CHICAGO — Continuous treatment with budesonide orodispensible tablets was associated with maintaining greater remission of eosinophilic esophagitis (EoE) at 48 weeks compared to intermittent use or placebo in a randomized controlled phase 3 study.

“EoE is a chronic immune-mediated disease of the esophagus. Left untreated, remodeling and strictures can occur, and it can cause food impaction,” Alain M. Schoepfer, head physician of gastroenterology and hepatology at Centre Hospitalier Universitaire Vaudois and associate professor of biology and medicine at the University of Lausanne in Lausanne, Switzerland, said here at Digestive Disease Week (DDW) 2026.

Although long-term treatment is thought to be necessary to prevent remodeling and strictures, nonadherence is common among patients with EoE at his institution, said Schoepfer, adding that the study was motivated by some of these patients.

“It’s mostly young males, who despite my brave efforts to educate them on the consequences of untreated EoE in the long term, try to outsmart me,” he said.

“In practical terms, they take the drug for a couple of weeks and they feel magically better. They stop it, and then when the symptoms return, they take [budesonide] again,” he explained. “It’s an on-off therapy regimen that was created by these patients.”

“They always say ‘I’m doing great with these minimum dosages,’” he added.

Schoepfer and colleagues performed the study to test if an intermittent dosing regimen might lead to better patient outcomes.

Methodology

The study included 112 patients aged 16-75 years with clinically and histologically confirmed EoE in remission. Schoepfer and colleagues defined EoE remission as a peak eosinophil count of < 16/mm² high-power field (hpf), an EoE Symptom Activity Index (EEsAI) score of ≤ 20, and dysphagia and odynophagia numerical rating scale (NRS, 0-10) score of < 2. 

The population was typical for EoE, Schoepfer said, about two thirds men, and the average age was about 40 years.

Study participants were randomized 2:2:1 to repeating cycles of 0.5 mg budesonide twice a day for 4 weeks followed by 8 weeks of placebo for a total of 48 weeks (intermittent group, n = 45), 0.5 mg budesonide continuously for 48 weeks, (continuous group, n = 45), or to placebo (n = 22).

The primary efficacy endpoint was the percentage of patients without any relapse over 48 weeks. Clinical relapse was defined as either a > 20-point increase in EEsAI, dysphagia or odynophagia NRS score of ≥ 4, indicating symptomatic relapse. Histologic relapse was defined as peak eosinophil levels of ≥ 48/mm² hpf.

The researchers also tracked EoE symptoms, including frequency of trouble swallowing, food impaction, duration of dysphagia, pain when swallowing, and more.

Key Findings

By week 48, 62% of patients in the continuous group did not experience relapse compared to 11% of patients in the intermittent group, and 9% of patients taking a placebo. There was a significant difference between the continuous and placebo groups (P < .0001), but not between the intermittent and placebo groups (P = .3998).

In terms of histologic relapse, patients on continuous treatment had a 2% relapse rate compared to 67% in the intermittent group and 82% in the placebo group.

Looked at another way, 88.9% of the continuous group experienced histologic remission compared to 24.4% of the intermittent group and 13.6% of placebo group.

The researchers also looked at eosinophil levels. The levels were stable throughout the study in the continuous group compared to an average increase of 233 eosinophils/mm² in the intermittent cohort and 305 eosinophils/mm² in the placebo group.

Regarding clinical remission measured with EEsAI scores, 82% of the continuous group, 44% of the intermittent group, and 32% of the placebo group met this metric.

Safety Profile

There were no new safety signals for budesonide orodispensible. Most discontinuations were related to aggravation of the patient’s EoE, Schoepfer reported. 

Oral and oropharyngeal candidiasis occurred in 20% in the continuous group, 6.7% in the intermittent group, and 4.5% in the placebo group, all of which were successfully treated, he said.

A meeting attendee questioned the 4 weeks on/8 weeks off regimen for the intermittent group, suggesting that shorter intervals might cause fewer relapses.

“In my clinical experience, about one quarter of all patients take [budesonide] for 3 or 4 weeks until they feel better, and then, when there is a very low symptom burden or no symptoms, they decide to let it go,” Schoepfer said.

Not Yet Approved in US

Although not approved for use in the US, the study underlines the importance of continuous treatment adherence, said meeting attendee Stuart Spechler, MD, chief of the Division of Gastroenterology and co-director of the Center for Esophageal Diseases at Baylor University Medical Center at Dallas. Budesonide orodispensible tablets, that disintegrate between the tongue and gum and stimulates saliva, are approved for use in Europe, the UK, Canada, and Australia.

“We don’t have [budesonide] here in this country; however, we are using steroids very frequently in these patients. Patients do stop them and start them on their own frequently, even though we tell them this is a chronic disease,” said Spechler, who was not involved in the study.

“The bottom line, again,” he said, “is you really should counsel your patients try to stay on therapy. It’s going to come back if you stop.”

The study was independently supported. Schoepfer disclosed being a member of the EEsAI study group and published on the EEsAI PRO instrument; being a consultant, speaker; and/or receiving research grants from AstraZeneca, Aptalis Pharma Inc., Dr. Falk Pharma, GlaxoSmithKline, Nestlé, and Receptos. Spechler had no relevant disclosures.

Damian McNamara is a freelance contributor to Medscape Medical News. He worked full-time for Medscape and WebMD from 2018 to 2024. McNamara has a BA in chemistry and an MA in science, health and environmental reporting/journalism.