Journal Article

Clinical outcome measures in dementia with Lewy bodies trials: critique and recommendations

Abdelnour Ruiz, Carla, Ferreira, Daniel, Aarsland, Dag, Urwyler, Prabitha, Kane, Joseph, Lewis, Simon J. G., Taylor, John-Paul, Rongve, Arvid, Pilotto, Andrea, McKeith, Ian, Rodriguez-Porcel, Federico, Boeve, Bradley, Toledo, Jon B., Wyman-Chick, Kathryn A., Weil, Rimona S.

2022

Overview

The selection of appropriate outcome measures is fundamental to the design of any successful clinical trial. Although dementia with Lewy bodies (DLB) is one of the most common neurodegenerative conditions, assessment of therapeutic benefit in clinical trials often relies on tools developed for other conditions, such as Alzheimer’s or Parkinson’s disease. These may not be sufficiently valid or sensitive to treatment changes in DLB, decreasing their utility. In this review, we discuss the limitations and strengths of selected available tools used to measure DLB-associated outcomes in clinical trials and highlight the potential roles for more specific objective measures. We emphasize that the existing outcome measures require validation in the DLB population and that DLB-specific outcomes need to be developed. Finally, we highlight how the selection of outcome measures may vary between symptomatic and disease-modifying therapy trials.

• Publisher: BioMed Central,Springer Nature B.V,BMC
• Subject: Alzheimer's disease / Biomarkers / Biomedical and Life Sciences / Biomedicine / Caregivers / Clinical outcomes / Clinical trials / Dementia / Dementia with Lewy bodies / Hallucinations / Humans / Lewy Body Disease - drug therapy / Lewy Body Disease - therapy / Measurement properties / Neurology / Neurosciences / Outcome Assessment, Health Care / Outcomes / Parkinson Disease - diagnosis / Parkinson Disease - epidemiology / Parkinson Disease - therapy / Parkinson's disease / Patients / Quality of life / Review / Validity