What Outcomes Are Important to Patients with Mild Cognitive Impairment or Alzheimer's Disease, Their Caregivers, and Health-care Professionals? A systematic Review

Overview

Journal Alzheimers Dement (Amst)

Date 2019 Mar 26

PMID 30906845

Citations 38

Authors

Claire Tochel

Michael Smith

Helen Baldwin

Anders Gustavsson

Amanda Ly

Christin Bexelius

Mia Nelson

Christophe Bintener

Enrico Fantoni

Josep Garre-Olmo

Olin Janssen

Christoph Jindra

Isabella F Jorgensen

Alex McKeown

Buket Ozturk

Anna Ponjoan

Michele H Potashman

Catherine Reed

Emilse Roncancio-Diaz

Stephanie Vos

Cathie Sudlow

Affiliations

Soon will be listed here.

Abstract

Introduction: Clinical trials involving patients with Alzheimer's disease (AD) continue to try to identify disease-modifying treatments. Although trials are designed to meet regulatory and registration requirements, many do not measure outcomes of the disease most relevant to key stakeholders.

Methods: A systematic review sought research that elicited information from people with AD, their caregivers, and health-care professionals on which outcomes of the disease were important. Studies published in any language between 2008 and 2017 were included.

Results: Participants in 34 studies described 32 outcomes of AD. These included clinical (memory, mental health), practical (ability to undertake activities of daily living, access to health information), and personal (desire for patient autonomy, maintenance of identity) outcomes of the disease.

Discussion: Evidence elicited directly from the people most affected by AD reveals a range of disease outcomes that are relevant to them but are not commonly captured in clinical trials of new treatments.

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References

Dai B, Mao Z, Mei J, Levkoff S, Wang H, Pacheco M . Caregivers in China: knowledge of mild cognitive impairment. PLoS One. 2013; 8(1):e53928. PMC: 3543358. DOI: 10.1371/journal.pone.0053928. View

MacRae H . Managing identity while living with Alzheimer's disease. Qual Health Res. 2009; 20(3):293-305. DOI: 10.1177/1049732309354280. View

Harrison J, Noel-Storr A, Demeyere N, Reynish E, Quinn T . Outcomes measures in a decade of dementia and mild cognitive impairment trials. Alzheimers Res Ther. 2016; 8(1):48. PMC: 5116815. DOI: 10.1186/s13195-016-0216-8. View

Dean K, Jenkinson C, Wilcock G, Walker Z . Exploring the experiences of people with mild cognitive impairment and their caregivers with particular reference to healthcare - a qualitative study. Int Psychogeriatr. 2013; 26(3):475-85. DOI: 10.1017/S104161021300207X. View

Webster L, Groskreutz D, Grinbergs-Saull A, Howard R, OBrien J, Mountain G . 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. Health Technol Assess. 2017; 21(26):1-192. PMC: 5494514. DOI: 10.3310/hta21260. View

Yektatalab S, Sharif F, Kaveh M, Fallahi Khoshknab M, Petramfar P . Living with and caring for patients with Alzheimer's disease in nursing homes. J Caring Sci. 2014; 2(3):187-95. PMC: 4134156. DOI: 10.5681/jcs.2013.023. View

MacRae H . 'Making the best you can of it': living with early-stage Alzheimer's disease. Sociol Health Illn. 2008; 30(3):396-412. DOI: 10.1111/j.1467-9566.2007.01056.x. View

Posner H, Curiel R, Edgar C, Hendrix S, Liu E, Loewenstein D . Outcomes Assessment in Clinical Trials of Alzheimer's Disease and its Precursors: Readying for Short-term and Long-term Clinical Trial Needs. Innov Clin Neurosci. 2017; 14(1-2):22-29. PMC: 5373792. View

Gordon M, Lenderking W, Duhig A, Chandler J, Lundy J, Miller D . Development of a patient-reported outcome instrument to assess complex activities of daily living and interpersonal functioning in persons with mild cognitive impairment: The qualitative research phase. Alzheimers Dement. 2015; 12(1):75-84. DOI: 10.1016/j.jalz.2015.04.008. View

10.

Malthouse R, Fox F . Exploring experiences of physical activity among people with Alzheimer's disease and their spouse carers: a qualitative study. Physiotherapy. 2014; 100(2):169-75. DOI: 10.1016/j.physio.2013.10.002. View

11.

Cheng S, Mak E, Lau R, Ng N, Lam L . Voices of Alzheimer Caregivers on Positive Aspects of Caregiving. Gerontologist. 2015; 56(3):451-60. DOI: 10.1093/geront/gnu118. View

12.

Lu Y, Haase J . Content validity and acceptability of the daily enhancement of meaningful activity program: intervention for mild cognitive impairment patient-spouse dyads. J Neurosci Nurs. 2011; 43(6):317-28. PMC: 3220889. DOI: 10.1097/JNN.0b013e318234e9dd. View

13.

Lu Y, Haase J . Experience and perspectives of caregivers of spouse with mild cognitive impairment. Curr Alzheimer Res. 2009; 6(4):384-91. PMC: 2867606. DOI: 10.2174/156720509788929309. View

14.

Ropacki M, Hannesdottir K, Hendrix S, Gordon M, Stephenson D, Coons S . Clinically Meaningful Outcomes in Early Alzheimer Disease: A Consortia-Driven Approach to Identifying What Matters to Patients. Ther Innov Regul Sci. 2018; 51(3):380-390. DOI: 10.1177/2168479016689712. View

15.

Makady A, de Boer A, Hillege H, Klungel O, Goettsch W . What Is Real-World Data? A Review of Definitions Based on Literature and Stakeholder Interviews. Value Health. 2017; 20(7):858-865. DOI: 10.1016/j.jval.2017.03.008. View

16.

Rothrock N, Kaiser K, Cella D . Developing a valid patient-reported outcome measure. Clin Pharmacol Ther. 2011; 90(5):737-42. PMC: 3753201. DOI: 10.1038/clpt.2011.195. View

17.

Jack Jr C, Bennett D, Blennow K, Carrillo M, Dunn B, Budd Haeberlein S . NIA-AA Research Framework: Toward a biological definition of Alzheimer's disease. Alzheimers Dement. 2018; 14(4):535-562. PMC: 5958625. DOI: 10.1016/j.jalz.2018.02.018. View

18.

Hauber A, Mohamed A, Reed Johnson F, Cook M, Arrighi H, Zhang J . Understanding the relative importance of preserving functional abilities in Alzheimer's disease in the United States and Germany. Qual Life Res. 2014; 23(6):1813-21. DOI: 10.1007/s11136-013-0620-5. View

19.

Oremus M, Tarride J, Pullenayegum E, Clayton N, Mugford G, Godwin M . Caregivers' willingness-to-pay for Alzheimer's disease medications in Canada. Dementia (London). 2013; 14(1):63-79. DOI: 10.1177/1471301213490709. View

20.

Cano S, Posner H, Moline M, Hurt S, Swartz J, Hsu T . The ADAS-cog in Alzheimer's disease clinical trials: psychometric evaluation of the sum and its parts. J Neurol Neurosurg Psychiatry. 2010; 81(12):1363-8. DOI: 10.1136/jnnp.2009.204008. View