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

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

doi:10.1186/s40035-022-00299-w

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

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

^*Corresponding author for this work

School of Medicine, Dentistry and Biomedical Sciences

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Abstract

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.

Original language	English
Article number	24
Journal	Translational Neurodegeneration
Volume	11
DOIs	https://doi.org/10.1186/s40035-022-00299-w
Publication status	Published - 02 May 2022

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Clinical outcome measures in dementia with Lewy bodies trials: critique and recommendations
Copyright 2022 the authors. This is an open access article published under a Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution and reproduction in any medium, provided the author and source are cited.
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Rodriguez-Porcel, F., Wyman-Chick, K., Abdelnour-Ruiz, C., Toledo, J., Ferreira, D., Urwyler, P., Weil, R., Kane, J., Pilotto, A., Rongve, A., Boeve, B. F., Taylor, J.-P., McKeith, I., Aarsland, D., & Lewis, S. J. G. (2022). Clinical outcome measures in dementia with Lewy bodies trials: critique and recommendations. Translational Neurodegeneration, 11, Article 24. https://doi.org/10.1186/s40035-022-00299-w

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abstract = "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{\textquoteright}s or Parkinson{\textquoteright}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.",

author = "Federico Rodriguez-Porcel and Kathryn Wyman-Chick and Carla Abdelnour-Ruiz and Jon Toledo and Daniel Ferreira and Prabitha Urwyler and Rimona Weil and Joseph Kane and Andrea Pilotto and Arvid Rongve and Boeve, {Bradley F.} and John-Paul Taylor and Ian McKeith and Dag Aarsland and Lewis, {Simon J G}",

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Rodriguez-Porcel, F, Wyman-Chick, K, Abdelnour-Ruiz, C, Toledo, J, Ferreira, D, Urwyler, P, Weil, R, Kane, J, Pilotto, A, Rongve, A, Boeve, BF, Taylor, J-P, McKeith, I, Aarsland, D & Lewis, SJG 2022, 'Clinical outcome measures in dementia with Lewy bodies trials: critique and recommendations', Translational Neurodegeneration, vol. 11, 24. https://doi.org/10.1186/s40035-022-00299-w

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T1 - Clinical outcome measures in dementia with Lewy bodies trials: critique and recommendations

AU - Rodriguez-Porcel, Federico

AU - Wyman-Chick, Kathryn

AU - Abdelnour-Ruiz, Carla

AU - Toledo, Jon

AU - Ferreira, Daniel

AU - Urwyler, Prabitha

AU - Weil, Rimona

AU - Kane, Joseph

AU - Pilotto, Andrea

AU - Rongve, Arvid

AU - Boeve, Bradley F.

AU - Taylor, John-Paul

AU - McKeith, Ian

AU - Aarsland, Dag

AU - Lewis, Simon J G

PY - 2022/5/2

Y1 - 2022/5/2

N2 - 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.

AB - 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.

U2 - 10.1186/s40035-022-00299-w

DO - 10.1186/s40035-022-00299-w

M3 - Article

VL - 11

JO - Translational Neurodegeneration

JF - Translational Neurodegeneration

M1 - 24

ER -

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

Abstract

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