Complex disorders usually affect multiple symptom domains measured by several outcomes. We recently proposed a novel composite desirability of outcome ranking (DOOR) approach for patient-centered evaluation of overall treatment benefits and developed the Patient-ranked Order of Function (PROOF) outcome for the evaluation of efficacy of amyotrophic lateral sclerosis (ALS) clinical trials. In this talk, Lu will introduce the motivation of the DOOR approach to incorporate individualized patient preferences, previous work to use the composite endpoint for clinical trials and an extension of this approach to a cohort study setting. He will report the results of 2021 and 2022 surveys of patients in the Netherlands ALS registry for their associations with survival time, factors associated with PROOF outcomes, and analysis of longitudinal data of the composite DOOR ranking. This is joint work with Dr. van Eijk and colleagues at Stanford University and UMC Utrecht.
Speaker: Ying Lu, PhD, Professor of Biomedical Data Science, Stanford University
To obtain Zoom link please email: http://[email protected]