@article{b51e2e4367514873a83f7b25f5818270,
title = "Good studies evaluate the disease while great studies evaluate the patient: Development and Application of a Desirability of Outcome Ranking Endpoint for Staphylococcus aureus Bloodstream Infection",
abstract = "Background. Desirability of outcome ranking (DOOR) is an innovative approach in clinical trials to evaluate the global benefits and risks of an intervention. We developed and validated a DOOR endpoint for Staphylococcus aureus bloodstream infection (BSI) through a survey to infectious diseases clinicians and secondary analysis of trial data. Methods. We administered a survey of 20 cases of S. aureus BSI, asking respondents to rank outcomes by global desirability. Correlations and percentage of pairwise agreement among rankings were estimated to inform development of a DOOR endpoint, which was applied to 2 prior S. aureus BSI trials. The probability that a patient randomly assigned to experimental treatment would have a better DOOR ranking than if assigned to control was estimated. Results were also analyzed using partial credit, which is analogous to scoring an academic test, assigning 100% to the most desirable outcome, 0% to the least, and “partial credit” to intermediate ranks. Results. Forty-two recipients (97%) completed the survey. The DOOR endpoint fitting these rankings (r = 0.89; 95% confidence interval, 0.67 to 0.94) incorporated survival plus cumulative occurrence of adverse events, cure, infectious complications, and ongoing symptoms. Tailored versions of this endpoint were applied to 2 S. aureus BSI trials, and both demonstrated no benefit of the experimental treatment using DOOR and partial credit analysis. Conclusions. Using S. aureus BSI as an exemplar, we developed a DOOR endpoint that can be used as a template for development of DOOR endpoints for other diseases. Future trials can incorporate DOOR to allow for global assessment of patient experience.",
keywords = "Bloodstream infection, Clinical trial, Staphylococcus aureus",
author = "{Antibacterial Resistance Leadership Group} and Doernberg, {Sarah B.} and Tran, {Thuy Tien Tram} and Tong, {Steven Y.C.} and Mical Paul and Dafna Yahav and Davis, {Joshua S.} and Leonard Leibovici and Boucher, {Helen W.} and {Ralph Corey}, G. and Cosgrove, {Sara E.} and Chambers, {Henry F.} and Fowler, {Vance G.} and Evans, {Scott R.} and Holland, {Thomas L.}",
note = "Publisher Copyright: {\textcopyright} The Author(s) 2018. Published by Oxford University Press for the Infectious Diseases Society of America. All rights reserved.",
year = "2019",
month = may,
day = "1",
doi = "10.1093/cid/ciy766",
language = "אנגלית",
volume = "68",
pages = "1691--1698",
journal = "Clinical Infectious Diseases",
issn = "1058-4838",
publisher = "Oxford University Press",
number = "10",
}