Statistical methods and data visualisation of patient-reported outcomes in early phase dose-finding oncology trials: a methodological review.

ABSTRACT:

Background

Traditionally, within dose-finding clinical trials, treatment toxicity and tolerability are assessed by clinicians. Research has shown that clinician reporting may have inadequate inter-rater reliability, poor correlation with patient reported outcomes, and under capture the true toxicity burden. The introduction of patient-reported outcomes (PROs), where the patient can assess their own symptomatic adverse events or quality of life, has potential to complement current practice to aid dose optimisation. There are no international recommendations offering guidance for the inclusion of PROs in dose-finding trial design and analysis. Our review aimed to identify and describe current statistical methods and data visualisation techniques employed to analyse and visualise PRO

SUBMITTER: Alger E 

PROVIDER: S-EPMC10541462 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature