Dysphagia Symptom Score for Eosinophilic Esophagitis
Dysphagia Symptom Score for Eosinophilic Esophagitis
Background Dysphagia is the hallmark of eosinophilic esophagitis (EoE), but no validated dysphagia instruments in this population exist.
Aim To develop and field test a patient-reported outcome (PRO) for dysphagia in subjects with EoE.
Methods This was a multi-centre/multi-phase prospective study. The first phase developed a dysphagia questionnaire using qualitative methods. The second phase was a 30-day field trial to test the instrument and assess content validity. Adolescents and adults with EoE, active symptoms of dysphagia and oesophageal eosinophilia (≥15 eosinophils per high-power field) were enrolled. Solid-food-avoidance days, dysphagia days and actions taken to get relief were recorded. A dysphagia score was calculated and compared to the Straumann Dysphagia Instrument (SDI).
Results Ten adolescents and 10 adults were included in the first phase and the Dysphagia Symptom Questionnaire (DSQ), a three-item daily electronic diary, was developed. In the second phase, 35 subjects finished the field trial (18 adults, 17 adolescents, mean age 24, 54% male, 95% white, 54% currently on topical corticosteroids). The median number of dysphagia days per week was 2 for adolescents vs. 4 for adults (P < 0.001), and 2 for those on topical steroids vs. 4 for those not on topical steroids (P < 0.001). The DSQ score strongly correlated with the number of dysphagia days (R = 0.96; P < 0.001) and the SDI (R = 0.77; P < 0.001).
Conclusions The DSQ, a three-question patient-reported outcome, was successfully developed and field tested. The DSQ had content validity and the score accurately measured dysphagia frequency and intensity. The Dysphagia Symptom Questionnaire is suitable for use in clinical trials of EoE patients with dysphagia.
Eosinophilic esophagitis (EoE) is an immune-mediated clinicopathological condition currently defined by symptoms of oesophageal dysfunction and a marked infiltration of the oesophageal mucosa with eosinophils. First described in its current form in the early 1990s, over the past decade the recognition, incidence and prevalence of EoE have increased, and EoE is now found in a large proportion of children and adults undergoing endoscopy for upper gastrointestinal symptoms.
In adolescents and adults, dysphagia is the most common symptom of EoE, and has been reported nearly universally in some series. Accordingly, clinical trials of therapeutic agents for EoE have frequently focused on this symptom as a primary outcome, but results have been conflicting. In a number of studies, although there is typically an improvement in histology with decreasing oesophageal eosinophil counts in subjects who received active medication, symptoms have not always improved above the rate seen in the placebo group. The discordance in these results is perplexing, and there are several possible explanations. First, there may be a simple lack of agreement between eosinophil counts and symptoms due to the pathophysiological nature of the condition. Second, because the understanding of EoE is still evolving, there are likely underappreciated or unrecognised factors at play. Fibrostenosis has recently been associated with symptom outcomes in EoE independent of oesophageal eosinophilia. Third, although there are published diagnostic guidelines for EoE, these are not universally followed, and if patients included in studies are misclassified as having EoE, their symptoms response may not be predictable. Fourth, because outcome measures have not been validated for EoE, quantification of symptoms may not have been accurate.
Accurately quantifying symptoms of dysphagia in EoE is a major challenge for studying existing medications and developing new pharmacological agents to treat EoE, because a well-developed patient-reported outcome (PRO) is required for drug approval by the US Food and Drug Administration (FDA). The aim of this study was to develop and field test a PRO for dysphagia in adolescents and young adults with EoE that meets FDA guidelines and could be used as a reliable outcome measure in future clinical trials.
Abstract and Introduction
Abstract
Background Dysphagia is the hallmark of eosinophilic esophagitis (EoE), but no validated dysphagia instruments in this population exist.
Aim To develop and field test a patient-reported outcome (PRO) for dysphagia in subjects with EoE.
Methods This was a multi-centre/multi-phase prospective study. The first phase developed a dysphagia questionnaire using qualitative methods. The second phase was a 30-day field trial to test the instrument and assess content validity. Adolescents and adults with EoE, active symptoms of dysphagia and oesophageal eosinophilia (≥15 eosinophils per high-power field) were enrolled. Solid-food-avoidance days, dysphagia days and actions taken to get relief were recorded. A dysphagia score was calculated and compared to the Straumann Dysphagia Instrument (SDI).
Results Ten adolescents and 10 adults were included in the first phase and the Dysphagia Symptom Questionnaire (DSQ), a three-item daily electronic diary, was developed. In the second phase, 35 subjects finished the field trial (18 adults, 17 adolescents, mean age 24, 54% male, 95% white, 54% currently on topical corticosteroids). The median number of dysphagia days per week was 2 for adolescents vs. 4 for adults (P < 0.001), and 2 for those on topical steroids vs. 4 for those not on topical steroids (P < 0.001). The DSQ score strongly correlated with the number of dysphagia days (R = 0.96; P < 0.001) and the SDI (R = 0.77; P < 0.001).
Conclusions The DSQ, a three-question patient-reported outcome, was successfully developed and field tested. The DSQ had content validity and the score accurately measured dysphagia frequency and intensity. The Dysphagia Symptom Questionnaire is suitable for use in clinical trials of EoE patients with dysphagia.
Introduction
Eosinophilic esophagitis (EoE) is an immune-mediated clinicopathological condition currently defined by symptoms of oesophageal dysfunction and a marked infiltration of the oesophageal mucosa with eosinophils. First described in its current form in the early 1990s, over the past decade the recognition, incidence and prevalence of EoE have increased, and EoE is now found in a large proportion of children and adults undergoing endoscopy for upper gastrointestinal symptoms.
In adolescents and adults, dysphagia is the most common symptom of EoE, and has been reported nearly universally in some series. Accordingly, clinical trials of therapeutic agents for EoE have frequently focused on this symptom as a primary outcome, but results have been conflicting. In a number of studies, although there is typically an improvement in histology with decreasing oesophageal eosinophil counts in subjects who received active medication, symptoms have not always improved above the rate seen in the placebo group. The discordance in these results is perplexing, and there are several possible explanations. First, there may be a simple lack of agreement between eosinophil counts and symptoms due to the pathophysiological nature of the condition. Second, because the understanding of EoE is still evolving, there are likely underappreciated or unrecognised factors at play. Fibrostenosis has recently been associated with symptom outcomes in EoE independent of oesophageal eosinophilia. Third, although there are published diagnostic guidelines for EoE, these are not universally followed, and if patients included in studies are misclassified as having EoE, their symptoms response may not be predictable. Fourth, because outcome measures have not been validated for EoE, quantification of symptoms may not have been accurate.
Accurately quantifying symptoms of dysphagia in EoE is a major challenge for studying existing medications and developing new pharmacological agents to treat EoE, because a well-developed patient-reported outcome (PRO) is required for drug approval by the US Food and Drug Administration (FDA). The aim of this study was to develop and field test a PRO for dysphagia in adolescents and young adults with EoE that meets FDA guidelines and could be used as a reliable outcome measure in future clinical trials.
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