Alzheimer’s Association. A public health approach to Alzheimer’s and other dementias. Alzheimer’s Association; 2016. Module 1. https://www.alz.org/media/documents/public-health-approach-alzheimers-faculty-guide-1.pdf. Accessed 15 June 2021.
Carmona R, Elders J, Novello A, Satchher D. U.S. surgeons general: dementia is our top public health crisis. Commentary. Us Against Alzheimer’s. October 10, 2019. https://www.usagainstalzheimers.org/blog/us-surgeons-general-dementia-our-top-public-health-crisis-commentary. Accessed 15 June 2021.
Resolution recognizing Alzheimer’s disease as a public health crisis impacting the nation’s health care infrastructure. National Lieutenant Governors Association; 2018. https://nlga.us/wp-content/uploads/Resolution-Recognizing-Alzheimer%E2%80%99s-Disease-as-a-Public-Health-Crisis-Impacting-the-Nation%E2%80%99s-Health-Care-Infrastructure.pdf. Accessed 15 June 2021.
Alzheimer's disease facts and figures [published online ahead of print, 2020 Mar 10]. Alzheimers Dement. 2020;2020. https://doi.org/10.1002/alz.12068.
Scheltens P, De Strooper B, Kivipelto M, Holstege H, Chételat G, Teunissen CE, et al. Alzheimer's disease. Lancet. 2021;397:1577–90.
Article
CAS
PubMed
Google Scholar
Siemers E, Holdridge KC, Sundell KL, Liu-Seifert H. Function and clinical meaningfulness of treatments for mild Alzheimer’s disease. Alzheimers Dement (Amst). 2016;2:105–12.
Article
Google Scholar
Leber P. Guidelines for the clinical evaluation of antidementia drugs (1990 draft). Rockville, MD: US Department of Health and Human Services. Food Drug Adm. 1990. https://doi.org/10.13140/2.1.4164.9445.
Blennow K, Zetterberg H. Biomarkers for Alzheimer’s disease: current status and prospects for the future. J Intern Med. 2018;284:643–63.
Article
CAS
PubMed
Google Scholar
Cummings J, Fox N. Defining disease modifying therapy for Alzheimer’s disease. J Prev Alzheimers Dis. 2017;4:109–15.
CAS
PubMed
PubMed Central
Google Scholar
Guidance for Industry: Early Alzheimer’s Disease: Developing Drugs for Treatment. US Department of Health and Human Services, Food and Drug Administration, Washington, DC; 2018. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/alzheimers-disease-developing-drugs-treatment-guidance-industy. Accessed 19 Oct 2020.
Jack CR Jr, Bennett DA, Blennow K, Carrillo MC, Dunn B, Haeberlein SB, et al. NIA-AA Research Framework: Toward a biological definition of Alzheimer’s disease. Alzheimers Dement. 2018;14(4):535–62.
Article
PubMed
PubMed Central
Google Scholar
Rosen WG, Mohs RC, Davis KL. A new rating scale for Alzheimer’s disease. Am J Psychiatry. 1984;141:1356–64.
Article
CAS
PubMed
Google Scholar
Webster L, Groskreutz D, Grinbergs-Saull A, Howard R, O'Brien JT, Mountain G, et al. Core outcome measures for interventions to prevent or slow the progress of dementia for people living with mild to moderate dementia: systematic review and consensus recommendations. PLoS One. 2017;12:e0179521.
Article
CAS
PubMed
PubMed Central
Google Scholar
Silverberg NB, Ryan LM, Carrillo MC, Sperling R, Petersen RC, Posner HB, et al. Assessment of cognition in early dementia. Alzheimers Dement. 2011;7:e60–76.
Article
PubMed
PubMed Central
Google Scholar
Weintraub S, Carrillo MC, Farias ST, Goldberg TE, Hendrix JA, Jaeger J, et al. Measuring cognition and function in the preclinical stage of Alzheimer’s disease. Alzheimers Dement (N Y). 2018;4:64–75.
