Modified Rankin Scale at 90 Days Versus National Institutes of Health Stroke Scale at 24 Hours as Primary Outcome in Acute Stroke Trials

Background We investigate whether the National Institutes of Health Stroke Scale (NIHSS) at 24 hours could serve as a primary outcome in acute ischemic stroke trials, and whether combining 90‐day modified Rankin Scale (mRS) and 24‐hour NIHSS in a hierarchical outcome could enhance detection of treat...

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Main Authors: Johanna M. Ospel, Scott Brown, Salome Bosshart, Alexander Stebner, Kazutaka Uchida, Andrew Demchuk, Jeffrey L. Saver, Philip White, Keith W. Muir, Diederik W. J. Dippel, Charles B. L. M. Majoie, Tudor G. Jovin, Bruce C. V. Campbell, Peter J. Mitchell, Serge Bracard, Francis Guillemin, Michael Hill, Mayank Goyal
Format: Article
Language:English
Published: Wiley 2025-03-01
Series:Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease
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Online Access:https://www.ahajournals.org/doi/10.1161/JAHA.124.037752
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author Johanna M. Ospel
Scott Brown
Salome Bosshart
Alexander Stebner
Kazutaka Uchida
Andrew Demchuk
Jeffrey L. Saver
Philip White
Keith W. Muir
Diederik W. J. Dippel
Charles B. L. M. Majoie
Tudor G. Jovin
Bruce C. V. Campbell
Peter J. Mitchell
Serge Bracard
Francis Guillemin
Michael Hill
Mayank Goyal
author_facet Johanna M. Ospel
Scott Brown
Salome Bosshart
Alexander Stebner
Kazutaka Uchida
Andrew Demchuk
Jeffrey L. Saver
Philip White
Keith W. Muir
Diederik W. J. Dippel
Charles B. L. M. Majoie
Tudor G. Jovin
Bruce C. V. Campbell
Peter J. Mitchell
Serge Bracard
Francis Guillemin
Michael Hill
Mayank Goyal
author_sort Johanna M. Ospel
collection DOAJ
description Background We investigate whether the National Institutes of Health Stroke Scale (NIHSS) at 24 hours could serve as a primary outcome in acute ischemic stroke trials, and whether combining 90‐day modified Rankin Scale (mRS) and 24‐hour NIHSS in a hierarchical outcome could enhance detection of treatment effect, using endovascular treatment (EVT) as an exemplary study intervention. Methods This was a post hoc analysis of pooled data from 7 randomized EVT trials. Twenty‐four‐hour NIHSS as a surrogate outcome for 90‐day mRS was assessed in a causal mediation model. A 7‐point ordinal NIHSS score was generated by grouping 24‐hour NIHSS, including death as a separate category (“ordinal” NIHSS). EVT effect sizes and sample sizes required for detecting EVT benefit with 80% power were compared when using granular 24‐hour NIHSS, ordinal 24‐hour NIHSS, 90‐day mRS, and a hierarchical outcome (win ratio) that combines 90‐day mRS and 24‐hour NIHSS. Results A total of 1720 patients were included. Twenty‐four‐hour NIHSS mediated the association between EVT and 90‐day mRS and met criteria for a useful surrogate outcome. Effect sizes were highest and sample sizes required to detect EVT benefit smallest for the win ratio approach (228), followed by 90‐day mRS (240) and ordinal 24‐hour NIHSS (242). In patients with baseline NIHSS <10 and ≥25, ordinal 24‐hour NIHSS resulted in the highest effect size. Conclusions Twenty‐four‐hour NIHSS is a useful surrogate outcome for 90‐day mRS in patients with acute ischemic stroke undergoing EVT, with a similar EVT effect size compared with 90‐day mRS. It could potentially enhance detection of EVT benefit in patients with very low or high baseline NIHSS. An ordered hierarchical outcome could improve detection of EVT treatment effect.
