Baseline characteristics, treatment patterns, and long-term outcomes in patients with eosinophilic esophagitis initiating dupilumab in routine clinical practice: study protocol of a phase IV, prospective, observational, multicenter registry (EDESIA).

BACKGROUND

Dupilumab, a fully human monoclonal antibody that blocks interleukin-4 and -13 signaling, is approved for the treatment of eosinophilic esophagitis (EoE) based on phase III clinical trials. However, real-world data on dupilumab use for EoE are lacking.

OBJECTIVES

To assess the characteristics, treatment patterns, and outcomes among patients with EoE aged 12 years or older who receive treatment with dupilumab for EoE as prescribed under the US prescribing information.

DESIGN

This study protocol describes a phase IV, prospective, observational, multicenter patient registry, EDESIA, which will enroll approximately 300 patients aged 12 years or older who initiate dupilumab treatment as part of routine care across approximately 50 sites in the USA.

METHODS AND ANALYSIS: Baseline data collection will include demographics, disease characteristics, medical history, and prior/concomitant therapies. Data will be collected at baseline (day 1 of treatment) and at follow-up visits through 36 months. Key primary outcomes assessed will include details of food elimination diet, history of food impaction and esophageal dilation, histologic and endoscopic findings, and patient-reported outcomes. Adverse events will be monitored throughout the study.

ETHICS: The EDESIA registry will be conducted in accordance with the Declaration of Helsinki, the International Council for Harmonization guidelines for Good Clinical Practice, and applicable regulatory requirements. The local institutional review board at each study center will approve the study. Written informed consent will be obtained from all patients and/or a parent/legal guardian.

DISCUSSION: EDESIA will address the safety and tolerability of long-term weekly dupilumab treatment in patients with EoE, and inform future treatment guidelines.

TRIAL REGISTRATION: A US registry of EoE adolescent and adult patients treated with DUPIXENT® as standard of care (EDESIA), NCT06693531 (https://www.clinicaltrials.gov/study/NCT06693531).