Progression of MRI markers in cerebral small vessel disease: Sample size considerations for clinical trials

Journal of Cerebral Blood Flow and Metabolism

Volumn 36, Issue 1, 2016, Pages 228-240

  Benjamin, Philip ^a   Zeestraten, Eva ^a   Lambert, Christian ^a   Chis Ster, Irina ^a   Williams, Owen A ^a   Lawrence, Andrew J ^b   Patel, Bhavini ^a   Mackinnon, Andrew D ^c   Barrick, Thomas R ^a   Markus, Hugh S ^b  

^a ST GEORGE'S UNIVERSITY OF LONDON   (United Kingdom)

^b UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^c ST GEORGE'S UNIVERSITY HOSPITALS NHS FOUNDATION TRUST   (United Kingdom)

Author keywords

Cerebral small vessel disease; clinical trials; diffusion tensor imaging; magnetic resonance imaging; vascular cognitive impairment

Indexed keywords

AGED; ARTICLE; BRAIN SIZE; CEREBROVASCULAR DISEASE; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DIFFUSION TENSOR IMAGING; DISEASE MARKER; EXECUTIVE FUNCTION; FEMALE; FOLLOW UP; HUMAN; LACUNES; MAJOR CLINICAL STUDY; MALE; MINI MENTAL STATE EXAMINATION; NERVOUS SYSTEM PARAMETERS; NEUROPSYCHOLOGICAL TEST; NUCLEAR MAGNETIC RESONANCE IMAGING; NUCLEAR MAGNETIC RESONANCE SCANNER; PRIORITY JOURNAL; PROSPECTIVE STUDY; SAMPLE SIZE; SENSITIVITY ANALYSIS; WHITE MATTER HYPERINTENSITY VOLUME; CLINICAL TRIAL (TOPIC); COGNITIVE DEFECT; DIFFUSION WEIGHTED IMAGING; LONGITUDINAL STUDY; PATHOLOGY; PROCEDURES; PSYCHOLOGY; WHITE MATTER;

AGED; CEREBRAL SMALL VESSEL DISEASES; CLINICAL TRIALS AS TOPIC; COGNITION DISORDERS; DIFFUSION MAGNETIC RESONANCE IMAGING; DIFFUSION TENSOR IMAGING; FEMALE; HUMANS; LONGITUDINAL STUDIES; MALE; NEUROPSYCHOLOGICAL TESTS; SAMPLE SIZE; WHITE MATTER;

EID: 84951569902     PISSN: 0271678X     EISSN: 15597016     Source Type: Journal    
DOI: 10.1038/jcbfm.2015.113     Document Type: Article

References (42)

