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

Published version
Peer-reviewed

Type

Article

Change log

Authors

Wason, James MS 

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.

Description

Keywords

Adaptive design, Clinical trial design, Multi-arm multi-stage trials, Multi-arm trials

Journal Title

BMC Cardiovasc Disord

Conference Name

Journal ISSN

1471-2261
1471-2261

Volume Title

18

Publisher

Springer Nature
Sponsorship
NIHR Academy (SRF-2015-08-001)
This work was supported in part by grants the National Institute for Health Research (NIHR-SRF-2015-08-001, TJ), the Medical Research Council (SLAH/210 JW).