Patient 'engagement' or 'involvement' in health research broadly refers to including people with lived experience (i.e. individuals with personal experience of a health issue and their friends, family and caregivers or carers) in the research process. Although previous reviews have systematically summarized approaches to patient engagement in research, it is unclear whether and how engagement activities have been implemented or adapted for research related to dementia. We conducted a scoping review to describe the extent and nature of patient engagement approaches that have been used to involve persons with dementia and their care partners in research. We then summarized the reported barriers, enablers, and impacts of this engagement. Fifty-four research articles were included in the review and almost all were published after 2010. Persons with dementia and their care partners have been engaged in diverse phases of the research process. The majority of engagement involved both persons with dementia and care partners. Barriers and enablers to engagement included those identified for general patient engagement in research, but some more specific to engaging persons with dementia and their care partners were also reported. Very few studies assessed the impact of patient engagement. While the arguments for patient engagement in research are compelling, research to demonstrate the impact - on the research process and outcomes as well as on persons with dementia, care partners, researchers, research institutions and society - is still needed.

Despite increasing numbers of people living with dementia, there are currently no disease modifying treatments. Across disease modification trials there is a large variety of outcomes used, making it difficult to compare and contrast results. An agreed core set of the best available and most appropriate outcomes for disease modification would make results comparable and meta-analysable in future trials. We aimed to produce the first evidence-based core outcome set for use in disease modification trials in mild-to-moderate dementia. We conducted a systematic review of published and ongoing disease modification trials, to extract the outcomes used across randomised and clinical controlled trials (RCT/ CCT). We divided outcomes into domains measured, and searched for validation data. We consulted with people with dementia and carers about the importance and acceptability of the outcome domains in disease modifying trials. We then presented all information at a conference attended by the wider body of National Institute of Health Research dementia researchers to reach consensus on the core outcome set. We screened 22, 918 references, finding 125 disease modification RCTs and CCTs. Eighty-one outcomes were used, including 72 scales (across five domains: 31 cognitive, 12 activities of daily living, ten global, 16 neuropsychiatric, and three quality of life) and nine biological techniques. We consulted with 18 people about the acceptability of outcomes. The conference attendees reached the consensus that only cognition and biological markers should be included in the core outcome set, measured via the Mini Mental State Examination (MMSE) or the Alzheimer's disease Assessment Scale - Cognitive subscale (ADAS-Cog), and brain changes through a structural Magnetic Resonance Imaging (MRI) scan in a subset of participants who agree to. All other domains are important but not core. Cognitive outcomes and biological markers form our core outcome set for future disease modification trials, measured via the MMSE or ADAS-Cog, and a structural MRI scan in a subset of participants. Most of the studies included participants with only Alzheimer's disease (AD), therefore these recommendations are mainly for AD. We envisage the core set may be superseded in the future, particularly for other dementias.

