An improved method for simple, assumption-free ordinal analysis of the modified Rankin Scale using generalized odds ratios

An improved method for simple, assumption-free ordinal analysis of the modified Rankin Scale using generalized odds ratios
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DOI:
10.1111/ijs.12364
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发表时间:
2014-12-01
影响因子:
6.7
通讯作者:
Donnan, Geoffrey A.
Donnan, Geoffrey A.
中科院分区:
医学2区
文献类型:
--
作者:
Churilov, Leonid;Arnup, Sarah;Donnan, Geoffrey A.

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Background and Purpose: Developing improved methods for analysis of the modified Rankin Scale (mRS) remains a critical issue for the stroke research community. A recently proposed permutation-based approach is assumption-free and easily interpretable but computationally intensive and does not provide confidence intervals to quantify the precision of the effect size estimate. We aimed to develop a method to overcome these limitations.Methods: We propose a procedure using generalized odds ratios to estimate the odds that a patient who received the investigational treatment will have a better outcome than a patient receiving standard treatment. This approach was validated against the permutation method using hypothetical clinical trial scenarios of neuroprotective effect, early recanalization effect, late recanalization effect, and random benefit.Results: The generalized odds ratio approach had strong agreement with the permutation approach provided sample size was >15 patients per treatment arm. Simulation established that the confidence intervals generated were accurate. Ignoring patient pairs with tied mRS scores overestimates the treatment effect compared with splitting tied mRS scores.Conclusions: In addition to all the advantages of the recently proposed permutation-based approach, our method generates confidence intervals without the need for intensive computational power. The resulting generalized odds ratios are particularly suitable for inclusion in meta-analyses and have a simple and intuitive connection with the number-needed-to-treat measure.