Functional measurements for clinical trials of stroke treatment_West China Medical Journal

Authors：

LIU Ziyue ,  ZHU Yicheng

Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, P. R. China;

Corresponding author：

ZHU Yicheng, Email: zhuych910@163.com

Keywords：

Stroke; Treatment; Clinical trial; Outcome assessment

DOI：

10.7507/1002-0179.202005061

Video：

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In recent years, clinical trial research on stroke intervention measures has been developing rapidly. In order to provide reliable conclusions, the outcome assessment of clinical trials is crucial. Tools for functional status evaluation have been widely adopted as outcome assessment, and have become mandatory for qualified clinical trials of stroke treatment. In this paper, the classification of functional assessment scales in stroke is reviewed, and the applications of functional assessment scales in clinical trials of stroke treatment are presented. National Institute of Health Stroke Scale, Barthel Index, and modified Rankin Scale are the top three frequently applied assessment scales in stroke trials. Also, their validity, reliability, responsiveness, and feasibility are described. Furthermore, analytical methods used to assess those functional assessments are highly heterogenous, while dichotomy of scale score is the most widely used. Although there is no consensus on designation of the functional assessment time, three-month is the most popular, reliable, and feasible choice in stroke trails.

Citation： LIU Ziyue, ZHU Yicheng. Functional measurements for clinical trials of stroke treatment. West China Medical Journal, 2020, 35(6): 637-641. doi: 10.7507/1002-0179.202005061 Copy

1.	Duncan PW, Jorgensen HS, Wade DT. Outcome measures in acute stroke trials: a systematic review and some recommendations to improve practice. Stroke, 2000, 31(6): 1429-1438.
2.	Rankin J. Cerebral vascular accidents in patients over the age of 60. II. Prognosis. Scott Med J, 1957, 2(5): 200-215.
3.	Quinn TJ, Langhorne P, Stott DJ. Barthel index for stroke trials: development, properties, and application. Stroke, 2011, 42(4): 1146-1151.
4.	Goldstein LB, Bertels C, Davis JN. Interrater reliability of the NIH stroke scale. Arch Neurol, 1989, 46(6): 660-662.
5.	World Health Organization. International classifcation of functioning, disability and health (ICF). Geneva: World Health Organization, 2001.
6.	Taylor-Rowan M, Wilson A, Dawson J, et al. Functional assessment for acute stroke trials: properties, analysis, and application. Front Neurol, 2018, 9: 191.
7.	Lees KR, Bath PM, Schellinger PD, et al. Contemporary outcome measures in acute stroke research: choice of primary outcome measure. Stroke, 2012, 43(4): 1163-1170.
8.	Quinn TJ, Dawson J, Walters MR, et al. Functional outcome measures in contemporary stroke trials. Int J Stroke, 2009, 4(3): 200-205.
9.	He S, Wu S, Zeng Q, et al. Assessment of methodological quality and outcome measures of acute stroke randomized controlled trials in China in recent 15 years. J Evid Based Med, 2012, 5(3): 174-182.
10.	Schrader J, Lüders S, Kulschewski A, et al. Morbidity and mortality after stroke, eprosartan compared with nitrendipine for secondary prevention: principal results of a prospective randomized controlled study (MOSES). Stroke, 2005, 36(6): 1218-1226.
11.	Mohr JP, Thompson JL, Lazar RM, et al. A comparison of warfarin and aspirin for the prevention of recurrent ischemic stroke. N Engl J Med, 2001, 345(20): 1444-1451.
12.	Feinstein AR. An additional basic science for clinical medicine: IV. The development of clinimetrics. Ann Intern Med, 1983, 99(6): 843-848.
13.	Wilson JT, Hareendran A, Hendry A, et al. Reliability of the modified Rankin Scale across multiple raters: benefits of a structured interview. Stroke, 2005, 36(4): 777-781.