Background: There is currently no disease-modifying treatment available to halt or delay the progression of the disease pathology in dementia. An agreed core set of the best-available and most appropriate outcomes for disease modification would facilitate the design of trials and ensure consistency across disease modification trials, as well as making results comparable and meta-analysable in future trials.Objectives: To agree a set of core outcomes for disease modification trials for mild to moderate dementia with the UK dementia research community and patient and public involvement (PPI).Data sources: We included disease modification trials with quantitative outcomes of efficacy from (1) references from related systematic reviews in workstream 1; (2) searches of the Cochrane Dementia and Cognitive Improvement Group study register, Cochrane Central Register of Controlled Trials, Cumulative Index to Nursing and Allied Health Literature, EMBASE, Latin American and Caribbean Health Sciences Literature and PsycINFO on 11 December 2015, and clinical trial registries [International Standard Randomised Controlled Trial Number (ISRCTN) and clinicaltrials. gov] on 22 and 29 January 2016; and (3) hand-searches of reference lists of relevant systematic reviews from database searches.Review methods: The project consisted of four workstreams.(1) We obtained related core outcome sets and work from co-applicants. (2) We systematically reviewed published and ongoing disease modification trials to identify the outcomes used in different domains. We extracted outcomes used in each trial, recording how many used each outcome and with how many participants. We divided outcomes into the domains measured and searched for validation data. (3) We consulted with PPI participants about recommended outcomes. (4) We presented all the synthesised information at a conference attended by the wider body of National Institute for Health Research (NIHR) dementia researchers to reach consensus on a core set of outcomes.Results: We included 149 papers from the 22,918 papers screened, referring to 125 individual trials. Eighty-one outcomes were used across trials, including 72 scales [31 cognitive, 12 activities of daily living (ADLs), 10 global, 16 neuropsychiatric and three quality of life] and nine biological techniques. We consulted with 18 people for PPI. The conference decided that only cognition and biological markers are core measures of disease modification. Cognition should be measured by the Mini Mental State Examination (MMSE) or the Alzheimer's Disease Assessment Scale -Cognitive subscale (ADAS-Cog), and brain changes through structural magnetic resonance imaging (MRI) in a subset of participants. All other domains are important but not core. We recommend using the Neuropsychiatric Inventory for neuropsychiatric symptoms: the Disability Assessment for Dementia for ADLs, the Dementia Quality of Life Measure for quality of life and the Clinical Dementia Rating scale to measure dementia globally.Limitations: Most of the trials included participants with Alzheimer's disease, so recommendations may not apply to other types of dementia. We did not conduct economic analyses. The PPI consultation was limited to members of the Alzheimer's Society Research Network.Conclusions: Cognitive outcomes and biological markers form the core outcome set for future disease modification trials, measured by the MMSE or ADAS-Cog, and structural MRI in a subset of participants.Future work: We envisage that the core set may be superseded in the future, particularly for other types of dementia. There is a need to develop an algorithm to compare scores on the MMSE and ADAS-Cog.Study registration: The project was registered with Core Outcome Measures in Effectiveness Trials [www. comet-initiative. org/studies/details/819? result= true (accessed 7 April 2016)]. The systematic review protocol is registered as PROSPERO CRD42015027346.Funding: The National Institute for Health Research Health Technology Assessment programme.

Objectives1. To appraise existing research into outcome sets being developed for use in psychosocial interventions (funded by the AS and JPND) and around what is most important to patients (measured by the ICHOM), in the light of the goals of this study.

Results of the systematic review-Development of a core outcome set for disease modification trials in mild to moderate dementia: a systematic review, patient and public consultation and consensus recommendations-NCBI Bookshelf

BackgroundThere are no disease-modifying treatments for dementia. There is also no consensus on disease modifying outcomes. We aimed to produce the first evidence-based consensus on core outcome measures for trials of disease modification in mild-to-moderate dementia.Methods and findingsWe defined disease-modification interventions as those aiming to change the underlying pathology. We systematically searched electronic databases and previous systematic reviews for published and ongoing trials of disease-modifying treatments in mild-to-moderate dementia. We included 149/22,918 of the references found; with 81 outcome measures from 125 trials. Trials involved participants with Alzheimer's disease (AD) alone (n = 111), or AD and mild cognitive impairment (n = 8) and three vascular dementia. We divided outcomes by the domain measured (cognition, activities of daily living, biological markers, neuropsychiatric symptoms, quality of life, global). We calculated the number of trials and of participants using each outcome. We detailed psychometric properties of each outcome. We sought the views of people living with dementia and family carers in three cities through Alzheimer's society focus groups. Attendees at a consensus conference (experts in dementia research, disease-modification and harmonisation measures) decided on the core set of outcomes using these results. Recommended core outcomes were cognition as the fundamental deficit in dementia and to indicate disease modification, serial structural MRIs. Cognition should be measured by Mini Mental State Examination or Alzheimer's Disease Assessment Scale-Cognitive Subscale. MRIs would be optional for patients. We also made recommendations for measuring important, but non-core domains which may not change despite disease modification.LimitationsMost trials were about AD. Specific instruments may be superseded. We searched one database for psychometric properties.InterpretationThis is the first review to identify the 81 outcome measures the research community uses for disease-modifying trials in mild-to-moderate dementia. Our recommendations will facilitate designing, comparing and meta-analysing disease modification trials in mild-to-moderate dementia, increasing their value.Trial registrationPROSPERO no. CRD42015027346.

PurposeThe purpose of PPI within this project was to present people both directly and indirectly affected by dementia, including those with experience of research participation, with some of the findings from our systematic review to seek their views on which of the domains they considered core and their assessment of the acceptability of individual and packages of measures. We also wanted to know their thoughts about general matters around completing outcome measures, including the length of testing that was acceptable, who they thought should complete outcomes, opinions about invasive tests and travel distances to a research site.