Effect of APOE genotype status on targeted clinical trials outcomes and efficiency in dementia and mild cognitive impairment resulting from Alzheimer's disease

Alzheimer's and Dementia

Volumn 10, Issue 3, 2014, Pages 349-359

  Kennedy, Richard E ^a   Cutter, Gary R ^a   Schneider, Lon S ^b  

^a UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

Alzheimer disease; Alzheimer's Disease Assessment Scale; Alzheimer's Disease Cooperative Study; Alzheimer's Disease Neuroimaging Initiative; Apolipoprotein E 4; Biomarkers; Clinical trial simulations; Clinical trials; Mild cognitive impairment

Indexed keywords

APOLIPOPROTEIN E; APOLIPOPROTEIN E4; NOOTROPIC AGENT;

ALZHEIMER DISEASE; ARTICLE; DATA BASE; DEMENTIA; EFFECT SIZE; EXPLANATORY VARIABLE; GENOTYPE; HUMAN; MILD COGNITIVE IMPAIRMENT; PRIORITY JOURNAL; SCREENING; SIMULATION; TREATMENT PLANNING; AGED; CLINICAL TRIAL (TOPIC); COMPLICATION; COMPUTER SIMULATION; FACTUAL DATABASE; FEMALE; GENETICS; HETEROZYGOTE; MALE; PATIENT SELECTION; SEVERITY OF ILLNESS INDEX;

AGED; ALZHEIMER DISEASE; APOLIPOPROTEIN E4; CLINICAL TRIALS AS TOPIC; COMPUTER SIMULATION; DATABASES, FACTUAL; FEMALE; HETEROZYGOTE; HUMANS; MALE; MILD COGNITIVE IMPAIRMENT; NOOTROPIC AGENTS; PATIENT SELECTION; SEVERITY OF ILLNESS INDEX;

EID: 84899621111     PISSN: 15525260     EISSN: 15525279     Source Type: Journal    
DOI: 10.1016/j.jalz.2013.03.003     Document Type: Article

References (48)