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spelling doaj-art-0f4a26f3a8bc4c1a8465741da8b6b9462025-08-20T02:24:58ZengWileyJournal of the American Heart Association: Cardiovascular and Cerebrovascular Disease2047-99802025-03-0114510.1161/JAHA.124.037752Modified Rankin Scale at 90 Days Versus National Institutes of Health Stroke Scale at 24 Hours as Primary Outcome in Acute Stroke TrialsJohanna M. Ospel0Scott Brown1Salome Bosshart2Alexander Stebner3Kazutaka Uchida4Andrew Demchuk5Jeffrey L. Saver6Philip White7Keith W. Muir8Diederik W. J. Dippel9Charles B. L. M. Majoie10Tudor G. Jovin11Bruce C. V. Campbell12Peter J. Mitchell13Serge Bracard14Francis Guillemin15Michael Hill16Mayank Goyal17Department of Diagnostic Imaging, Foothills Medical Center University of Calgary AB CanadaBRIGHT Research Minneapolis MN USADepartment of Diagnostic Imaging, Foothills Medical Center University of Calgary AB CanadaDepartment of Diagnostic Imaging, Foothills Medical Center University of Calgary AB CanadaDepartment of Diagnostic Imaging, Foothills Medical Center University of Calgary AB CanadaDepartment of Diagnostic Imaging, Foothills Medical Center University of Calgary AB CanadaDavid Geffen School of Medicine University of Los Angeles CA USATranslational and Clinical Research Institute Newcastle University Newcastle upon Tyne UKInstitute of Neuroscience &amp; Psychology University of Glasgow, Queen Elizabeth University Hospital Glasgow UKErasmus MC University Medical Center Rotterdam NetherlandsAmsterdam University Medical Centers Location University of Amsterdam NetherlandsDepartment of Neurology Cooper Health New Jersey NJ USADepartment of Medicine and Neurology, Melbourne Brain Centre, Royal Melbourne Hospital University of Melbourne VIC AustraliaDepartment of Radiology, Royal Melbourne Hospital University of Melbourne VIC AustraliaDepartment of Diagnostic and Therapeutic Neuroradiology, CHRU‐Nancy University of Lorraine Nancy FranceINSERM CIC 1433 Clinical Epidemiology Universite de Lorraine Nancy FranceDepartment of Diagnostic Imaging, Foothills Medical Center University of Calgary AB CanadaDepartment of Diagnostic Imaging, Foothills Medical Center University of Calgary AB CanadaBackground We investigate whether the National Institutes of Health Stroke Scale (NIHSS) at 24 hours could serve as a primary outcome in acute ischemic stroke trials, and whether combining 90‐day modified Rankin Scale (mRS) and 24‐hour NIHSS in a hierarchical outcome could enhance detection of treatment effect, using endovascular treatment (EVT) as an exemplary study intervention. Methods This was a post hoc analysis of pooled data from 7 randomized EVT trials. Twenty‐four‐hour NIHSS as a surrogate outcome for 90‐day mRS was assessed in a causal mediation model. A 7‐point ordinal NIHSS score was generated by grouping 24‐hour NIHSS, including death as a separate category (“ordinal” NIHSS). EVT effect sizes and sample sizes required for detecting EVT benefit with 80% power were compared when using granular 24‐hour NIHSS, ordinal 24‐hour NIHSS, 90‐day mRS, and a hierarchical outcome (win ratio) that combines 90‐day mRS and 24‐hour NIHSS. Results A total of 1720 patients were included. Twenty‐four‐hour NIHSS mediated the association between EVT and 90‐day mRS and met criteria for a useful surrogate outcome. Effect sizes were highest and sample sizes required to detect EVT benefit smallest for the win ratio approach (228), followed by 90‐day mRS (240) and ordinal 24‐hour NIHSS (242). In patients with baseline NIHSS <10 and ≥25, ordinal 24‐hour NIHSS resulted in the highest effect size. Conclusions Twenty‐four‐hour NIHSS is a useful surrogate outcome for 90‐day mRS in patients with acute ischemic stroke undergoing EVT, with a similar EVT effect size compared with 90‐day mRS. It could potentially enhance detection of EVT benefit in patients with very low or high baseline NIHSS. An ordered hierarchical outcome could improve detection of EVT treatment effect.https://www.ahajournals.org/doi/10.1161/JAHA.124.037752acute ischemic strokeendovascular treatmentmRSNIHSSthrombectomy
spellingShingle Johanna M. Ospel
Scott Brown
Salome Bosshart
Alexander Stebner
Kazutaka Uchida
Andrew Demchuk
Jeffrey L. Saver
Philip White
Keith W. Muir
Diederik W. J. Dippel
Charles B. L. M. Majoie
Tudor G. Jovin
Bruce C. V. Campbell
Peter J. Mitchell
Serge Bracard
Francis Guillemin
Michael Hill
Mayank Goyal
Modified Rankin Scale at 90 Days Versus National Institutes of Health Stroke Scale at 24 Hours as Primary Outcome in Acute Stroke Trials
Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease
acute ischemic stroke
endovascular treatment
mRS
NIHSS
thrombectomy
title Modified Rankin Scale at 90 Days Versus National Institutes of Health Stroke Scale at 24 Hours as Primary Outcome in Acute Stroke Trials
title_full Modified Rankin Scale at 90 Days Versus National Institutes of Health Stroke Scale at 24 Hours as Primary Outcome in Acute Stroke Trials
title_fullStr Modified Rankin Scale at 90 Days Versus National Institutes of Health Stroke Scale at 24 Hours as Primary Outcome in Acute Stroke Trials
title_full_unstemmed Modified Rankin Scale at 90 Days Versus National Institutes of Health Stroke Scale at 24 Hours as Primary Outcome in Acute Stroke Trials
title_short Modified Rankin Scale at 90 Days Versus National Institutes of Health Stroke Scale at 24 Hours as Primary Outcome in Acute Stroke Trials
title_sort modified rankin scale at 90 days versus national institutes of health stroke scale at 24 hours as primary outcome in acute stroke trials
topic acute ischemic stroke
endovascular treatment
mRS
NIHSS
thrombectomy
url https://www.ahajournals.org/doi/10.1161/JAHA.124.037752
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