Improved stratification of ALS clinical trials using predicted survival.

ABSTRACT:

Introduction

In small trials, randomization can fail, leading to differences in patient characteristics across treatment arms, a risk that can be reduced by stratifying using key confounders. In ALS trials, riluzole use (RU) and bulbar onset (BO) have been used for stratification. We hypothesized that randomization could be improved by using a multifactorial prognostic score of predicted survival as a single stratifier.

Methods

We defined a randomization failure as a significant difference between treatment arms on a characteristic. We compared randomization failure rates when stratifying for RU and BO ("traditional stratification") to failure rates when stratifying for predicted survival using a predictive algorithm. We simulated virtual trials using the PRO-ACT database wi

SUBMITTER: Berry JD 

PROVIDER: S-EPMC5899911 | biostudies-literature | 2018 Apr

REPOSITORIES: biostudies-literature