A Conceptual Approach to Understanding Treatment Response in Eosinophilic Esophagitis

Abstract

While the diagnosis and initial treatment of eosinophilic esophagitis are becoming more standardized, there are still major gaps in knowledge related to measuring treatment response. One such question centers on how to measure treatment response and what treatment endpoints should be. This impacts not only patient care and engagement in decision-making, but also the field of drug development. In addition, studies so far have use a myriad of treatment endpoints including over a dozen histologic endpoint criteria. This review will discuss the various stakeholders involved in assessment of treatment endpoints of a complex condition, including patients, practitioners and regulatory agencies, and the care settings in which treatment response is assessed, including routine clinical care, clinical trials, and observational studies. Potential parameters or treatment endpoints such as histology, symptoms, patient-reported outcomes, endoscopy, and biomarkers are discussed along with associated challenges and opportunities. A framework on how to define treatment outcomes is discussed and a conceptual approach treatment response is proposed. This takes into account histology, symptoms, and endoscopic findings and harnesses existing, validated tools. It includes definitions of nonresponse, complete normalization, and a graded response category between these 2 extremes, and also permits flexibility and latitude for modifications as newer knowledge emerges. In addition, ways to position the pediatric population in these endeavors are discussed as are future research directions.