Win statistics in pulmonary arterial hypertension clinical trials

To the Editor: Pulmonary arterial hypertension (PAH) is a rare and progressive disease that often leads to right heart failure and death. Although most phase III PAH trials have used 6-minute-walk distance (6MWD) as the primary endpoint, others have used composite outcomes. Analyses of time to clinical worsening in PAH trials have typically counted only the first event and equally weighted the clinical impact of events. For example, adding a new PAH medication in one arm of the trial would be equivalent to a hospitalization in the other, despite patients potentially ranking one as more important. Win statistics account for the relative priority of the individual components of a composite endpoint (i.e., they are hierarchal outcome measures). These may better reflect the benefit-risk trade-off for individual patients than current approaches. The aim of this study was to derive and assess the win ratio, win odds, and net benefit in PAH clinical trials.

Canadian Institutes of Health Research
Heart and Stroke Foundation of Canada
National Heart, Lung, and Blood Institute
Cardiovascular Medical Research and Education Fund

Jason Weatherald, Jude Moutchia, Nadine Al-Naamani, Robyn L McClelland, Corey E Ventetuolo, Harold I Palevsky, Michael O Harhay, Steven M Kawut