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

Jaki, Thomas and Wason, James M. S. (2018) Multi-arm multi-stage trials can improve the efficiency of finding effective treatments for stroke:a case study. BMC Cardiovascular Disorders, 18 (1). ISSN 1471-2261

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Abstract

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.

Item Type:
Journal Article
Journal or Publication Title:
BMC Cardiovascular Disorders
Uncontrolled Keywords:
/dk/atira/pure/subjectarea/asjc/2700/2705
Subjects:
ID Code:
129585
Deposited By:
Deposited On:
11 Jan 2019 11:30
Refereed?:
Yes
Published?:
Published
Last Modified:
23 Sep 2020 04:52