Article
Google Scholar
Schneider LS, Goldberg TE. Composite cognitive and functional measures for early stage Alzheimer’s disease trials. Alzheimers Dement (Amst). 2020;12:e12017.
Google Scholar
Donohue MC, Sperling RA, Petersen P, Sun KC, Weiner MW, Aisen PS. Alzheimer’s Disease Neuroimaging Initiative. Association between elevated brain amyloid and subsequent cognitive decline among cognitively normal persons. JAMA. 2017;317:2305–16.
Article
CAS
PubMed
PubMed Central
Google Scholar
Sperling RA, Donohue MC, Raman R, Sun CK, Yaari R, Holdridge K, et al. Association of factors with elevated amyloid burden in clinically normal older individuals. JAMA Neurol. 2020;77:735–45.
Article
PubMed
Google Scholar
Tariot PN, Lopera F, Langbaum JB, Thomas RG, Hendrix S, Schneider LS, et al. The Alzheimer’s Prevention Initiative Autosomal-Dominant Alzheimer’s Disease Trial: a study of crenezumab versus placebo in preclinical PSEN1 E280A mutation carriers to evaluate efficacy and safety in the treatment of autosomal-dominant Alzheimer’s disease, including a placebo-treated noncarrier cohort. Alzheimers Dement (N Y). 2018;4:150–60.
Article
Google Scholar
Ayutyanont N, Langbaum JB, Hendrix SB, Chen K, Fleisher AS, Friesenhahn M, et al. The Alzheimer's prevention initiative composite cognitive test score: sample size estimates for the evaluation of preclinical Alzheimer’s disease treatments in presenilin 1 E280A mutation carriers. J Clin Psychiatry. 2014;75:652–60.
Article
PubMed
PubMed Central
Google Scholar
Robert P, Ferris S, Gauthier S, Ihl R, Winblad B, Tennigkeit F. Review of Alzheimer’s disease scales: is there a need for a new multi-domain scale for therapy evaluation in medical practice? Alzheimers Res Ther. 2010;2:24.
Article
PubMed
PubMed Central
Google Scholar
Kueper JK, Speechley M, Montero-Odasso M. The Alzheimer’s Disease Assessment Scale-Cognitive Subscale (ADAS-Cog): modifications and responsiveness in pre-dementia populations. a narrative review. J Alzheimers Dis. 2018;63:423–44.
Article
PubMed
PubMed Central
Google Scholar
Harrison J, Minassian SL, Jenkins L, Black RS, Koller M, Grundman M. A neuropsychological test battery for use in Alzheimer disease clinical trials. Arch Neurol. 2007;64:1323–9.
Article
PubMed
Google Scholar
Harrison J, Rentz DM, McLaughlin T, Niecko T, Gregg KM, Black RS, et al. Cognition in MCI and Alzheimer’s disease: baseline data from a longitudinal study of the NTB. Clin Neuropsychol. 2014;28:252–68.
Article
PubMed
Google Scholar
Harrison JE, Rentz DM, Brashear HR, Arrighi HM, Ropacki MT, Liu E. Psychometric evaluation of the Neuropsychological Test Battery in individuals with normal cognition, mild cognitive impairment, or mild to moderate Alzheimer’s disease: results from a longitudinal study. J Prev Alzheimers Dis. 2018;5:236–44.
CAS
PubMed
Google Scholar
O'Bryant SE, Lacritz LH, Hall J, Waring SC, Chan W, Khodr ZG, et al. Validation of the new interpretive guidelines for the Clinical Dementia Rating Scale Sum of Boxes Score in the national Alzheimer's coordinating center database. Arch Neurol. 2010;67:746–9.
PubMed
PubMed Central
Google Scholar
Wang J, Logovinsky V, Hendrix SB, Stanworth SH, Perdomo C, Xu L, et al. ADCOMS: A composite clinical outcome for prodromal Alzheimer’s disease trials. J Neurol Neurosurg Psychiatry. 2016;87:993–9.
Article
PubMed
Google Scholar
Wessels AM, Andersen SW, Dowsett SA, Siemers ER. The integrated Alzheimer’s Disease Rating Scale (iADRS) findings from the EXPEDITION3 Trial. J Prev Alzheimers Dis. 2018;5:134–6.
