Clin Infect Dis. 2025 Jun 13:ciaf314. doi: 10.1093/cid/ciaf314. Online ahead of print.
ABSTRACT
Hierarchical composite endpoints (HCEs), combining features of simple composite endpoints and conventional ordinal endpoints, are increasingly being used in infectious diseases research. However, many clinicians may be unfamiliar with these novel endpoints, including the variety of different target parameters that may be of interest and the methods that can be used to estimate them. In this review, we provide a conceptual overview of HCEs by defining them and providing examples from the infectious diseases literature. We explain different methods for analyzing HCEs, including: (a) the Wilcoxon rank sum approach (often used in studies with a Desirability of Outcome Ranking [DOOR] endpoint); (b) generalized pairwise comparisons (used to estimate a win ratio or win odds); (c) proportional odds model (and the relevance of the proportional odds assumption); and, (d) the probabilistic index model. This review will help infectious disease clinicians and healthcare providers interpret current and future research using such endpoints.
PMID:40512968 | DOI:10.1093/cid/ciaf314
