Safroneeva Ekaterina, Schoepfer Alain M
Institute of Social and Preventive Medicine, University of Bern, Bern.
Division of Gastroenterology and Hepatology, Centre Hospitalier Universitaire Vaudois/CHUV, University of Lausanne, Lausanne, Switzerland.
Curr Opin Allergy Clin Immunol. 2019 Apr;19(2):169-174. doi: 10.1097/ACI.0000000000000514.
In adults with eosinophilic esophagitis (EoE), a chronic, inflammatory immune-mediated condition of the esophagus, both inflammation and fibrosis are likely associated with symptom generation. Therefore, assessing symptom-based patient-reported outcomes (PROs), defined by US Food and Drug Administration as 'any report of the status of a patient's health condition that comes directly from the patients, without interpretation of the patient's response by a clinician or anyone else', is important in the context of trials and observational studies of emerging therapies.
For purposes of treatment monitoring, lack of symptoms does not predict the absence of biologic inflammation; hence, endoscopy with esophageal biopsies should be performed to check for residual inflammation. Lack of inflammation does not predict lack of symptoms, and the presence of subepithelial fibrosis cannot be excluded. No published instrument currently measures the frequency of dysphagia described all possible ways, strategies of living with this symptom and various pain types. In randomized controlled trials, in which symptom response was detected using validated PRO measures, only modest decreases in symptom scores were observed.
Accessing full EoE symptom spectrum and optimizing PRO measures remains a challenge that should be tackled to reliably assess response to existing and emerging therapies.
在嗜酸性粒细胞性食管炎（EoE）成人患者中，EoE是一种食管的慢性炎症性免疫介导疾病，炎症和纤维化都可能与症状产生有关。因此，在新兴疗法的试验和观察性研究中，评估基于症状的患者报告结局（PROs）很重要，美国食品药品监督管理局将其定义为“直接来自患者的关于其健康状况的任何报告，无需临床医生或其他任何人对患者的回答进行解读”。
为了进行治疗监测，无症状并不预示不存在生物性炎症；因此，应进行食管活检的内镜检查以检查是否存在残留炎症。无炎症并不预示无症状，且不能排除上皮下纤维化的存在。目前没有已发表的工具能全面测量吞咽困难的频率、应对该症状的生活策略以及各种疼痛类型。在使用经过验证的PRO测量方法检测症状反应的随机对照试验中，仅观察到症状评分有适度下降。
了解完整的EoE症状谱并优化PRO测量方法仍然是一项挑战，为了可靠地评估对现有和新兴疗法的反应，这一挑战应予以应对。
