An industry-funded randomized controlled trial (RCT) of 204 adult patients with eosinophilic esophagitis (EoE) compared the efficacy and safety of two dosages of a budesonide orodispersible tablet versus placebo in maintaining remission of the disorder. Participants were in clinical and histologic remission, were from 29 European study sites, and were randomly assigned to groups given 0.5 mg of the drug twice daily (n=68), 1.0 mg twice daily (n=68), or placebo twice daily (n=68) for up to 48 weeks. At end of treatment, 73.5% of patients receiving 0.5 mg twice daily and 75% of those receiving 1.0 mg twice daily were in persistent remission compared with 4.4% of patients in the placebo group (P<0.001 for both comparisons).
The study was published online on July 25, 2020, by Gastroenterology. The following commentary by ACP Resident/Fellow Member Sachin Srinivasan, MD, and Prateek Sharma, MD, FACP, appeared in the ACP Journal Club section of the April Annals of Internal Medicine.
A recent guideline on EoE recommends topical steroids as first-line treatment in addition to proton-pump inhibitors, elimination diets, and esophageal dilation. In a meta-analysis of 8 RCTs (n=437), not achieving remission was less common at 8 weeks with topical steroids vs. placebo (relative risk, 0.39 [95% CI, 0.26 to 0.58]). An orodispersible formulation of budesonide uses saliva for drug delivery, increasing exposure of the esophageal surface to the drug. An earlier RCT of this formulation showed remission in 58% of treated patients at 6 weeks and 85% at 12 weeks.
In a multicenter RCT, Straumann and colleagues showed that both doses of budesonide increased maintenance of remission compared with placebo over 48 weeks. Up to 10% of patients in the budesonide groups had relapse vs. 60% in the placebo group. Patients with a longer duration of disease (>9 y) or more extensive esophageal involvement did better with the higher dose. The main adverse event was local candidiasis.
The trial shows that both doses of budesonide are safe and effective in maintaining remission and preventing relapse for up to 48 weeks. The 0.5-mg dose may be enough to maintain remission, but patients with long-standing symptoms or extensive disease might benefit from a 1-mg dose. This needs further evaluation. Longer-term safety, dose tapering, and steroid-sparing studies akin to the inflammatory bowel disease literature are needed to determine induction and maintenance regimens for guidelines.