CAS
PubMed
Google Scholar
Goldberg TE, Koppel J, Keehlisen L, Christen E, Dreses-Werringloer U, Conejero-Goldberg C, et al. Performance-based measures of everyday function in mild cognitive impairment. Am J Psychiatry. 2010;167:845–53.
Article
PubMed
PubMed Central
Google Scholar
Marshall GA, Dekhtyar M, Bruno JM, Jethwani K, Amariglio RE, Johnson KA, et al. The Harvard Automated Phone Task: new performance-based activities of daily living tests for early Alzheimer's disease. J Prev Alzheimers Dis. 2015;2:242–53.
PubMed
PubMed Central
Google Scholar
Atkins AS, Khan A, Ulshen D, Vaughan A, Balentin D, Dickerson H, et al. Assessment of instrumental activities of daily living in older adults with subjective cognitive decline using the Virtual Reality Functional Capacity Assessment Tool (VRFCAT). J Prev Alzheimers Dis. 2018;5:216–34.
CAS
PubMed
Google Scholar
Marson DC, Gerstenecker A, Triebel K, Martin R, Christianson R, Swenson-Dravis D, et al. Detecting functional impairment in preclinical Alzheimer’s disease using a brief performance measure of financial skills. Alzheimers Dement. 2016;12:373.
Article
Google Scholar
Farias ST, Mungas D, Reed BR, Cahn-Weiner D, Jagust W, Baynes K, et al. The measurement of everyday cognition (ECog): scale development and psychometric properties. Neuropsychology. 2008;22:531–44.
Article
PubMed
PubMed Central
Google Scholar
Li C, Neugroschl J, Luo X, Zhu C, Aisen P, Ferris S, et al. The utility of the Cognitive Function Instrument (CFI) to detect cognitive decline in non-demented older adults. J Alzheimers Dis. 2019;60:427–37.
Article
Google Scholar
Amariglio RE, Sikkes SAM, Marshall GA, Buckley RF, Gatchel JR, Johnson KA, et al. Item-level investigation of participant and study partner report on the cognitive function index from the A4 study screening data. J Prev Alzheimers Dis. 2021;8:257–62.
CAS
PubMed
PubMed Central
Google Scholar
Galasko D, Bennett DA, Sano M, Marson D, Kaye J, Edland SD. ADCS Prevention Instrument Project: assessment of instrumental activities of daily living for community-dwelling elderly individuals in dementia prevention clinical trials. Alzheimer Dis Assoc Disord. 2006;20(4 Suppl 3):S152–69.
Article
PubMed
Google Scholar
Marshall GA, Sikkes SAM, Amariglio RE, Gatchel JR, Rentz DM, Johnson KA, et al. Instrumental activities of daily living, amyloid, and cognition in cognitively normal older adults screening for the A4 Study. Alzheimers Dement (Amst). 2020;12:e12118.
Google Scholar
Frank L, Lenderking WR, Howard K, Cantillon M. Patient self-report for evaluating mild cognitive impairment and prodromal Alzheimer's disease. Alzheimers Res Ther. 2011;3:35.
Article
PubMed
PubMed Central
Google Scholar
Pedrosa H, De Sa A, Guerreiro M, Maroco J, Simoes MR, Galasko D, et al. Functional evaluation distinguishes MCI patients from healthy elderly people--the ADCS/MCI/ADL scale. J Nutr Health Aging. 2010;14:703–9.
Article
CAS
PubMed
Google Scholar
Sikkes SA, De Lange-de Klerk ES, Pijnenburg YA, Gillissen F, Romkes R, Knol DL, et al. A new informant-based questionnaire for instrumental activities of daily living in dementia. Alzheimers Dement. 2012;8:536–43.
Article
PubMed
Google Scholar
Koster N, Knol DL, Uitdehaag BM, Scheltens P, Sikkes SA. The sensitivity to change over time of the Amsterdam IADL Questionnaire(©). Alzheimers Dement. 2015;11:1231–40.
