Speaker
Anup Katheria, MD
Director of the Neonatal Research Institute
Sharp Mary Birch Hospital for Women and Newborns
Keywords
Endpoint; Outcome; DOOR; Neonatology; Patient-Important Outcomes
Key Points
- Neonatal trials often use binary and composite outcomes that fail to distinguish differential importance of events – death, for instance, being more impactful than a non-fatal event – and obscure the differences that matter most to families. There is a need to integrate multiple endpoints into outcome measures that reflect relative importance.
- The Desirability Of Outcome Ranking (DOOR) approach shifts the unit of analysis from outcomes to overall patient improvement. When applied retroactively to trials involving delayed cord clamping and cord milking, it revealed a clinical benefit that was originally masked by traditional composite outcomes.
- This “whole-child” approach comes with its own set of challenges. Parents care about long-term outcomes, and long-term follow-up is often not feasible. The study team, seeking to identify short-term neonatal intensive care unit outcomes that predict meaningful long-term outcomes, created a neonatal inpatient outcome called neoDOOR.
Discussion Themes
Trials that utilize DOOR will still require significant power to detect clinical differences.
Dr. Katheria emphasized the importance that neonatology adopts advanced trial methodologies that are already more common in adult medicine, e.g. platform trials.