14.	Wilson JT, Hareendran A, Grant M, et al. Improving the assessment of outcomes in stroke: use of a structured interview to assign grades on the modified Rankin Scale. Stroke, 2002, 33(9): 2243-2246.
15.	Banks JL, Marotta CA. Outcomes validity and reliability of the modified Rankin scale: implications for stroke clinical trials: a literature review and synthesis. Stroke, 2007, 38(3): 1091-1096.
16.	Dromerick AW, Edwards DF, Diringer MN. Sensitivity to changes in disability after stroke: a comparison of four scales useful in clinical trials. J Rehabil Res Dev, 2003, 40(1): 1-8.
17.	Leung SO, Chan CC, Shah S. Development of a Chinese version of the Modified Barthel Index--validity and reliability. Clin Rehabil, 2007, 21(10): 912-922.
18.	Green J, Forster A, Young J. A test-retest reliability study of the Barthel Index, the Rivermead Mobility Index, the Nottingham Extended Activities of Daily Living Scale and the Frenchay Activities Index in stroke patients. Disabil Rehabil, 2001, 23(15): 670-676.
19.	Ellul J, Watkins C, Barer D. Estimating total Barthel scores from just three items: the European Stroke Database “minimum dataset” for assessing functional status at discharge from hospital. Age Ageing, 1998, 27(2): 115-122.
20.	Brott T, Adams HP, Olinger CP, et al. Measurements of acute cerebral infarction: a clinical examination scale. Stroke, 1989, 20(7): 864-870.
21.	Saver JL, Johnston KC, Homer D, et al. Infarct volume as a surrogate or auxiliary outcome measure in ischemic stroke clinical trials. The RANTTAS Investigators. Stroke, 1999, 30(2): 293-298.
22.	Adams HP, Davis PH, Leira EC, et al. Baseline NIH Stroke Scale score strongly predicts outcome after stroke: a report of the Trial of Org 10172 in Acute Stroke Treatment (TOAST). Neurology, 1999, 53(1): 126-131.
23.	Goldstein LB, Samsa GP. Reliability of the National Institutes of Health Stroke Scale. Extension to non-neurologists in the context of a clinical trial. Stroke, 1997, 28(2): 307-310.
24.	Balu S. Differences in psychometric properties, cut-off scores, and outcomes between the Barthel Index and Modified Rankin Scale in pharmacotherapy-based stroke trials: systematic literature review. Curr Med Res Opin, 2009, 25(6): 1329-1341.
25.	Feng W, Vasquez G, Suri MF, et al. Repeated-measures analysis of the National Institute of Neurological Disorders and Stroke rt-PA stroke trial. J Stroke Cerebrovasc Dis, 2011, 20(3): 241-246.
26.	Makin SD, Doubal FN, Quinn TJ, et al. The effect of different combinations of vascular, dependency and cognitive endpoints on the sample size required to detect a treatment effect in trials of treatments to improve outcome after lacunar and non-lacunar ischaemic stroke. Eur Stroke J, 2018, 3(1): 66-73.
27.	Uyttenboogaart M, Stewart RE, Vroomen PC, et al. Optimizing cutoff scores for the Barthel index and the modified Rankin scale for defining outcome in acute stroke trials. Stroke, 2005, 36(9): 1984-1987.
28.	Bath PM, Gray LJ, Collier T, et al. Can we improve the statistical analysis of stroke trials? Statistical reanalysis of functional outcomes in stroke trials. Stroke, 2007, 38(6): 1911-1915.
29.	Mandava P, Krumpelman CS, Shah JN, et al. Quantification of errors in ordinal outcome scales using Shannon entropy: effect on sample size calculations. PLoS One, 2013, 8(7): e67754.
30.	Langhorne P, Bernhardt J, Kwakkel G. Stroke care 2: Stroke rehabilitation. Lancet, 2011, 377(9778): 1693-1702.
31.	Huybrechts KF, Caro JJ, Xenakis JJ, et al. The prognostic value of the modified Rankin Scale score for long-term survival after first-ever stroke. Results from the Athens Stroke Registry. Cerebrovasc Dis, 2008, 26(4): 381-387.
32.	Jcrgensen HS, Nakayama H, Raaschou HO. Outcome and time course of recovery in stroke. Part II: time course of recovery. The Copenhagen Stroke Study. Arch Phys Med Rehabil, 1995, 76(5): 406-412.

