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Multi-arm multi-stage trials can improve the efficiency of finding effective treatments for stroke: a case study

Lookup NU author(s): Professor James WasonORCiD



This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


BACKGROUND: Many recent Stroke trials fail to show a beneficial effect of the intervention late in the development. Currently a large number of new treatment options are being developed. Multi-arm multi-stage (MAMS) designs offer one potential strategy to avoid lengthy studies of treatments without beneficial effects while at the same time allowing evaluation of several novel treatments. In this paper we provide a review of what MAMS designs are and argue that they are of particular value for Stroke trials. We illustrate this benefit through a case study based on previous published trials of endovascular treatment for acute ischemic stroke. We show in this case study that MAMS trials provide additional power for the same sample size compared to alternative trial designs. This level of additional power depends on the recruitment length of the trial, with most efficiency gained when recruitment is relatively slow. We conclude with a discussion of additional considerations required when starting a MAMS trial. CONCLUSION: MAMS trial designs are potentially very useful for stroke trials due to their improved statistical power compared to the traditional approach.

Publication metadata

Author(s): Jaki T, Wason JMS

Publication type: Article

Publication status: Published

Journal: BMC Cardiovascular Disorders

Year: 2018

Volume: 18

Issue: 1

Online publication date: 27/11/2018

Acceptance date: 15/11/2018

Date deposited: 13/12/2018

ISSN (electronic): 1471-2261

Publisher: BioMed Central Ltd.


DOI: 10.1186/s12872-018-0956-4

PubMed id: 30482176


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