was 48.9 (12.0), higher than the RCTs [37.4 (8.9)] and EPIC [43.0 (9.8)]. Mean disease duration was 12.2 (9.4) in MS PATHS, compared with 7.7 (6.5) and 9.0 (8.8) in the RCTs and EPIC, respectively. The percent of patients with zero relapses in the last 12 months was higher in MS PATHS (47.3) and EPIC (58.0) vs. RCTs (1.4). For MS PATHS, RCTs and EPIC, the 9HPT means (SD) were 28.8 (7.1), 22.7 (11.2) and 21.9 (5.3), whereas T25FW means (SD) were 8.1 (5.4), 6.7 (6.9) and 6.0 (6.2). All standardized differences were >10% and all p-values were < 0.05, except for sex. MS PATHS multivariate c-statistics for membership was 0.88 vs. RCTs and 0.82 vs. EPIC, showing the overall uniqueness of the MS PATHS cohort.
Conclusions: MS PATHS patients were older with longer disease duration, less active disease and worse functional performance compared to patients enrolled in RCTs and EPIC. Given the high participation rate, the MS PATHS cohort is likely representative of MS patients seeking care at MS centers. MS PATHS provides an opportunity to answer key clinical questions that might be generalizable beyond findings from RCTs and other selected cohorts.
Disclosure: Project funded by Biogen, Inc.
Author Disclosures:
Fabio Pellegrini, Carl de Moor, Ulrich Freudensprung and Richard Rudick are employees of, and stockholders in, Biogen.
Ellen M. Mowry has received research funding from Biogen and free medication for a clinical trial from Teva Neuroscience. She is a site PI for trials funded by Biogen and Sun Pharma, and receives royalties for editorial duties with UpToDate.
Massimiliano Copetti received consulting fees from Biogen, Teva and Eisai.
Francesca Bovis has nothing to disclose.
Maria Pia Sormani received consulting fees from Biogen, Novartis, Genzyme, Roche, Teva, GeNeuro, Merck Serono and Medday.