A nationwide experience of biological treatments in children with eosinophilic esophagitis

Abstract

Background: No biological treatment has been approved for pediatric eosinophilic esophagitis (EoE) in France. For patients refractory to conventional treatments, although compassionate use of monoclonal antibodies has developed, experience remains limited.

Methods: We conducted a national multicenter study across French pediatric tertiary care centers where children (younger than 18 years) who presented with EoE were treated with biological therapies between January 2015 and December 2023. The main objective was to characterize this patient population, and the indications for prescribing biologics. The secondary goals were to assess these patients' clinical, endoscopic, and histologic finding, as well as patient tolerance, and to compare our cohort at baseline with pediatric patients from two European registers of EoE treated with conventional therapies.

Results: Thirty-six patients were prescribed 37 biologics (omalizumab, n = 1; mepolizumab, n = 6; dupilumab, n = 30). At diagnosis, the mean patient age was 7.4 (±4.4) years, and most patients had at least one atopic comorbidity (91.7%, n = 33). Failure of first-line treatments was the main reason for starting biological therapy (75.7%, n = 28), prescribed as compassionate use (54.1%, n = 20). Dupilumab showed significant clinical (48%, p < 0.01) and histological (82.6%, p < 0.01) improvement. Compared with children treated with conventional therapies, patients in our cohort at baseline presented significantly more asthma, food allergies, and atopic dermatitis, as well as more fibrostenotic phenotype and digestive symptoms. No severe side effect was reported within a 6-12-month follow-up.

Conclusion: Dupilumab is the most frequently prescribed, and appears to be the most effective biotherapy, regarding clinical and histologic remission. All biologics were well-tolerated.

Impact: Pending marketing authorization, biological therapies for pediatric eosinophilic esophagitis are mainly prescribed after failure of first-line treatments and on a compassionate basis in France. Dupilumab is the biotherapy most frequently used, is associated with clinical and histological efficacy and is well-tolerated. Children and adolescents requiring biologics appear to be younger and more severe at diagnosis than naive pediatric patients in Europeans registries.