1. Reitz C, Brayne C, Mayeux R. Epidemiology of Alzheimer disease. Nat Rev Neurol 2011;7:137-52.
2. Farlow MR, He Y, Tekin S, Xu J, Lane R, Charles HC. Impact of APOE in mild cognitive impairment. Neurology 2004;63:1898-901.
3. Linton MF, Gish R, Hubl ST, Butler E, Esquivel C, Bry WI, et al. Phenotypes of apolipoprotein B and apolipoprotein E after liver transplantation. J Clin Invest 1991;88:270-81.
4. Tokuda T, CaleroM, Matsubara E, Vidal R, Kumar A, Permanne B, et al. Lipidation of apolipoprotein E influences its isoform-specific interaction with Alzheimer's amyloid beta peptides. Biochem J 2000;348:359-65.
5. Aerssens J, Raeymaekers P, Lilienfeld S, Geerts H, Konings F, Parys W. APOE genotype: no influence on galantamine treatment efficacy nor on rate of decline in Alzheimer's disease. Dement Geriatr Cogn Disord 2001;12:69-77.
6. Blesa R, Aguilar M, Casanova JP, Boada M, Martinez S, Alom J, et al. Relationship between the efficacy of rivastigmine and apolipoprotein E (epsilon4) in patients with mild to moderately severe Alzheimer disease. Alzheimer Dis Assoc Disord 2006;20:248-54.
7. Poirier J, Delisle MC, Quirion R, Aubert I, Farlow M, Lahiri D, et al. Apolipoprotein E4 allele as a predictor of cholinergic deficits and treatment outcome in Alzheimer disease. Proc Natl Acad Sci U S A 1995;92:12260-4.
8. Rigaud A, Traykov L, Latour F, Couderc R, Moulin F, Forette F. Presence or absence of at least one epsilon 4 allele and gender are not predictive for the response to donepezil treatment in Alzheimer's disease. Pharmacogenetics 2002;12:415-20.
9. Lane R, Feldman HH, Meyer J, He Y, Ferris SH, Nordberg A, et al. Synergistic effect of apolipoprotein E epsilon4 and butyrylcholinesterase K-variant on progression from mild cognitive impairment to Alzheimer's disease. Pharmacogenet Genomics 2008;18:289-98.
10. Petersen RC, Thomas RG, Grundman M, Bennett D, Doody R, Ferris S, et al. Vitamin E and donepezil for the treatment of mild cognitive impairment. N Engl J Med 2005;352:2379-88.
11. Cummings JL. Alzheimer's disease clinical trials: changing the paradigm. Curr Psychiatry Rep 2011;13:437-42.
12. Salloway S, Sperling R, Gilman S, Fox NC, Blennow K, Raskind M, et al. A phase 2 multiple ascending dose trial of bapineuzumab in mild to moderate Alzheimer disease. Neurology 2009;73:2061-70.
13. Thal L. Development of the Alzheimer's Disease Cooperative Study. Int J Geriatr Psychopharmacol 1997;1:6-9.
14. Petersen RC, Aisen PS, Beckett LA, Donohue MC, Gamst AC, Harvey DJ, et al. Alzheimer's Disease Neuroimaging Initiative (ADNI): clinical characterization. Neurology 2010;74:201-9.
15. Mohs RC, Knopman D, Petersen RC, Ferris SH, Ernesto C, Grundman M, et al. Development of cognitive instruments for use in clinical trials of antidementia drugs: additions to the Alzheimer's Disease Assessment Scale that broaden its scope. Alzheimer Dis Assoc Disord 1997;11:S13-21.
16. McKhann G, Drachman D, Folstein M, Katzman R, Price D, Stadlan EM. Clinical diagnosis of Alzheimer's disease: report of the NINCDS-ADRDAWork Group under the auspices of Department of Health and Human Services Task Force on Alzheimer's Disease. Neurology 1984;34:939-44.
17. Morris JC. The Clinical Dementia Rating (CDR): current version and scoring rules. Neurology 1993;43:2412-4.
18. Folstein MF, Folstein S, McHugh P. Mini-mental state: a practical method for grading the cognitive state of patients for the clinician. J Psychiatry Res 1975;12:189-98.
19. Wechsler D.Wechsler Adult Intelligence Scale-revised. San Antonio, TX: Psychological Corporation; 1981.
20. Burton A, Altman DG, Royston P, Holder RL. The design of simulation studies in medical statistics. Stat Med 2006;25:4279-92.
21. Doody RS, Ferris SH, Salloway S, Sun Y, Goldman R, Watkins WE, et al. Donepezil treatment of patients with MCI: a 48-week randomized, placebo-controlled trial. Neurology 2009;72:1555-61.
22. Cohen J. Statistical power analysis for the behavioral sciences. 2nd ed. Hillsdale, NJ: L. Erlbaum Associates; 1988.
23. Brown H, Prescott R. Applied mixed models in medicine. 2nd ed. Chichester: Wiley; 2006.
24. Siddiqui O, Hung HMJ, O'Neill R. MMRM vs. LOCF: a comprehensive comparison based on simulation study and 25 NDA datasets. J Biopharmacol Stat 2009;19:227-46.
25. Schneider LS, Kennedy RE, Cutter GR. Estimating power with effect size versus slop differences: both means and variance matter. Alzheimers Dement 2011;7:247-9.
26. R Development Core Team. R: a language and environment for statistical computing. Vienna, Austria: R Foundation for Statistical Computing; 2010.
