International Psychogeriatric Association consensus statement on defining and measuring treatment benefits in dementia

Abstract

Current symptomatic treatments for dementia have only modest efficacy. Assessing meaningful benefits in this variably progressive syndrome is complex and difficult. This consensus statement was generated by an international group representing caregivers, organizations and professionals with expertise in dementia. We recommend the statement of clear, pre-defined diagnostic and severity criteria and outcome measures, which include functional and executive capacity, in treatment trials. Outcomes can include effects on people with dementia (PWD) with regard to cognition, behavioral and psychological symptoms, quality of life, global assessments, and activities of daily living, and must be tailored to the education and culture of the participants. Outcomes can also appropriately encompass effects on caregivers. New instruments may be needed, as validation of many existing measures has been in relatively homogenous populations. Treatment goals can be to prevent dementia emerging, or in those with established dementia to slow deterioration, and to postpone, stabilize or improve symptoms. Comparisons between treatment groups should be on the basis of clinically relevant measures with both risk and benefit reported for all participants regardless of whether or not they continue to receive treatment throughout the trial. Characterization of any groups that respond well to treatment has been unsuccessful to date, but may be facilitated in the future by measurement of putative biomarkers. Despite considerable recent progress and several 'candidate' biomarkers, none is yet satisfactory for determining diagnosis, severity, progression or prediction of response. To provide meaningful data, economic analyses should use up-to-date, country-specific data. Health economic measures should be incorporated as secondary outcomes in all Phase 3 trials since health systems are concerned with cost-effectiveness as well as clinical outcome. Health utility measures are not, however, validated satisfactorily in dementia, thus calling into question previous health economic analyses. While current drugs appear to reduce the amount of family caregiver time required by PWD, these costs fall in the main on older individuals who often exert little political leverage, rather than on society at large. Traditionally, elderly people have been marginalized in the political process. The growth in the older population across the world, and their potential for increasing political empowerment may lead to a radical re-evaluation of the economics of treatment in dementia.

Discrepancies Regarding the Quality of Life of Patients with Alzheimer's Disease: A Three-Year Longitudinal Study

“…Although their caregivers may share these concerns, they are also con-cerned with other more therapeutic issues, such as the medication and treatment that are available [1]. Evaluating the ability of therapeutic interventions to improve the quality of life of patients (QoL-p) with AD is now recognized as an important issue [2][3][4][5].…”

Generic and Disease-Specific Measures of Quality of Life in Patients with Mild Alzheimer’s Disease

“…Cognitive performance, activities of daily living (ADL) and global improvement have been used widely as outcome measures in clinical trials of dementia and Alzheimer's disease (AD) [2] . However, dementia has a broad social impact in the form of years lived with disability, and thus, there is a growing need to include psychological dimensions as outcome targets in both pharmacological and psychosocial intervention studies [4] . In this regard, quality of life (QoL) has gained prominence as a new dimension of outcome measurement in AD clin-ical trials [5] .…”

“…In this regard, quality of life (QoL) has gained prominence as a new dimension of outcome measurement in AD clin-ical trials [5] . Consensus reports that focus on endpoints of clinical trials have now recognized QoL as a significant psychosocial outcome measure in AD [2,4] .…”

Review of Goal Attainment Scaling as a Useful Outcome Measure in Psychogeriatric Patients with Cognitive Disorders

“…There is an ongoing debate about the best way to measure clinically relevant outcomes of the treatment of dementia [2][3][4] such as with cholinesterase inhibitors [5,6] . Outcome measures should not only be responsive, reliable and valid, but should also be tailored to the personal goals and needs of patients and their caregivers in relation to their daily life.…”