Gastroenterologists' Assessment of EoE in Adults
Gastroenterologists' Assessment of EoE in Adults
Objectives There is no "gold standard" for assessing disease activity in patients with eosinophilic esophagitis (EoE). We aimed to compare physicians' judgment of EoE activity with patients' judgment of symptom severity. We also aimed to examine the relative contribution of symptoms as well as endoscopic and histologic findings in shaping physicians' judgment of EoE activity.
Methods Six gastroenterologists (all EoE experts) assessed EoE-associated symptoms in adult patients. Patients completed a symptom instrument and provided global assessment of EoE symptom severity (PatGA) (Likert scale: 0 (inactive) to 10 (most active)). Following esophagogastroduodenoscopy with biopsy sampling, gastroenterologists provided a global assessment of EoE activity (PhysGA) (Likert scale from 0 to 10) based on patient history and endoscopic and histologic findings. Linear regression and analysis of variance was used to quantify the extent to which variations in severity of EoE symptoms and endoscopic and histologic findings explain variations in PhysGA.
Results A total of 149 EoE patients were prospectively included (71.8% male, median age at inclusion 38 years, 71.8% with concomitant allergies). A moderate positive correlation between PhysGA and PatGA (rho=0.442, P<0.001) was observed and the mean difference in the Bland–Altman plot was 1.77. Variations in severity of endoscopic findings, symptoms, and histologic findings alone explained 53%, 49%, and 30%, of the variability in PhysGA, respectively. Together, these findings explained 75% of variability in PhysGA.
Conclusions Gastroenterologists rate EoE activity mainly on the basis of endoscopic findings and symptoms and, to a lesser extent, on histologic findings.
Eosinophilic esophagitis (EoE) has been defined as "a chronic, immune/antigen-mediated, esophageal disease characterized clinically by symptoms related to esophageal dysfunction and histologically by eosinophil-predominant inflammation". EoE is a "young" disease, as the first case series describing it as a distinct entity were published a little over 20 years ago. A current prevalence of ~1/2,000 has been reported for adult EoE patients in Europe and the United States. Dysphagia is the leading symptom of adult EoE patients, but swallowing-associated pain and heartburn not responding to acid-suppressive medication can also occur.
The activity of a given disease can be assessed using various patient-reported outcome (PRO) measures, biologic markers, or a combination of both. The choice of the appropriate entity as an end point to measure the activity of a given disease depends on the influence of this entity on the natural history of the disease. As an example, arterial hypertension is assessed by measuring blood pressure (biomarker); if elevated, it can be associated with myocardial infarction (outcome in the natural history). Assessing PRO measures is of limited value in arterial hypertension, as this disease is not necessarily associated with any symptoms. On the other hand, the activity of diseases, such as low back pain or migraine, is mainly assessed using PRO measures (pain) and assessing biomarkers (e.g., magnetic resonance imaging of the spine or brain) is of limited value. In EoE, both PRO measures of symptoms and psychosocial functioning as well as biologic markers have been evaluated as end points in clinical trials. Symptom severity correlates with severity of EoE-associated endoscopic and/or histologic alterations in some but not all clinical studies. The utility of PRO measures and biologic markers as end points in clinical studies and observational trials is, therefore, the subject of ongoing research and debate.
In clinical practice, the judgment of disease activity is based on very diverse sources of information, such as patient complaints, physical examination, and endoscopic, laboratory, and histologic findings. Physicians synthesize all of this information in order to generate an overall impression of disease activity. The physicians' overall impression of disease activity is frequently sought in both observational studies and clinical trials in a variety of different conditions including ankylosing spondylitis, systemic lupus erythematosus, and rheumatoid and psoriatic arthritis. Physicians are likely to consider biologic measures as most important in assessing disease activity because of the perceived objective nature of these data and put less weight on PRO measures. In the absence of an accepted "gold standard" for assessing disease activity in EoE, the physician global assessment of EoE activity (PhysGA) as quintessence of the expert opinion is often regarded as the best available measure of overall EoE activity. However, the extent to which this expert opinion is explained by EoE-associated symptoms and endoscopic and histologic findings is currently unknown.
In 2009, the International Eosinophilic Esophagitis Activity Index (EEsAI) study group set out to develop different instruments that assess EoE activity from physician's and patient's perspective. The EEsAI instruments for physicians examine EoE-associated endoscopic and histologic findings, and the recently validated EEsAI PRO instrument assesses various characteristics of dysphagia, swallowing-associated pain, and behavioral adaptations to living with dysphagia. The objective of this study was to evaluate the relationship between physician assessment of EoE activity and patient assessment of EoE symptom severity and to examine the contributions of endoscopic and histologic findings as well as symptoms to the physician assessment of EoE activity.