Article
PubMed
Google Scholar
Jutten RJ, Peeters CFW, Leijdesdorff SMJ, Visser PJ, Maier AB, Terwee CB, et al. Detecting functional decline from normal aging to dementia: Development and validation of a short version of the Amsterdam IADL Questionnaire. Alzheimers Dement (Amst). 2017;8:26–35.
Article
Google Scholar
Papp KV, Buckley R, Mormino E, Maruff P, Villemagne VL, Masters CL, et al. Clinical meaningfulness of subtle cognitive decline on longitudinal testing in preclinical AD. Alzheimers Dement. 2020;16:552–60.
Article
PubMed
PubMed Central
Google Scholar
Hung A, Schneider M, Lopez MH, McClellan M. Preclinical Alzheimer disease drug development: early considerations based on phase 3 clinical trials. J Manag Care Spec Pharm. 2020;26(7):888–900.
PubMed
Google Scholar
Weldring T, Smith SM. Patient-reported outcomes (PROs) and patient-reported outcome measures (PROMs). Health Serv Insights. 2013;6:61–8.
PubMed
PubMed Central
Google Scholar
Watson J, Saunders S, Muniz Terrera G, Ritchie C, Evans A, Luz S, et al. What matters to people with memory problems, healthy volunteers and health and social care professionals in the context of developing treatment to prevent Alzheimer's dementia? A qualitative study. Health Expect. 2019;22:504–17.
Article
PubMed
PubMed Central
Google Scholar
Hauber B, Paulsen R, Callahan L, Potashman M, Lee D, Hartry A, et al. Quantifying what matters most to patients and care partners in Alzheimer’s disease. Poster presented at: 2020 Alzheimer’s Association Virtual International Conference (AAIC); 2020.
Google Scholar
Hartry A, Menne H, Wronski S, Paulsen R, Callahan L, Potashman M, et al. Evaluation of what matters most in existing clinical outcomes assessments in Alzheimer’s disease. Poster presented at: 2020 Alzheimer’s Association Virtual International Conference (AAIC); 2020.
Google Scholar
Gordon MF, Lenderking WR, Duhig A, Chandler J, Lundy JJ, Miller DS, et al. 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. 2016;12:75–84.
Article
PubMed
Google Scholar
Saunders S, Muniz-Terrera G, Sheehan S, Ritchie CW, Luz S. A UK-wide study employing natural language processing to determine what matters to people about brain health to improve drug development: The Electronic Person-Specific Outcome Measure (ePSOM) Programme. J Prev Alzheimers Dis. 2021;8:448–56.
CAS
PubMed
Google Scholar
Cognition Working Group. Prepared for: 11th Annual PRO Consortium workshop; April 22-23, 2020. PRO Consortium Critical Path Institute. https://c-path.org/wp-content/uploads/2020/11/2020_Cognition_WG_Poster.pdf Accessed 20 Oct 2021.
Mercieca-Bebber R, King MT, Calvert MJ, Stockler MR, Friedlander M. The importance of patient-reported outcomes in clinical trials and strategies for future optimization. Patient Relat Outcome Meas. 2018;9:353–67.
Article
PubMed
PubMed Central
Google Scholar
Patient-Focused Drug Development: Collecting Comprehensive and Representative Input Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders. US Department of Health and Human Services, Food and Drug Administration, Washington, DC; 2018. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drug-development-collecting-comprehensive-and-representative-input. Accessed 28 Sept 2021.
El-Hayek YH, Wiley RE, Khoury CP, Daya RP, Ballard C, Evans AR, et al. Tip of the iceberg: assessing the global socioeconomic costs of Alzheimer's disease and related dementias and strategic implications for stakeholders. J Alzheimers Dis. 2019;70(2):323–41.
Article
PubMed
PubMed Central
Google Scholar
Wimo A, Gustavsson A, Jönsson L, Winblad B, Hsu MA, Gannon B. Application of resource utilization in dementia (RUD) instrument in a global setting. Alzheimers Dement. 2013;9(4):429–35.
