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Abstract Details

Interpretation of Clinical Progression in Multiple System Atrophy Using Percentage-wise Slowing in the Unified Multiple System Atrophy Rating Scale Score
Movement Disorders
P10 - Poster Session 10 (8:00 AM-9:00 AM)
16-002

To examine the appropriateness and clinical relevance of expressing trial results as a percentage slowing of clinical progression between treatment and placebo in Multiple System Atrophy (MSA), and to identify the threshold at which such a difference becomes clinically meaningful.

Treatment effects have been assessed and reported as absolute changes from baseline at a fixed time point. However, there is growing interest in evaluating trial outcomes as percentage changes over the entire treatment duration – particularly in the context of therapies with potential to slow clinical progression of neurodegenerative conditions such as MSA.

International clinical experts in MSA (n=12) participated in a Delphi panel to evaluate consensus (defined as ≥70% agreement) on two key points: (1) the suitability of expressing treatment effects as percentage slowing in the Unified Multiple System Atrophy Rating Scale (UMSARS) to interpret clinical progression in MSA, and (2) the percentage change threshold in UMSARS considered clinically meaningful for individuals in the early stages of the disease.

Consensus was reached on the appropriateness of expressing treatment effect as percentage-wise slowing in UMSARS for the interpretation of clinical progression in MSA. Consensus was reached that percentage-wise slowing at 21% and above would be clinically meaningful. Over 50% agreed the lower range of 16–20% would be clinically meaningful, however, consensus was not met.

An international panel of MSA experts reached consensus that expressing treatment effects as percentage slowing in UMSARS scores is appropriate, identifying a threshold of 21% or greater as clinically meaningful.

Authors/Disclosures
Rachael Kershaw, PhD
PRESENTER
Dr. Kershaw has received personal compensation for serving as an employee of Prime Global, providing medical writing support.
Stefano Zanigni Stefano Zanigni has received personal compensation for serving as an employee of Lundbeck A/S.
Krishan Nighah, BSc Mr. Nighah has nothing to disclose.
Daniel Oudin Astrom, PhD Dr. Oudin Astrom has received personal compensation for serving as an employee of H Lundbeck A/S.
Jonas Wiedemann, MSc Mr. Wiedemann has received personal compensation for serving as an employee of Lundbeck.
Anna-Karin E. Berger, PhD Dr. Berger has nothing to disclose.