Co-designing a Novel Ordinal Endpoint for an Adaptive Platform Trial, BANDICOOT, in Pediatric Hematopoietic Stem Cell Transplant

Abstract

An adaptive platform trial (APT) offers the ability to incorporate several research questions in the same target population across multiple domains (interventions), with the ability to add new questions in a perpetual manner. An APT is particularly appealing for pediatric hematopoietic stem cell transplant (HCT); an area of high heterogeneity, limited trial availability, and high mortality. Ideally, all domains in an APT would have the same primary endpoint. Therefore, an ordinal endpoint with multiple categories that combines various clinical outcomes into a single outcome measure is particularly appealing for APTs. Unfortunately, there is no accepted ordinal endpoint for pediatric HCT tria..