title: Outcome measures in relapsing-remitting multiple sclerosis: capturing disability and disease progression in clinical trials.
abstract: Multiple sclerosis (MS) is a chronic inflammatory and neurodegenerative disease that manifests as acute relapses and progressive disability. As a primary endpoint for clinical trials in MS, disability is difficult to both characterize and measure. Furthermore, the recovery from relapses and the rate of disability vary considerably among patients. Given these challenges, investigators have developed and studied the performance of various outcome measures and surrogate endpoints in MS clinical trials. This review defines the outcome measures and surrogate endpoints used to date in MS clinical trials and presents challenges in the design of both adult and pediatric trials.
authors.affiliation: Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
authors.forename: Amy M
authors.initials: AM
authors.lastname: Lavery
authors.affiliation: The Pediatric Demyelinating Disease Program, Program in Neuroscience & Mental Health, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada M5G 1X8.
authors.forename: Leonard H
authors.initials: LH
authors.lastname: Verhey
authors.affiliation: Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA ; Departments of Neurology and Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
authors.forename: Amy T
authors.initials: AT
authors.lastname: Waldman