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Background One third of patients with eosinophilic esophagitis (EoE) do not achieve histological remission with standard medical or dietary treatment. The outcome of these patients undergoing various rescue treatments is not known and whether these patients constitute a distinct subset remains unclear. Objective To analyze EoE treatment outcomes in a predominantly pediatric population, including after initial treatment failure (rescue treatment) for differences in outcomes and clinical presentation. Methods We identified 100 serial cases of confirmed EoE from our REDCap® database established at Massachusetts General Hospital starting from January 2007. Demographic data, clinical symptoms, treatment regimens, endoscopic findings, skin testing results, food triggers and clinical outcome of various rescue treatment strategies were presented. We defined clinical response as histological remission with peak eosinophil count of at least 6 biopsies less than 10 per high power field. RESULTS Ninety-seven EoE patients underwent initial treatments. Eighty-one elected dietary treatment (7 elemental diet, 54 multiple food elimination diet, and 20 milk-free diet and 16 elected medical treatment (15 swallowed fluticasone and 1 budesonide). Initial response rate to dietary and medical treatment was 67% (54/81) and 56% (9/16) respectively. Of the 34 who failed initial treatment, 24 of them elected various second treatment regimens (3 medical therapy, 2 milk-free diet, 14 multiple food elimination diet and 5 elemental diet) and 54% (13/24) achieved histological remission. Eight of the remaining 11 who failed second treatment underwent additional treatments and 2 ultimately achieved histological remission. The overall response rate by intention-to-treat analysis increased from 65% (63/97) with initial treatment to 78% (76/97) with rescue treatment, and further to 80% (78/97) with multiple rescue treatments. On a per-protocol basis, the overall response rate was 93% (78/84); however, patients who failed the first two rounds of therapy had only a 20% response rate. Patients who responded to initial treatment were found to have more symptoms and endoscopic abnormalities. In contrast, comparison of patients who failed both initial and rescue therapy to those who responded to rescue therapy did not identify any differentiating clinical features. CONCLUSION More than half of the patients who failed initial EoE treatment could still achieve histological remission with rescue treatments. Elemental diet is the most effective initial and rescue therapy in achieving histological remission. No clinical features could not identified to reliably predict response to rescue treatment.

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