缺血性腦中風取栓器械療效之比較－非劣性試驗之臨界值的影響

Novel endovascular thrombectomy (EVT) devices for acute ischemic stroke are often cleared by regulatory agencies on the basis of non-inferiority trials. Defining non-inferiority margins is an essential step for designing non-inferiority trials. However, the relation between the non-inferiority margins used in these trials and the minimal clinically important difference (MCID) determined by experts has never been studied. We performed an online survey to international neuro-interventionalists and noninterventional vascular neurologists and investigate their opinions of MCID for the substantial reperfusion of endovascular thrombectomy devices. According to the reply from 58 international experts, the median MCID for substantial reperfusion was 3.1-5%. Thereafter, we performed systematic searches to identify all non-inferiority design or non-inferiority-presented stroke-EVT trials. Stroke-EVT trial results were reanalyzed using the broad non-inferiority margins originally used and narrower non-inferiority margins derived from formal MCID studies. For both reperfusion and functional outcomes, all 7 trials demonstrated non-inferiority with the broadest non-inferiority margin, but only 4 and 3 trials demonstrated non-inferiority with actual expert-derived margins for reperfusion and functional outcome, respectively. In conclusion, non-inferiority margins may significantly impact the results of non-inferiority trials. The rationale of margin selection in non-inferiority trials needs to be closely reviewed when interpreting the results of the non-inferiority studies.

並列關鍵字

endovascular thrombectomy (EVT) ； minimal clinically important difference (MCID) ； non-inferiority margin ； ischemic stroke

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