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

Federico Rodriguez-Porcel; Kathryn A. Wyman-Chick; Carla Abdelnour Ruiz; Jon B. Toledo; Daniel Ferreira; Prabitha Urwyler; Rimona S. Weil; Joseph Kane; Andrea Pilotto; Arvid Rongve; Bradley Boeve; John-Paul Taylor; Ian McKeith; Dag Aarsland; Simon J. G. Lewis; the Lewy Body Dementias Clinical Trials Workgroup from the Lewy Body Dementias Professional Interest Area - Alzheimer’s Association International Society to Advance Alzheimer’s Research and Treatment (ISTAART)+

doi:10.1186/s40035-022-00299-w

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

Federico Rodriguez-Porcel^*, Kathryn A. Wyman-Chick, Carla Abdelnour Ruiz, Jon B. Toledo, Daniel Ferreira, Prabitha Urwyler, Rimona S. Weil, Joseph Kane, Andrea Pilotto, Arvid Rongve, Bradley Boeve, John-Paul Taylor, Ian McKeith, Dag Aarsland, Simon J. G. Lewis, the Lewy Body Dementias Clinical Trials Workgroup from the Lewy Body Dementias Professional Interest Area - Alzheimer’s Association International Society to Advance Alzheimer’s Research and Treatment (ISTAART)+

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

21 Citations (Scopus)

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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
Pages (from-to)	1-18
Number of pages	18
Journal	Translational Neurodegeneration
Volume	11
Issue number	1
DOIs	https://doi.org/10.1186/s40035-022-00299-w
Publication status	Published - 2 May 2022
Externally published	Yes

Bibliographical note

Copyright the Author(s) 2022. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.

A correction exists for this article, the original has been updated. Correction can be found at doi: 10.1186/s40035-022-00306-0

Keywords

Clinical trials
Dementia with Lewy bodies
Measurement properties
Outcomes

Access to Document

10.1186/s40035-022-00299-wLicence: CC BY

Publisher version (open access)Final published version, 848 KBLicence: CC BY

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Rodriguez-Porcel, F., Wyman-Chick, K. A., Abdelnour Ruiz, C., Toledo, J. B., Ferreira, D., Urwyler, P., Weil, R. S., Kane, J., Pilotto, A., Rongve, A., Boeve, B., Taylor, J.-P., McKeith, I., Aarsland, D., Lewis, S. J. G., & the Lewy Body Dementias Clinical Trials Workgroup from the Lewy Body Dementias Professional Interest Area - Alzheimer’s Association International Society to Advance Alzheimer’s Research and Treatment (ISTAART)+ (2022). Clinical outcome measures in dementia with Lewy bodies trials: critique and recommendations. Translational Neurodegeneration, 11(1), 1-18. Article 24. https://doi.org/10.1186/s40035-022-00299-w

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title = "Clinical outcome measures in dementia with Lewy bodies trials: critique and recommendations",

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.",

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note = "Copyright the Author(s) 2022. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher. A correction exists for this article, the original has been updated. Correction can be found at doi: 10.1186/s40035-022-00306-0",

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doi = "10.1186/s40035-022-00299-w",

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Rodriguez-Porcel, F, Wyman-Chick, KA, Abdelnour Ruiz, C, Toledo, JB, Ferreira, D, Urwyler, P, Weil, RS, Kane, J, Pilotto, A, Rongve, A, Boeve, B, Taylor, J-P, McKeith, I, Aarsland, D, Lewis, SJG & the Lewy Body Dementias Clinical Trials Workgroup from the Lewy Body Dementias Professional Interest Area - Alzheimer’s Association International Society to Advance Alzheimer’s Research and Treatment (ISTAART)+ 2022, 'Clinical outcome measures in dementia with Lewy bodies trials: critique and recommendations', Translational Neurodegeneration, vol. 11, no. 1, 24, pp. 1-18. https://doi.org/10.1186/s40035-022-00299-w

TY - JOUR

T1 - Clinical outcome measures in dementia with Lewy bodies trials

T2 - critique and recommendations

AU - Rodriguez-Porcel, Federico

AU - Wyman-Chick, Kathryn A.

AU - Abdelnour Ruiz, Carla

AU - Toledo, Jon B.

AU - Ferreira, Daniel

AU - Urwyler, Prabitha

AU - Weil, Rimona S.

AU - Kane, Joseph

AU - Pilotto, Andrea

AU - Rongve, Arvid

AU - Boeve, Bradley

AU - Taylor, John-Paul

AU - McKeith, Ian

AU - Aarsland, Dag

AU - Lewis, Simon J. G.

AU - the Lewy Body Dementias Clinical Trials Workgroup from the Lewy Body Dementias Professional Interest Area - Alzheimer’s Association International Society to Advance Alzheimer’s Research and Treatment (ISTAART)+

N1 - Copyright the Author(s) 2022. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher. A correction exists for this article, the original has been updated. Correction can be found at doi: 10.1186/s40035-022-00306-0

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.

KW - Clinical trials

KW - Dementia with Lewy bodies

KW - Measurement properties

KW - Outcomes

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UR - https://doi.org/10.1186/s40035-022-00306-0

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U2 - 10.1186/s40035-022-00299-w

DO - 10.1186/s40035-022-00299-w

M3 - Review article

C2 - 35491418

AN - SCOPUS:85131646880

SN - 2047-9158

VL - 11

SP - 1

EP - 18

JO - Translational Neurodegeneration

JF - Translational Neurodegeneration

IS - 1

M1 - 24

ER -

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

Abstract

Bibliographical note

Keywords

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