A latent variable model for improving inference in trials assessing the effect of dose on toxicity and composite efficacy endpoints.

ABSTRACT: It is often of interest to explore how dose affects the toxicity and efficacy properties of a novel treatment. In oncology, efficacy is often assessed through response, which is defined by a patient having no new tumour lesions and their tumour size shrinking by 30%. Usually response and toxicity are analysed as binary outcomes in early phase trials. Methods have been proposed to improve the efficiency of analysing response by utilising the continuous tumour size information instead of dichotomising it. However, these methods do not allow for toxicity or for different doses. Motivated by a phase II trial testing multiple doses of a treatment against placebo, we propose a latent variable model that can estimate the probability of response and no toxicity (or other related outcomes) for diff

SUBMITTER: Wason JM 

PROVIDER: S-EPMC6986906 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature