Predictors of response to steroid therapy for eosinophilic esophagitis and treatment of steroid-refractory patients.

BACKGROUND & AIMS: Eosinophilic esophagitis (EoE) is commonly treated with swallowed (topical) corticosteroids (tCS). However, few factors have been described that predict outcomes of steroid therapy. We aimed to identify factors associated with nonresponse to tCS and report outcomes of second-line treatment for patients with steroid-refractory EoE. METHODS: We performed a retrospective cohort study by using the University of North Carolina EoE Clinicopathologic Database to identify patients who received tCS for EoE from 2006 through 2013. Demographic, symptom, endoscopic, and histologic data were extracted from medical records. Immunohistochemistry was performed on archived biopsies. Responders and nonresponders to tCS were compared. RESULTS: Of 221 patients with EoE who received tCS, 71% had endoscopic improvement, 79% had symptomatic improvement, and 57% had histologic response (<15 eosinophils/high-power field). After multivariate logistic regression, esophageal dilation at the baseline examination predicted nonresponse (odds ratio, 2.9; 95% confidence interval, 1.4-6.3), and abdominal pain predicted response (odds ratio for nonresponse, 0.31; 95% confidence interval, 0.12-0.83); no other clinical features were predictive. On the basis of immunohistochemical analysis, higher baseline levels of tryptase (244 vs 157 mast cells/mm(2), P = .04) and eotaxin-3 (2425 vs 239 cells/mm(2), P = .02) were associated with steroid response, but levels of major basic protein were not. Among 27 steroid-refractory patients, a mean of 2 additional therapies were tried; only 48% of the patients eventually responded to any second-line therapy. CONCLUSIONS: On the basis of a retrospective analysis of a large group of patients with EoE, only 57% have a histologic response to steroid therapy. Baseline esophageal dilation and decreased levels of mast cells and eotaxin-3 predicted which patients would not respond to therapy. Combining clinical factors and immunohistochemistry might therefore be used to direct therapy.

Evaluation of Histologic Cutpoints for Treatment Response in Eosinophilic Esophagitis.

AIM: No consensus exists on the definition of successful treatment in eosinophilic esophagitis (EoE). The aim of this study was to identify the optimal histologic cutpoint to define successful treatment of EoE by assessing rates of symptomatic and endoscopic improvement. MATERIALS AND METHODS: We performed a retrospective cohort study utilizing the University of North Carolina EoE Clinicopathologic Database between 2006 and 2013. Rates of symptomatic and endoscopic improvement were determined, as were post-treatment eosinophil counts. The area under the receiver operator characteristic curve (AUC) was calculated for symptomatic and endoscopic response at several possible eosinophil count cutpoints (eos/hpf). Predictors of response were also assessed. RESULTS: Of 224 treatments in 199 patients, 76% were associated with symptomatic improvement, 68% with endoscopic improvement, and 60% with both. Of treatments that resulted in a post-treatment count of <15 eos/hpf, 90% were associated with an endoscopic response, 88% with a symptomatic response, and 81% with both symptomatic and endoscopic responses. Using a <15 eos/hpf threshold, the area under the curves (AUCs) were 0.70, 0.78, and 0.75 for symptomatic, endoscopic, and symptomatic/endoscopic responses, respectively. Lower histologic cut-points did not result in a substantial gain in response, but decreased the AUC. CONCLUSION: In this large cohort of EoE patients, rates of symptomatic and endoscopic improvement were generally associated with histologic improvement. A histologic cutoff for treatment response of <15 eos/hpf may balance clinical outcomes and test performance.