Center for Esophageal Diseases and Swallowing, Division of Gastroenterology and Hepatology, University of North Carolina School of Medicine, Chapel Hill, North Carolina; Center for Gastrointestinal Biology and Disease, Division of Gastroenterology and Hepatology, University of North Carolina School of Medicine, Chapel Hill, North Carolina.
Division of Pediatric Gastroenterology, University of Illinois College of Medicine, Peoria, IL.
Clin Gastroenterol Hepatol. 2019 Oct;17(11):2149-2160. doi: 10.1016/j.cgh.2019.01.030. Epub 2019 Jan 30.
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.
虽然嗜酸粒细胞性食管炎的诊断和初始治疗变得更加标准化，但在测量治疗反应方面仍存在许多知识空白。其中一个问题是如何衡量治疗反应以及应该使用哪些治疗终点。这不仅影响患者的护理和参与决策，也影响药物开发领域。此外，迄今为止的研究使用了多种治疗终点，包括十几种组织学终点标准。这篇综述将讨论评估复杂疾病治疗终点的各种利益相关者，包括患者、医生和监管机构，以及评估治疗反应的护理环境，包括常规临床护理、临床试验和观察性研究。还讨论了潜在的参数或治疗终点，如组织学、症状、患者报告的结果、内镜检查和生物标志物，以及相关的挑战和机遇。还讨论了如何定义治疗结果的框架，并提出了一种治疗反应的概念方法。这考虑到了组织学、症状和内镜检查结果，并利用了现有的经过验证的工具。它包括无反应、完全正常化和这两个极端之间的分级反应类别定义，并且还允许在出现新的知识时灵活调整和放宽。此外，还讨论了如何在这些努力中定位儿科人群以及未来的研究方向。
