Budesonide Oral Suspension Is a Promising Therapy for Young Children with Eosinophilic Esophagitis

The twice-daily novel formulation of the corticosteroid budesonide may change health outcomes for young people diagnosed with eosinophilic esophagitis (EoE). Results from the PEDEOS-1 study showed that budesonide oral suspension (BOS) improved clinical, histologic, and endoscopic features of EoE in children and adolescents who maintained the twice-daily therapy for 12 weeks or longer. Alfredo J. Lucendo, MD, a gastroenterologist at the Hospital General de Tomelloso, in Castilla-La Mancha, Spain, presented efficacy and safety data from the trial at the Digestive Disease Week 2024 meeting.

Topical corticosteroids have previously demonstrated efficacy as a first-line therapy for adults with EoE. Earlier this year, BOS became the first oral therapy to receive approval for the treatment of pediatric patients aged 11 years and older diagnosed with EoE in the United States. The approval came after two randomized, multicenter, placebo-controlled studies showed that a 12-week course of treatment was safe and effective in this population.

In Europe, the multisite PEDEOS-1 trial enrolled children and adolescents between the ages of 2 and 17 years with the aim to assess the efficacy and tolerability of treatment with two doses of BOS. After undergoing endoscopic examination and assessment of symptoms, children between the ages of 2 and 11 years were randomized to receive either a high dose of BOS (0.5 mg twice daily), a low dose of BOS (0.5 mg once daily), or placebo. Older children and adolescents between the ages of 12 and 17 years received 1 mg BOS twice daily, 1 mg BOS once daily, or placebo. All participants had active, symptomatic EoE and a peak eosinophil count of 20 or more eosinophils per high-power field (hpf), derived from six biopsy samples. The trial excluded patients with other causes of esophageal eosinophilia, active esophageal infections, esophageal surgery, esophageal dilation within 8 weeks of the screening visit, onset of dietary restrictions within 4 weeks before screening, or a high disease burden requiring immediate treatment.

Patients who did not respond to treatment by 8 weeks after initiation, or presented with bolus impaction at any moment, had the option to participate in an additional 12-week open-label induction period. Once remission was achieved, participants could enter a 24-week open-label maintenance period, followed by a period of tapering and follow-up. The primary endpoint included histologic remission (peak eosinophil count of <5 eos/hpf) at week 12 plus clinical response, defined as a 30% or greater drop in the initial symptom severity score from baseline to week 12.

The results showed that BOS outperformed placebo in inducing histologic remission and clinical response at week 12. The speaker noted that the higher doses of BOS produced better results in all age groups in terms of meaningful clinical improvement, mucosal healing, and endoscopic improvement of inflammation and fibrosis. “The reduction in eosinophil count from baseline was greater in patients treated with BOS, especially with the higher dose,” Lucendo said. “Histological healing [was observed] in most treated patients, but not in the placebo [arm]. There was a higher proportion of patients with a completely normal esophagus in the groups treated with the higher dose.”

There were no significant differences between the three groups in terms of symptomatic response and remission by week 12. However, the dysphagia severity scale showed a trend in favor of BOS. Treatment with the high dose showed some benefit over the low dose, and therapy with both doses outperformed placebo. The authors noted that the safety data were consistent with the known profile of topical budesonide. Participants experienced mild and moderate adverse events, with no reports of food bolus impaction.