1. Duncan PW, Jorgensen HS, Wade DT. Outcome measures in acute stroke trials: a systematic review and some recommendations to improve practice. Stroke, 2000, 31(6): 1429-1438.
2. Rankin J. Cerebral vascular accidents in patients over the age of 60. II. Prognosis. Scott Med J, 1957, 2(5): 200-215.
3. Quinn TJ, Langhorne P, Stott DJ. Barthel index for stroke trials: development, properties, and application. Stroke, 2011, 42(4): 1146-1151.
4. Goldstein LB, Bertels C, Davis JN. Interrater reliability of the NIH stroke scale. Arch Neurol, 1989, 46(6): 660-662.
5. World Health Organization. International classifcation of functioning, disability and health (ICF). Geneva: World Health Organization, 2001.
6. Taylor-Rowan M, Wilson A, Dawson J, et al. Functional assessment for acute stroke trials: properties, analysis, and application. Front Neurol, 2018, 9: 191.
7. Lees KR, Bath PM, Schellinger PD, et al. Contemporary outcome measures in acute stroke research: choice of primary outcome measure. Stroke, 2012, 43(4): 1163-1170.
8. Quinn TJ, Dawson J, Walters MR, et al. Functional outcome measures in contemporary stroke trials. Int J Stroke, 2009, 4(3): 200-205.
9. He S, Wu S, Zeng Q, et al. Assessment of methodological quality and outcome measures of acute stroke randomized controlled trials in China in recent 15 years. J Evid Based Med, 2012, 5(3): 174-182.
10. Schrader J, Lüders S, Kulschewski A, et al. Morbidity and mortality after stroke, eprosartan compared with nitrendipine for secondary prevention: principal results of a prospective randomized controlled study (MOSES). Stroke, 2005, 36(6): 1218-1226.
11. Mohr JP, Thompson JL, Lazar RM, et al. A comparison of warfarin and aspirin for the prevention of recurrent ischemic stroke. N Engl J Med, 2001, 345(20): 1444-1451.
12. Feinstein AR. An additional basic science for clinical medicine: IV. The development of clinimetrics. Ann Intern Med, 1983, 99(6): 843-848.
13. Wilson JT, Hareendran A, Hendry A, et al. Reliability of the modified Rankin Scale across multiple raters: benefits of a structured interview. Stroke, 2005, 36(4): 777-781.
14. Wilson JT, Hareendran A, Grant M, et al. Improving the assessment of outcomes in stroke: use of a structured interview to assign grades on the modified Rankin Scale. Stroke, 2002, 33(9): 2243-2246.
15. Banks JL, Marotta CA. Outcomes validity and reliability of the modified Rankin scale: implications for stroke clinical trials: a literature review and synthesis. Stroke, 2007, 38(3): 1091-1096.
16. Dromerick AW, Edwards DF, Diringer MN. Sensitivity to changes in disability after stroke: a comparison of four scales useful in clinical trials. J Rehabil Res Dev, 2003, 40(1): 1-8.
17. Leung SO, Chan CC, Shah S. Development of a Chinese version of the Modified Barthel Index--validity and reliability. Clin Rehabil, 2007, 21(10): 912-922.
18. Green J, Forster A, Young J. A test-retest reliability study of the Barthel Index, the Rivermead Mobility Index, the Nottingham Extended Activities of Daily Living Scale and the Frenchay Activities Index in stroke patients. Disabil Rehabil, 2001, 23(15): 670-676.
19. Ellul J, Watkins C, Barer D. Estimating total Barthel scores from just three items: the European Stroke Database “minimum dataset” for assessing functional status at discharge from hospital. Age Ageing, 1998, 27(2): 115-122.
20. Brott T, Adams HP, Olinger CP, et al. Measurements of acute cerebral infarction: a clinical examination scale. Stroke, 1989, 20(7): 864-870.
21. Saver JL, Johnston KC, Homer D, et al. Infarct volume as a surrogate or auxiliary outcome measure in ischemic stroke clinical trials. The RANTTAS Investigators. Stroke, 1999, 30(2): 293-298.
22. Adams HP, Davis PH, Leira EC, et al. Baseline NIH Stroke Scale score strongly predicts outcome after stroke: a report of the Trial of Org 10172 in Acute Stroke Treatment (TOAST). Neurology, 1999, 53(1): 126-131.
23. Goldstein LB, Samsa GP. Reliability of the National Institutes of Health Stroke Scale. Extension to non-neurologists in the context of a clinical trial. Stroke, 1997, 28(2): 307-310.
24. Balu S. Differences in psychometric properties, cut-off scores, and outcomes between the Barthel Index and Modified Rankin Scale in pharmacotherapy-based stroke trials: systematic literature review. Curr Med Res Opin, 2009, 25(6): 1329-1341.
25. Feng W, Vasquez G, Suri MF, et al. Repeated-measures analysis of the National Institute of Neurological Disorders and Stroke rt-PA stroke trial. J Stroke Cerebrovasc Dis, 2011, 20(3): 241-246.
26. Makin SD, Doubal FN, Quinn TJ, et al. The effect of different combinations of vascular, dependency and cognitive endpoints on the sample size required to detect a treatment effect in trials of treatments to improve outcome after lacunar and non-lacunar ischaemic stroke. Eur Stroke J, 2018, 3(1): 66-73.
27. Uyttenboogaart M, Stewart RE, Vroomen PC, et al. Optimizing cutoff scores for the Barthel index and the modified Rankin scale for defining outcome in acute stroke trials. Stroke, 2005, 36(9): 1984-1987.
28. Bath PM, Gray LJ, Collier T, et al. Can we improve the statistical analysis of stroke trials? Statistical reanalysis of functional outcomes in stroke trials. Stroke, 2007, 38(6): 1911-1915.
29. Mandava P, Krumpelman CS, Shah JN, et al. Quantification of errors in ordinal outcome scales using Shannon entropy: effect on sample size calculations. PLoS One, 2013, 8(7): e67754.
30. Langhorne P, Bernhardt J, Kwakkel G. Stroke care 2: Stroke rehabilitation. Lancet, 2011, 377(9778): 1693-1702.
31. Huybrechts KF, Caro JJ, Xenakis JJ, et al. The prognostic value of the modified Rankin Scale score for long-term survival after first-ever stroke. Results from the Athens Stroke Registry. Cerebrovasc Dis, 2008, 26(4): 381-387.
32. Jcrgensen HS, Nakayama H, Raaschou HO. Outcome and time course of recovery in stroke. Part II: time course of recovery. The Copenhagen Stroke Study. Arch Phys Med Rehabil, 1995, 76(5): 406-412.

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Functional measurements for clinical trials of stroke treatment

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