Article
PubMed
Google Scholar
Robinson RL, Rentz DM, Andrews JS, Zagar A, Kim Y, Bruemmer V, et al. Costs of early stage Alzheimer's disease in the United States: cross-sectional analysis of a prospective cohort study (GERAS-US)1. J Alzheimers Dis. 2020;75(2):437–50.
Article
PubMed
PubMed Central
Google Scholar
Cummings JL. The Neuropsychiatric Inventory: assessing psychopathology in dementia patients. Neurology. 1997;48:S10–6.
Article
CAS
PubMed
Google Scholar
Ismail Z, Agüera-Ortiz L, Brodaty H, Cieslak A, Cummings J, Fischer CE, et al. The Mild Behavioral Impairment Checklist (MBI-C): a rating scale for neuropsychiatric symptoms in pre-dementia populations. J Alzheimers Dis. 2017;56(3):929–38.
Article
PubMed
PubMed Central
Google Scholar
Jessen F. What are we trying to prevent in Alzheimer disease? Dialogues Clin Neurosci. 2019;21(1):27–34.
Article
PubMed
PubMed Central
Google Scholar
Lanctôt KL, Amatniek J, Ancoli-Israel S, et al. Neuropsychiatric signs and symptoms of Alzheimer's disease: New treatment paradigms. Alzheimers Dement (N Y). 2017;3(3):440–9.
Article
Google Scholar
Ismail Z, Smith EE, Geda Y, et al. Neuropsychiatric symptoms as early manifestations of emergent dementia: Provisional diagnostic criteria for mild behavioral impairment. Alzheimers Dement. 2016;12(2):195–202.
Article
PubMed
Google Scholar
Liew TM. Neuropsychiatric symptoms in early stage of Alzheimer's and non-Alzheimer's dementia, and the risk of progression to severe dementia. Age Ageing. 2021;50(5):1709–18.
Article
PubMed
PubMed Central
Google Scholar
Eikelboom WS, van den Berg E, Singleton EH, Baart SJ, Coesmans M, Leeuwis AE, et al. Neuropsychiatric and cognitive symptoms across the Alzheimer Disease clinical spectrum: cross-sectional and longitudinal associations. Neurology. 2021;97(13):e1276–87.
Article
CAS
PubMed
PubMed Central
Google Scholar
Peters KR. Utility of an effect size analysis for communicating treatment effectiveness: a case study of cholinesterase inhibitors for Alzheimer's disease. J Am Geriatr Soc. 2013;61:1170–4.
Article
PubMed
Google Scholar
Keefe RS, Kraemer HC, Epstein RS, Frank E, Haynes G, Laughren TP, et al. Defining a clinically meaningful effect for the design and interpretation of randomized controlled trials. Innov Clin Neurosci. 2013;10(5-6 Suppl A):4S–19S.
PubMed
PubMed Central
Google Scholar
McGough JJ, Faraone SV. Estimating the size of treatment effects: moving beyond p values. Psychiatry (Edgmont). 2009;6:21–9.
Google Scholar
Schuele CM, Justice LM. The importance of effect sizes in the interpretation of research. Primer on Research: Part 3. ASHA Leader. 2006;11(10) https://leader.pubs.asha.org/doi/full/10.1044/leader.FTR4.11102006.14. Accessed 15 June 2021.
Atri A, Shaughnessy LW, Locascio JJ, Growdon JH. Long-term course and effectiveness of combination therapy in Alzheimer disease. Alzheimer Dis Assoc Disord. 2008;22:209–21.
Article
CAS
PubMed
PubMed Central
Google Scholar
Nordahl-Hansen A, Øien RA, Volkmar F, Shic F, Cicchetti DV. Enhancing the understanding of clinically meaningful results: A clinical research perspective. Psychiatry Res. 2018;270:801–6.
Article
PubMed
Google Scholar
Page P. Beyond statistical significance: clinical interpretation of rehabilitation research literature. Int J Sports Phys Ther. 2014;9:726–36.
PubMed
PubMed Central
Google Scholar
Siwek J, Newman DH. Introducing medicine by the numbers: a collaboration of the NNT Group and AFP. Am Fam Physician. 2015;91:434–5.
