TY - JOUR

T1 - Multi-arm multi-stage trials can improve the efficiency of finding effective treatments for stroke

T2 - a case study

AU - Jaki, Thomas

AU - Wason, James M. S.

PY - 2018/11/27

Y1 - 2018/11/27

N2 - 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.

AB - 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.

KW - Adaptive design

KW - Clinical trial design

KW - Multi-arm multi-stage trials

KW - Multi-arm trials

U2 - 10.1186/s12872-018-0956-4

DO - 10.1186/s12872-018-0956-4

M3 - Journal article

VL - 18

JO - BMC Cardiovascular Disorders

JF - BMC Cardiovascular Disorders

SN - 1471-2261

IS - 1

M1 - 215

ER -