Abstract and Introduction
Abstract
Objectives There is no "gold standard" for assessing disease activity in patients with eosinophilic esophagitis (EoE). We aimed to compare physicians' judgment of EoE activity with patients' judgment of symptom severity. We also aimed to examine the relative contribution of symptoms as well as endoscopic and histologic findings in shaping physicians' judgment of EoE activity.
Methods Six gastroenterologists (all EoE experts) assessed EoE-associated symptoms in adult patients. Patients completed a symptom instrument and provided global assessment of EoE symptom severity (PatGA) (Likert scale: 0 (inactive) to 10 (most active)). Following esophagogastroduodenoscopy with biopsy sampling, gastroenterologists provided a global assessment of EoE activity (PhysGA) (Likert scale from 0 to 10) based on patient history and endoscopic and histologic findings. Linear regression and analysis of variance was used to quantify the extent to which variations in severity of EoE symptoms and endoscopic and histologic findings explain variations in PhysGA.
Results A total of 149 EoE patients were prospectively included (71.8% male, median age at inclusion 38 years, 71.8% with concomitant allergies). A moderate positive correlation between PhysGA and PatGA (rho=0.442, P<0.001) was observed and the mean difference in the Bland–Altman plot was 1.77. Variations in severity of endoscopic findings, symptoms, and histologic findings alone explained 53%, 49%, and 30%, of the variability in PhysGA, respectively. Together, these findings explained 75% of variability in PhysGA.
Conclusions Gastroenterologists rate EoE activity mainly on the basis of endoscopic findings and symptoms and, to a lesser extent, on histologic findings.
Introduction
Eosinophilic esophagitis (EoE) has been defined as "a chronic, immune/antigen-mediated, esophageal disease characterized clinically by symptoms related to esophageal dysfunction and histologically by eosinophil-predominant inflammation". EoE is a "young" disease, as the first case series describing it as a distinct entity were published a little over 20 years ago. A current prevalence of ~1/2,000 has been reported for adult EoE patients in Europe and the United States. Dysphagia is the leading symptom of adult EoE patients, but swallowing-associated pain and heartburn not responding to acid-suppressive medication can also occur.
The activity of a given disease can be assessed using various patient-reported outcome (PRO) measures, biologic markers, or a combination of both. The choice of the appropriate entity as an end point to measure the activity of a given disease depends on the influence of this entity on the natural history of the disease. As an example, arterial hypertension is assessed by measuring blood pressure (biomarker); if elevated, it can be associated with myocardial infarction (outcome in the natural history). Assessing PRO measures is of limited value in arterial hypertension, as this disease is not necessarily associated with any symptoms. On the other hand, the activity of diseases, such as low back pain or migraine, is mainly assessed using PRO measures (pain) and assessing biomarkers (e.g., magnetic resonance imaging of the spine or brain) is of limited value. In EoE, both PRO measures of symptoms and psychosocial functioning as well as biologic markers have been evaluated as end points in clinical trials. Symptom severity correlates with severity of EoE-associated endoscopic and/or histologic alterations in some but not all clinical studies. The utility of PRO measures and biologic markers as end points in clinical studies and observational trials is, therefore, the subject of ongoing research and debate.
In clinical practice, the judgment of disease activity is based on very diverse sources of information, such as patient complaints, physical examination, and endoscopic, laboratory, and histologic findings. Physicians synthesize all of this information in order to generate an overall impression of disease activity. The physicians' overall impression of disease activity is frequently sought in both observational studies and clinical trials in a variety of different conditions including ankylosing spondylitis, systemic lupus erythematosus, and rheumatoid and psoriatic arthritis. Physicians are likely to consider biologic measures as most important in assessing disease activity because of the perceived objective nature of these data and put less weight on PRO measures. In the absence of an accepted "gold standard" for assessing disease activity in EoE, the physician global assessment of EoE activity (PhysGA) as quintessence of the expert opinion is often regarded as the best available measure of overall EoE activity. However, the extent to which this expert opinion is explained by EoE-associated symptoms and endoscopic and histologic findings is currently unknown.
In 2009, the International Eosinophilic Esophagitis Activity Index (EEsAI) study group set out to develop different instruments that assess EoE activity from physician's and patient's perspective. The EEsAI instruments for physicians examine EoE-associated endoscopic and histologic findings, and the recently validated EEsAI PRO instrument assesses various characteristics of dysphagia, swallowing-associated pain, and behavioral adaptations to living with dysphagia. The objective of this study was to evaluate the relationship between physician assessment of EoE activity and patient assessment of EoE symptom severity and to examine the contributions of endoscopic and histologic findings as well as symptoms to the physician assessment of EoE activity.
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