PubMed
Google Scholar
Edgar CJ, Vradenburg G, Hassenstab J. The 2018 revised FDA guidance for early Alzheimer's disease: establishing the meaningfulness of treatment effects. J Prev Alzheimers Dis. 2019;6:223–7.
CAS
PubMed
Google Scholar
Rockwood K. Size of the treatment effect on cognition of cholinesterase inhibition in Alzheimer’s disease [published correction appears in J Neurol Neurosurg Psychiatry. 2004;75(7):1086]. J Neurol Neurosurg Psychiatry. 2004;75:677–85.
Smith M, Wells J, Borrie M. Treatment effect size of memantine therapy in Alzheimer disease and vascular dementia. Alzheimer Dis Assoc Disord. 2006;20:133–7.
Article
CAS
PubMed
Google Scholar
Atri A, Rountree SD, Lopez OL, Doody RS. Validity, significance, strengths, limitations, and evidentiary value of real-world clinical data for combination therapy in Alzheimer’s disease: comparison of efficacy and effectiveness studies. Neurodegener Dis. 2012;10(1-4):170–4.
Article
CAS
PubMed
PubMed Central
Google Scholar
Gensini GF, Conti AA. Evidence-based evaluation of benefits in therapeutic interventions: methodologically controlled and non-randomly assigned reflections on the number needed to treat. Ital Heart J. 2003;4:80–3.
PubMed
Google Scholar
Watt JA, Veroniki AA, Tricco AC, Straus SE. Using a distribution-based approach and systematic review methods to derive minimum clinically important differences. BMC Med Res Methodol. 2021;21:41.
Article
PubMed
PubMed Central
Google Scholar
McMichael AJ, Rolison JJ, Boeri M, Kane JPM, O'Neill FA, Kee F. How do psychiatrists apply the minimum clinically important difference to assess patient responses to treatment? MDM Policy Pract. 2016;1:2381468316678855.
PubMed
PubMed Central
Google Scholar
Patient-Focused Drug Development Guidance Public Workshop: Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision-Making. US Department of Health and Human Services, Food and Drug Administration, Washington, DC; 2019. https://www.fda.gov/media/132505/download. Accessed 21 Oct 2021.
Liu KY, Schneider LS, Howard R. The need to show minimum clinically important differences in Alzheimer’s disease trials. Lancet Psychiatry. 2021;8(11):1013–6.
Article
PubMed
Google Scholar
Cummings JL, Zhong K. Clinical trials and drug development in neurodegenerative diseases: unifying principles. In: Cummings JL, Pillai JA, editors. Neurodegenerative Diseases Unifying Principles. Oxford: Oxford University Press; 2017. p. 323–36.
Google Scholar
Halpin DM, Tashkin DP. Defining disease modification in chronic obstructive pulmonary disease. COPD. 2009;6:211–25.
Article
PubMed
Google Scholar
Amato MP, Fonderico M, Portaccio E, Pastò L, Razzolini L, Prestipino E, et al. Disease-modifying drugs can reduce disability progression in relapsing multiple sclerosis. Brain. 2020;143:3013–24.
Article
PubMed
Google Scholar
Cummings JL. Challenges to demonstrating disease-modifying effects in Alzheimer's disease clinical trials. Alzheimers Dement. 2006;2:263–71.
Article
PubMed
PubMed Central
Google Scholar
Claflin SB, Broadley S, Taylor BV. The effect of disease modifying therapies on disability progression in multiple sclerosis: a systematic overview of meta-analyses. Front Neurol. 2019;9:1150.
Article
PubMed
PubMed Central
Google Scholar
Kalincik T, Diouf I, Sharmin S, Malpas C, Spelman T, Horakova D, et al. Effect of disease-modifying therapy on disability in relapsing-remitting multiple sclerosis over 15 years. Neurology. 2021;96:e783–97.
Article
CAS
PubMed
PubMed Central
Google Scholar
Harding K, Williams O, Willis M, Hrastelj J, Rimmer A, Joseph F, et al. Clinical outcomes of escalation vs early intensive disease-modifying therapy in patients with multiple sclerosis. JAMA Neurol. 2019;76:536–41.
