Alternative outcome measures for pediatric clinical sepsis trials

Abstract

To review nonmortality outcome measures in clinical trials of therapies to treat sepsis in children. Literature review using the search word terms "sepsis" + "surrogate markers," "sepsis" + "biomarkers," or "sepsis" + "outcomes." Articles were generally categorized as those dealing with review of patient-centered outcomes, characteristics of good surrogate markers, resolution of organ dysfunction, morbidity and functional status, quality-of-life measures, intensive care unit costs, and biomarkers. Information potentially relevant for development of surrogate markers for pediatric sepsis trials was extracted and organized as noted above. Multiple potential surrogate markers are being actively investigated for their potential validity and utility in pediatric clinical trials of sepsis and increasingly for adult sepsis trials. This is important because as mortality decreases, it becomes an impractical primary end point. Surrogate end points that address patient-related morbidity and intensive care unit costs may also be quantified. Treatment of sepsis and corresponding end points for clinical trials should be aimed at both the duration and the quality of survival.