Parent-reported responses to the Pediatric Eosinophilic Esophagitis Symptom Score questionnaire, corresponded to clinical and biological features of the allergic disease in their children, according to recent study data.
“We have validated a survey document, nowadays referred to as a patient reported outcome (PRO) metric, which we call ‘pediatric eosinophilic esophagitis symptom score (PEESS) version 2,’” Marc E. Rothenberg, MD, PhD, from Cincinnati Children’s and the University of Cincinnati School of Medicine, told Healio Gastroenterology. “This type of instrument is useful to measure patient well-being, especially when tracking the impact of a therapy. The FDA is now focused on using PROs to truly gauge the value of new drugs.”
The PEESS v2.0 is a content-validated metric designed to identify eosinophilic esophagitis (EoE) symptoms reported from children and parents. It has a 100-point scale and four domains: dysphagia, GERD, nausea/vomiting and pain.
“Because EoE is a disease with multiple symptoms, the ability to capture patient and parent perceptions of these symptoms is a major unmet need,” Lisa J. Martin, PhD, professor of human genetics at Cincinnati Children's, said in a press release. “By having validated tools to measure how patients feel, our reported findings provide a new opportunity for evaluating treatments going forward. The validation of the PEESS symptoms with biologically and clinically pertinent assessments is therefore a major step forward for improving the outcomes in patients with EoE.”
Marc E. Rothenberg
Martin, Rothenberg and colleagues recruited 46 children with treated and untreated EoE (median age, 6.9 years; median duration since diagnosis, 2.4 years), and collected PEESS v2.0 scores and esophageal biopsy samples. They then aimed to validate parent-reported scores by identifying associations with clinical and biological features of EoE.
“We used the PEESS v2.0 to show that specific elements of the clinical symptoms (such as dysphagia) track with cellular and molecular components of the disease,” Rothenberg said. “We had a unique opportunity to do this as we were able to get tissue biopsies from all patients and then perform deep molecular and comprehensive histological probing to understand which clinical features were related to the tissue processes.”
They found the PEESS domains correlated with parent-reported symptoms of dysphagia (P = .0012), GERD (P = .0001), and nausea/vomiting (P < .0001); and pain correlated with multiple symptoms (P < .0005). They also observed esophageal activity markers were associated with dysphagia (strongest r = 0.37; P = .02). Distal eosinophil count was most strongly associated with the pain domain (r = 0.28; P = .06) and proximal eosinophil count was most strongly associated with the dysphagia domain (r = 0.24; P = .13). The dysphagia domain was most strongly correlated with esophageal gene transcript levels, primarily with mast cell-specific genes.
“We not only validated the PRO instrument, proved that parents can accurately complete the form for young children (proxy), but we also identified that the cardinal symptom (dysphagia) was strongly related to tissue mast cell levels and markers of eosinophil activation, as well as specific genetic signatures,” Rothenberg said. – by Adam Leitenberger
Disclosures: Martin reports she has received research support from the National Institutes of Health and the Children’s Heart Foundation. Please see the study for a full list of all other authors’ relevant financial disclosures.
Editor's Note: This article was updated on June 11 to reflect additional information.