The Utility of Domain-Specific End Points in Acute Stroke Trials
Year: 2021
Type: editorial
Abstract: Domain-specific endpoints are assessments that correspond to the output of individual neural systems and are useful for capturing treatment effects on specific behaviors. By contrast, global endpoints combine several attributes into a single score and are useful for capturing broad treatment effects in a summary way. While global endpoints have become the de facto mechanism required to define benefit in stroke trials, they also have important limitations, some of which might be addressed by simultaneously measuring domain-specific endpoints. Substantial opportunity remains to identify quantifiable patient benefit that would otherwise not be captured by global endpoints. Potential advantages of incorporating domain-specific endpoints in acute stroke trials are discussed, such as increased granularity of measurement, improved understanding of how therapies affect the brain between acute treatment and day 90, and optimized therapeutic translation. Potential disadvantages are also considered, including time and cost of administering domain-specific endpoints, as well as statistical implications. Domain-specific endpoints and global endpoints are not mutually exclusive, and both capture clinical benefits to patients. Incorporating a broader set of outcome assessments in stroke trials, including both global and domain-specific endpoints, is warranted.
Source: Stroke
Authors: Steven C. Cramer, Steven L. Wolf, Jeffrey L. Saver, Karen C. Johnston, J Mocco, Maarten G. Lansberg, Sean I. Savitz, David S. Liebeskind, Wade Smith, Max Wintermark, Jordan J. Elm, Pooja Khatri, Joseph P. Broderick, Scott Janis, Daofen Chen, Alexander Dromerick, Kari Dunning, Andrew W. Grande, Randolph S. Marshall, Caitlyn Meinzer, Stephen Page, Aimee Reiss, Lorie Richards, Lawrence Wechsler, Carolee Winstein, Cheryl Bushnell, Dorothy Edwards, Warren Lo, Jin-Moo Lee, Catherine M. Amlie-Lefond, Gregory W. Albers, Robert Dempsey, Toby I. Gropen, Edward C. Jauch, Enrique C. Leira, Renee Martin, Brett C. Meyer, Kiva Schindler, Phillip Scott, Aneesh B. Singhal, Claudia S. Moy
Institutions: University of California, Los Angeles, Emory University, University of Virginia, Mount Sinai Hospital, Icahn School of Medicine at Mount Sinai, Stanford Medicine, Stanford University, The University of Texas Health Science Center at Houston, University of California, San Francisco, Medical University of South Carolina, University of Cincinnati, University of Cincinnati Medical Center, National Institutes of Health, National Institute of Neurological Disorders and Stroke
Language: English
Subfield: Epidemiology
Field: Medicine
Domain: Health Sciences
Open Access status: green
OpenAlex ID: W3126534192