Topic
Topic
Multiple Sclerosis
Sometimes the severity of progressive multiple sclerosis improves naturally, and we know little about this.
Setup
Design
R
Realized in R-Statistics, SQL
We investigated the natural occurrence of disability progression with a focus on disability improvements in people diagnosed with progressive multiple sclerosis (PMS) who were not on treatment given a nonactive disease.
To investigate disease improvements and progressions, we combined clinical trial data (RCT) and real-world registry data (RWD). The RWD came from a longitudinal outpatient neurology registry and included patients diagnosed with progressive multiple sclerosis who received no treatment and had been two years relapse-free. A confirmed disease improvement was defined following the literature as a clinically meaningful disease severity improvement at 12 to 24 months which lasted 6 months or longer based on a clinician-assessed standard measure of multiple sclerosis disease severity (expanded disability status scale, EDSS). Experiencing a confirmed disease progression was defined analogously, following the literature. We described the occurrence rates of disability improvements and progressions. Beyond this, we aligned the real-world data to trial data. The reason for this was because the trial had a single arm (treated) implying a scientific need to contextualize the disease improvements that were found in the trial’s treated population by results from a non-treated sample. After aligning the trial population to the real-world population (RWD-to-RCT alignment) following a target-trial emulation framework and using propensity-score alignments (PS weighting) we, again, reported the key outcomes. The results, after ensuring that the propensity-score weighting resulted in balanced cohorts, showed percentages of patients with a confirmed disability improvement ranged from 1.9% to 2.8% measured at 12 to 24 months. The percentages of patients with a confirmed progression ranged from 11.5% to 20.3%, respectively, for untreated non-active persons who were diagnosed with progressive multiple sclerosis.
You can read the abstract (V469) in the ACTRIMS 2024 archives.