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Review
. 2022 Jan-Mar;36(1):64-72.
doi: 10.1097/WAD.0000000000000473.

Outcome Measures for Dementia With Lewy Body Clinical Trials: A Review

Bhavana Patel  1 , David J Irwin  2 , Daniel Kaufer  3 , Bradley F Boeve  4 , Angela Taylor  5   6 , Melissa J Armstrong  1
Affiliations
Review

Outcome Measures for Dementia With Lewy Body Clinical Trials: A Review

Bhavana Patel et al. Alzheimer Dis Assoc Disord. 2022 Jan-Mar.

Abstract

Background: Dementia with Lewy bodies (DLB) is one of the most common degenerative dementias. Clinical trials for individuals with DLB are increasing. We aimed to identify commonly used outcome measures for trials in DLB.

Methods: A pragmatic literature search of PubMed and clinicaltrials.gov identified interventional studies including populations with DLB. Studies were included if they enrolled participants with DLB and met the National Institutes of Health criteria for a clinical trial. Data were collected using standardized forms. Outcome measures were categorized according to core and supportive features of DLB.

Results: After de-duplication, 58 trials were identified. The most common cognitive outcome measures were the Mini Mental State Examination (n=24) and Cognitive Drug Research computerized Assessment System (n=5). The Clinician's Assessment of Fluctuations was the most commonly used measure for fluctuations (n=4). Over half of studies used the Neuropsychiatric Inventory to assess behavioral symptoms (n=31). The Unified Parkinson's Disease Rating Scale was frequently used for motor assessment (n=23).

Conclusions and relevance: Clinical trial outcomes used in DLB are rarely validated in this population and some lack face validity. There is a need to validate existing scales in DLB and develop DLB-specific outcome measures.

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Conflict of interest statement

B.P. has received a training grant from the American Brain Foundation. She received compensation for consultation with Medtronic. D.K. received research support from Acadia, Alector, Axovant, and EIP Pharma, as well as NIH, AHRQ, the National Football League, and the Bryan Family Foundation. He has served as a consultant for Axovant and VeraSci, and receives royalties from UptoDate. B.F.B. has served as an investigator for clinical trials sponsored by Biogen, Alector, and EIP Pharma. He receives royalties from the publication of a book entitled Behavioral Neurology of Dementia (Cambridge Medicine, 2009, 2017). He serves on the Scientific Advisory Board of the Tau Consortium. He receives research support from the NIH, the Mayo Clinic Dorothy and Harry T. Mangurian Jr. Lewy Body Dementia Program, the Little Family Foundation, and the Turner Family Foundation. A.T. is an employee of the Lewy Body Dementia Association. M.J.A. is supported by an ARHQ K08 career development award (K08HS24159); receives compensation from the AAN for work as an evidence-based medicine methodology consultant and is on the level of evidence Editorial Board for Neurology and related publications (uncompensated); receives publishing royalties for Parkinson’s Disease: Improving Patient Care (Oxford University Press, 2014); and has received honoraria from Medscape CME. D.J.I. declares no conflicts of interest.

Figures

Figure 1.
Figure 1.
Steps to Clinical Outcome Measure Development in DLB.* Legend: *Adapted and modified from the U.S. Food and Drug Administration Center for Drug Evaluation and Research Office of New Drugs
See this image and copyright information in PMC

References

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