27. Pinheiro JC, Bates DM. Mixed-effects models in S and S-PLUS. New York: Springer; 2000.
28. Schneider LS, Kennedy RE, Cutter GR. Alzheimer's Disease Neuroimaging Initiative: requiring an amyloid-beta 1-42 biomarker for prodromal Alzheimer's disease or mild cognitive impairment does not lead to more efficient clinical trials. Alzheimers Dement 2010; 6:367-77.
29. Maitournam A, Simon R. On the efficiency of targeted clinical trials. Stat Med 2005;24:329-39.
30. Schneider LS, Sano M. Current Alzheimer's disease clinical trials: methods and placebo outcomes. Alzheimers Dement 2009;5:388-97.
31. Gold M, Alderton C, Zvartau-Hind M, Egginton S, Saunders AM, Irizarry M, et al. Rosiglitazone monotherapy in mild-to-moderate Alzheimer's disease: results froma randomized, double-blind, placebo-controlled phase III study. Dement Geriatr Cogn Disord 2010;30:131-46.
32. Vemuri P, Wiste HJ, Weigand SD, Knopman DS, Shaw LM, Trojanowski JQ, et al. Effect of apolipoprotein E on biomarkers of amyloid load and neuronal pathology in Alzheimer disease. Ann Neurol 2010;67:308-16.
33. Fleisher AS, Chen K, Liu X, Ayutyanont N, Roontiva A, Thiyyagura P, et al. Apolipoprotein E ?4 and age effects on florbetapir positron emission tomography in healthy aging and Alzheimer disease. Neurobiol Aging 2013;34:1-12.
34. Drzezga A, Grimmer T, Henriksen G, Muhlau M, Perneczky R, Miederer I, et al. Effect of APOE genotype on amyloid plaque load and gray matter volume in Alzheimer disease. Neurology 2009;72:1487-94.
35. Caselli RJ, Dueck AC, Osborne D, Sabbagh MN, Connor DJ, Ahern GL, et al. Longitudinal modeling of age-related memory decline and the APOE epsilon4 effect. N Engl J Med 2009;361:255-63.
36. Kryscio RJ, Schmitt FA, Salazar JC, Mendiondo MS, Markesbery WR. Risk factors for transitions from normal to mild cognitive impairment and dementia. Neurology 2006;66:828-32.
37. Lopez OL, Jagust WJ, Dulberg C, Becker JT, DeKosky ST, Fitzpatrick A, et al. Risk factors for mild cognitive impairment in the Cardiovascular Health Study Cognition Study: part 2. Arch Neurol 2003;60:1394-9.
38. Luck T, Riedel-Heller SG, Luppa M, Wiese B, Wollny A, Wagner M, et al. Risk factors for incident mild cognitive impairment: results from the German Study on Ageing, Cognition and Dementia in Primary Care Patients (AgeCoDe). Acta Psychiatry Scand 2010;121:260-72.
39. Petersen RC,Waring SC, Smith GE, Tangalos EG, Thibodeau SN. Predictive value of APOE genotyping in incipient Alzheimer's disease. Ann N YAcad Sci 1996;802:58-69.
40. Petersen RC, Smith GE, Ivnik RJ, Tangalos EG, Schaid DJ, Thibodeau SN, et al. Apolipoprotein E status as a predictor of the development of Alzheimer's disease in memory-impaired individuals. JAMA 1995;273:1274-8.
41. Risacher SL, Shen L, West JD, Kim S, McDonald BC, Beckett LA, et al. Longitudinal MRI atrophy biomarkers: relationship to conversion in the ADNI cohort. Neurobiol Aging 2010;31:1401-8.
42. Schuff N, Woerner N, Boreta L, Kornfield T, Shaw LM, Trojanowski JQ, et al. MRI of hippocampal volume loss in early Alzheimer's disease in relation to ApoE genotype and biomarkers. Brain 2009;132:1067-77.
43. Devanand DP, Pelton GH, Zamora D, Liu X, Tabert MH, Goodkind M, et al. Predictive utility of apolipoprotein E genotype for Alzheimer disease in outpatients with mild cognitive impairment. Arch Neurol 2005; 62:975-80.
44. Mungas D, Beckett L, Harvey D, Farias ST, Reed B, Carmichael O, et al. Heterogeneity of cognitive trajectories in diverse older persons. Psychol Aging 2010;25:606-19.
45. Khachaturian AS, Corcoran CD, Mayer LS, Zandi PP, Breitner JCS, Cache County Study Investigators. Apolipoprotein E epsilon4 count affects age at onset of Alzheimer disease, but not lifetime susceptibility: the Cache County Study. Arch Gen Psychiatry 2004;61:518-24.
46. Schmand B, Huizenga HM, van Gool WA. Meta-analysis of CSF and MRI biomarkers for detecting preclinical Alzheimer's disease. Psychol Med 2010;40:135-45.
47. Visser PJ, Verhey F, Knol DL, Scheltens P,Wahlund L, Freund-Levi Y, et al. Prevalence and prognostic value of CSF markers of Alzheimer's disease pathology in patients with subjective cognitive impairment or mild cognitive impairment in the DESCRIPA study: a prospective cohort study. Lancet Neurol 2009;8:619-27.
48. Stone DJ, Molony C, Suver C, Schadt EE, Potter WZ. ApoE genotyping as a progression-rate biomarker in phase II disease-modification trials for Alzheimer's disease. Pharmacogenomics J 2010;10:161-4.