2905. Development of a Desirability of Outcome Ranking (DOOR) for Skin Infection Clinical Trials

Abstract

Abstract Background Infectious disease clinical trials treatment outcomes have almost exclusively been dichotomized into cure and failure. The novel Desirability of Outcome Ranking (DOOR) method uses a hierarchical ordinal outcome of intervention outcomes. DOOR measures are more sensitive for detection of differences between groups compared to dichotomous outcomes and thus can lead to dramatic reduction of the number of required trial participants. Additionally, the DOOR design addresses patient-centered outcomes that are ignored in traditional infectious diseases clinical trial outcomes. DOOR methodology has neither been used for skin and soft tissue infection (SSTI) clinical trial outcomes nor incorporated patients about their experiences with SSTIs. Methods We conducted 6 focus groups of providers, patients, and parents at two medical centers in Los Angeles and St. Louis. Focus groups were composed of clinicians who treat SSTIs (n=2), patients who had SSTIs (n=3) and parents of children who had an SSTI (n=1). Focus group participants were given a series of SSTI clinical scenarios with various outcomes and asked to rank these 10 outcomes on a numerical scale from most to least desirable. We fitted 4 CART (classification and regression tree) models for overall, provider, patient and parent responders, respectively. Results In total, we had 62 focus group participants (24 providers, 30 patients, and 8 parents). Overall, the most desirable outcome was no complications (rank value = 1.0), followed by rash or nausea (mean rank values = 4.9 - 5.1), diarrhea or recurrent infection (= 5.9), dizziness or stomach ache (= 6.1 - 6.2), recurrent drainage procedure or household transmission (= 7.0 - 7.2), and hospitalization (= 8.3). Least desirable outcomes differed between groups, with providers choosing hospitalization while patient/parents indicated household transmission. Variance in outcome desirability differed more widely between patients compared to providers. (Figure). Conclusion We developed a DOOR outcome for SSTIs clinical trials that incorporates both provider and patient perspectives. This metric could be tested in clinical trials as a means to reduce sample size and to incorporate patient-focused outcomes. Disclosures Loren G. Miller, MD MPH, ContraFect: Grant/Research Support|GSK: Grant/Research Support|Medline: Grant/Research Support|Merck: Grant/Research Support|Paratek: Grant/Research Support