1. Pantoni L. Cerebral small vessel disease: from pathogenesis and clinical characteristics to therapeutic challenges. Lancet Neurol 2010; 9: 689-701
2. Patel B and Markus HS. Magnetic resonance imaging in cerebral small vessel disease and its use as a surrogate disease marker. Int J Stroke 2011; 6: 47-59
3. Van Norden AGW, van Dijk EJ, de Laat KF, Scheltens P, OldeRikkert MGM and de Leeuw FE. Dementia: Alzheimer pathology and vascular factors: from mutually exclusive to interaction. Biochim Biophys Acta 2012; 1822: 340-349
4. Schmidt R, Berghold A, Jokinen H, Gouw AA, van Flier WM, der, Barkhof F, et al. White matter lesion progression in LADIS frequency, clinical effects, and sample size calculations. Stroke 2012; 43: 2643-2647
5. Holtmannspötter M, Peters N, Opherk C, Martin D, Herzog J, Brückmann H, et al. Diffusion magnetic resonance histograms as a surrogate marker and predictor of disease progression in CADASIL: a two-year follow-up study. Stroke J Cereb Circ 2005; 36: 2559-2565
6. Lawrence AJ, Chung AW, Morris RG, Markus HS and Barrick TR. Structural network efficiency is associated with cognitive impairment in small-vessel disease. Neurology 2014; 83: 304-311
7. Fleming TR and DeMets DL. Surrogate end points in clinical trials: are we being misled? Ann Intern Med 1996; 125: 605-613
8. Lawrence AJ, Patel B, Morris RG, MacKinnon AD, Rich PM, Barrick TR, et al. Mechanisms of cognitive impairment in cerebral small vessel disease: multimodal MRI results from the St George's Cognition and Neuroimaging in Stroke (SCANS) Study. PLoS One 2013; 8: e61014
9. Arani Nitkunan SL. Brain atrophy and cerebral small vessel disease: a prospective follow-up study. Stroke J Cereb Circ 2011; 42: 133-138
10. Jokinen H, Lipsanen J, Schmidt R, Fazekas F, Gouw AA, van der Flier WM, et al. Brain atrophy accelerates cognitive decline in cerebral small vessel disease: the LADIS study. Neurology 2012; 78: 1785-1792
11. Peters N, Holtmannspötter M, Opherk C, Gschwendtner A, Herzog J, Sämann P, et al. Brain volume changes in CADASIL: a serial MRI study in pure subcortical ischemic vascular disease. Neurology 2006; 66: 1517-1522
12. Nitkunan A, Barrick TR, Charlton RA, Clark CA and Markus HS. Multimodal MRI in cerebral small vessel disease its relationship with cognition and sensitivity to change over time. Stroke 2008; 39: 1999-2005
13. Van Norden AGW, de Laat KF, van Dijk EJ, van Uden IWM, van Oudheusden LJB, Gons RAR, et al. Diffusion tensor imaging and cognition in cerebral small vessel disease: the RUN DMC study. Biochim Biophys Acta 2012; 1822: 401-407
14. Benjamin P, Lawrence AJ, Lambert C, Patel B, Chung AW, MacKinnon AD, et al. Strategic lacunes and their relationship to cognitive impairment in cerebral small vessel disease. Neuroimage Clin 2014; 4: 828-837
15. Jokinen H, Gouw AA, Madureira S, Ylikoski R, van Straaten ECW, van der Flier WM, et al. Incident lacunes influence cognitive decline: the LADIS study. Neurology 2011; 76: 1872-1878
16. Wardlaw JM, Smith EE, Biessels GJ, Cordonnier C, Fazekas F, Frayne R, et al. Neuroimaging standards for research into small vessel disease and its contribution to ageing and neurodegeneration. Lancet Neurol 2013; 12: 822-838
17. Fazekas F, Kleinert R, Offenbacher H, Schmidt R, Kleinert G, Payer F, et al. Pathologic correlates of incidental MRI white matter signal hyperintensities. Neurology 1993; 43: 1683-1689
18. Smith SM, Jenkinson M, Woolrich MW, Beckmann CF, Behrens TEJ, Johansen-Berg H, et al. Advances in functional and structural MR image analysis and implementation as FSL. NeuroImage 2004; 23: S208-S219
19. Altmann DR, Jasperse B, Barkhof F, Beckmann K, Filippi M, Kappos LD, et al. Sample sizes for brain atrophy outcomes in trials for secondary progressive multiple sclerosis. Neurology 2009; 72: 595-601
20. Anderson VM, Schott JM, Bartlett JW, Leung KK, Miller DH and Fox NC. Gray matter atrophy rate as a marker of disease progression in AD. Neurobiol Aging 2012; 33: 1194-1202
