TY - JOUR
T1 - Clinical outcome measures in dementia with Lewy bodies trials
T2 - critique and recommendations
AU - 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)+
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
N1 - © 2022. The Author(s).
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 - Humans
KW - Lewy Body Disease/drug therapy
KW - Outcome Assessment, Health Care
KW - Parkinson Disease/diagnosis
U2 - 10.1186/s40035-022-00299-w
DO - 10.1186/s40035-022-00299-w
M3 - Review article
C2 - 35491418
SN - 2047-9158
VL - 11
SP - 24
JO - Translational Neurodegeneration
JF - Translational Neurodegeneration
IS - 1
ER -