Article
PubMed
PubMed Central
Google Scholar
Cerqueira JJ, Compston DAS, Geraldes R, Rosa MM, Schmierer K, Thompson A, et al. Time matters in multiple sclerosis: can early treatment and long-term follow-up ensure everyone benefits from the latest advances in multiple sclerosis? J Neurol Neurosurg Psychiatry. 2018;89:844–50.
Article
PubMed
Google Scholar
Beach TG. A review of biomarkers for neurodegenerative disease: will they swing us across the valley? Neurol Ther. 2017;6(Suppl 1):5–13.
Article
PubMed
PubMed Central
Google Scholar
McGhee DJ, Ritchie CW, Thompson PA, Wright DE, Zajicek JP, Counsell CE. A systematic review of biomarkers for disease progression in Alzheimer’s disease [published correction appears in PLoS One. 2014;9(5):e97960]. PLoS One. 2014;9:e88854.
Article
CAS
PubMed
PubMed Central
Google Scholar
Twaddell S. Surrogate outcome markers in research and clinical practice. Aust Prescri. 2009;32:47–50.
Article
Google Scholar
Martin JH, Fay MF. Surrogate end-points in clinical practice: are we providing worse care? Intern Med J. 2010;40:395–8.
Article
CAS
PubMed
Google Scholar
Surrogate endpoint resources for drug and biologic development. US Department of Health and Human Services, Food and Drug Administration, Washington, DC; updated July 24, 2018. https://www.fda.gov/drugs/development-resources/surrogate-endpoint-resources-drug-and-biologic-development. Accessed 15 June 2021.
Cummings J, Aisen P, Lemere C, Atri A, Sabbagh M, Salloway S. Aducanumab produced a clinically meaningful benefit in association with amyloid lowering. Alzheimers Res Ther. 2021;13:98.
Article
PubMed
PubMed Central
Google Scholar
Lawrence E, Vegvari C, Ower A, Hadjichrysanthou C, De Wolf F, Anderson RM. A systematic review of longitudinal studies which measure Alzheimer’s disease biomarkers. J Alzheimers Dis. 2017;59:1359–79.
Article
CAS
PubMed
PubMed Central
Google Scholar
Weiner MW, Veitch DP, Aisen PS, Beckett LA, Cairns NJ, Green RC, et al. Recent publications from the Alzheimer’s Disease Neuroimaging Initiative: reviewing progress toward improved AD clinical trials. Alzheimers Dement. 2017;13:e1–e85.
Article
PubMed
PubMed Central
Google Scholar
Cullen NC, Leuzy A, Janelidze S, Palmqvist S, Svenningsson AL, Stomrud E, et al. Plasma biomarkers of Alzheimer's disease improve prediction of cognitive decline in cognitively unimpaired elderly populations. Nat Commun. 2021;12(1):3555.
Article
CAS
PubMed
PubMed Central
Google Scholar
Hansson O, Cullen N, Zetterberg H. Alzheimer’s Disease Neuroimaging Initiative, Blennow K, Mattsson-Carlgren N. Plasma phosphorylated tau181 and neurodegeneration in Alzheimer’s disease. Ann Clin Transl Neurol. 2021;8(1):259–65.
Article
CAS
PubMed
Google Scholar
Cash DM, Rohrer JD, Ryan NS, Ourselin S, Fox NC. Imaging endpoints for clinical trials in Alzheimer's disease. Alzheimers Res Ther. 2014;6:87.
Article
CAS
PubMed
PubMed Central
Google Scholar
Gaetani L, Parnetti L, Calabresi P, Di Filippo M. Tracing neurological diseases in the presymptomatic phase: insights from neurofilament light chain. Front Neurosci. 2021;15:672954.
Article
PubMed
PubMed Central
Google Scholar
Robertson D, Moreo N. Disease-modifying therapies in multiple sclerosis: overview and treatment considerations. Fed Pract. 2016;33(6):28–34.
PubMed
PubMed Central
Google Scholar