Patient-Centered Approach Might Effectively Tackle The Definition Of Progression In Chronic Neurological Diseases: Results From the EmBioProMS Trial in Progressive Multiple Sclerosis.

BackgroundProper identification of disability accumulation in the routine clinical care of progressive multiple sclerosis (PMS) patients is usually a challenging task. Patient-reported outcome measurements (PROMs) can provide a practical, cost-efficient, and remotely accessible tool to assess disease progression. MethodsEmBioProMS is a prospective, multicentric cohort, conducted in 7 specialized MS centers in Germany. PROMs were evaluated at inclusion and compared between patients with retrospective evidence of disease progression in the last two years and those with stable disease. Patients with either primary or secondary progressive MS according to the McDonald criteria 2017 were included in the analysis, while patients with incomplete PROMs scores, MS relapses, other neurological or systemic inflammatory diseases were excluded. The disease progression was assessed using a combined outcome parameter, including EDSS score, timed 25-foot walk test, and nine-hole-peg test. Results185 patients were included in the final analysis (SPMS, n=77; PPMS, n=108). The median age and disease duration were 55 years and 13 years, respectively. Disease progression was diagnosed in 114 of 185 patients (61.6%). BDI-II, MSIS-29, and FSMC scores were worse in patients with evidence of disease progression in the last two years. Patients with any of the included PROMs above the 90th percentile had an odds ratio of 3.8 (95% confidence interval: 1.4-10.6, P=0.01) for having progression in the last two years in a binomial regression model adjusted for age, sex, disease duration, treatment status, center effect, and Expanded Disability Status Scale (EDSS). Similar results were observed in patients with PROM scores in the 80th and 70th percentile (OR: 2.9 and 3.7, P=0.015 and 0.003, respectively). ConclusionPROMs can be a simple and effective way to detect disability worsening in a chronic neurological disease like PMS and, therefore, substantially contribute to better classification and prognostication of the disease course through objective and structural patient-doctor communication. Trial RegistrationGerman Clinical Trials Register (Deutsches Register Klinischer Studien - DRKS), DRKS00020132