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Lookup NU author(s): Professor James WasonORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2017 The Authors. Statistics in Medicine Published by John Wiley & Sons Ltd. In many phase II trials in solid tumours, patients are assessed using endpoints based on the Response Evaluation Criteria in Solid Tumours (RECIST) scale. Often, analyses are based on the response rate. This is the proportion of patients who have an observed tumour shrinkage above a predefined level and no new tumour lesions. The augmented binary method has been proposed to improve the precision of the estimator of the response rate. The method involves modelling the tumour shrinkage to avoid dichotomising it. However, in many trials the best observed response is used as the primary outcome. In such trials, patients are followed until progression, and their best observed RECIST outcome is used as the primary endpoint. In this paper, we propose a method that extends the augmented binary method so that it can be used when the outcome is best observed response. We show through simulated data and data from a real phase II cancer trial that this method improves power in both single-arm and randomised trials. The average gain in power compared to the traditional analysis is equivalent to approximately a 35% increase in sample size. A modified version of the method is proposed to reduce the computational effort required. We show this modified method maintains much of the efficiency advantages.
Author(s): Lin C-J, Wason JMS
Publication type: Article
Publication status: Published
Journal: Statistics in Medicine
Print publication date: 20/12/2017
Online publication date: 28/08/2017
Acceptance date: 07/08/2017
Date deposited: 13/12/2017
ISSN (print): 0277-6715
ISSN (electronic): 1097-0258
Publisher: John Wiley and Sons Ltd
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