21. Ashburner J and Friston KJ. Diffeomorphic registration using geodesic shooting and Gauss-Newton optimisation. Neuroimage 2011; 55: 954-967
22. Lambert C, Lutti A, Helms G, Frackowiak R and Ashburner J. Multiparametric brainstem segmentation using a modified multivariate mixture of Gaussians. Neuroimage Clin 2013; 2: 684-694
23. Nagahama Y, Okina T, Suzuki N, Matsuzaki S, Yamauchi H, Nabatame H, et al. Factor structure of a modified version of the Wisconsin Card Sorting Test: an analysis of executive deficit in Alzheimer's disease and mild cognitive impairment. Dement Geriatr Cogn Disord 2003; 16: 103-112
24. Delis DC, Kaplan E and Kramer JH. Delis-Kaplan executive function system. San Antonio, TX: Psychological Corporation, 2001
25. Wechsler D. Wais-III, Wechsler Adult Intelligence Scale, Third Edition: WMS-III, Wechsler Memory Scale, 3rd ed. Technical Manual. Psychological Corporation, San Antonio, TX, 1997
26. Coughlan A, Oddy M and Crawford J. The BIRT memory and information processing battery (B-MIPB). Wakefield, UK: The Brain Injury Rehabilitation Trust (BIRT), 2009
27. Matthews C and Klove H. Instruction manual for the adult neuropsychological test battery. Madison, WI: University of Wisconsin Medical School, 1964
28. Diggle PJ, Heagerty P, Liang K-Y and Zeger SL. Analysis of longitudinal data. Oxford University Press, 2002
29. Liu G and Liang K-Y. Sample size calculations for studies with correlated observations. Biometrics 1997; 53: 937-947
30. Lu K, Luo X and Chen P-Y. Sample size estimation for repeated measures analysis in randomized clinical trials with missing data. Int J Biostat 2008; 4, article 9: 1-16
31. Pearce LA, McClure LA, Anderson DC, Jacova C, Sharma M, Hart RG, et al. Effects of long-term blood pressure lowering and dual antiplatelet treatment on cognitive function in patients with recent lacunar stroke: a secondary analysis from the SPS3 randomised trial. Lancet Neurol 2014; 13: 1177-1185
32. Liem MK, Lesnik Oberstein SJ, Haan J, van der Neut IL, Ferrari MD, van Buchem MA et al. MRI correlates of cognitive decline in CADASIL: a 7-year follow-up study. Neurology 2009; 72: 143-148
33. Charlton RA, Barrick TR, McIntyre DJ, Shen Y, O'Sullivan M, Howe FA, et al. White matter damage on diffusion tensor imaging correlates with age-related cognitive decline. Neurology 2006; 66: 217-222
34. Fisher CM. The arterial lesions underlying lacunes. Acta Neuropathol (Berl) 1968; 12: 1-15
35. Boiten J, Lodder J and Kessels F. Two clinically distinct lacunar infarct entities? A hypothesis. Stroke 1993; 24: 652-656
36. Khan U, Porteous L, Hassan A and Markus HS. Risk factor profile of cerebral small vessel disease and its subtypes. J Neurol Neurosurg Psychiatry 2006; 78: 702-706
37. Patel B, Lawrence AJ, Chung AW, Rich P, MacKinnon AD, Morris RG, et al. Cerebral microbleeds and cognition in patients with symptomatic small vessel disease. Stroke 2013; 44: 356-361
38. Glymour MM, Chene G, Tzourio C and Dufouil C. Brain MRI markers and dropout in a longitudinal study of cognitive aging. Neurology 2012; 79: 1340-1348
39. Vrenken H, Jenkinson M, Horsfield MA, Battaglini M, van Schijndel RA, Rostrup E, et al. Recommendations to improve imaging and analysis of brain lesion load and atrophy in longitudinal studies of multiple sclerosis. J Neurol 2013; 260: 2458-2471
40. Garde E, Lykke Mortensen E, Rostrup E and Paulson OB. Decline in intelligence is associated with progression in white matter hyperintensity volume. J Neurol Neurosurg Psychiatry 2005; 76: 1289-1291
41. Van den Heuvel DMJ, ten Dam VH, de Craen AJM, Admiraal-Behloul F, Olofsen H, Bollen ELEM, et al. Increase in periventricular white matter hyperintensities parallels decline in mental processing speed in a nondemented elderly population. J Neurol Neurosurg Psychiatry 2006; 77: 149-153
42. Schmidt R, Ropele S, Enzinger C, Petrovic K, Smith S, Schmidt H, et al. White matter lesion progression, brain atrophy, and cognitive decline: the Austrian stroke prevention study. Ann Neurol 2005; 58: 610-616.