Budesonide orodispersible tablets (BOTs) effectively maintained remission in patients with eosinophilic esophagitis (EoE), according to study data published by researchers in Gastroenterology.
Although topical-acting corticosteroids such as BOT have been shown to bring active EoE into remission, their long-term efficacy remains unclear. This phase-3, randomized, double-blind, placebo-controlled maintenance trial sought to assess the efficacy and safety of BOT for long-term maintenance therapy in patients with EoE. Investigators recruited patients aged 18 to 75 years with proton pump inhibitor-refractory EoE in clinical and histological remission from 29 clinical sites in Europe between 2016 and 2018. All patients had achieved remission after treatment with BOT 1 mg twice daily for ≥6 weeks as part of an affiliated clinical trial. Researchers randomly assigned 204 patients with remitted EoE in a 1:1:1 ratio to BOT 0.5 mg twice daily (n=68), BOT 1 mg twice daily (n=68), or placebo twice daily (n=68) for ≤48 weeks. The primary outcome was maintenance of clinical and histological remission at week 48. Adverse events were monitored throughout the trial duration.
Treatment groups had comparable demographic and clinical characteristics at baseline. Mean age in the pooled cohort was 36±10.6 years; 169 (82.8%) participants were men; and all participants (100%) were white. Patients in the BOT 0.5 mg, BOT 1 mg, and placebo groups maintained remission at week 48 by 73.5%, 75%, and 4.4%, respectively (P <.001 for both BOT vs placebo comparisons). Median time to relapse in the placebo group was 87 days compared with >350 and >354 days in the BOT 1 mg and BOT 0.5 mg groups, respectively (Ptrend <.001). Remission rates in the BOT groups were generally similar across patient subgroups defined by comorbid conditions and disease characteristics; however, among patients with extended inflammation and patients with extended disease history (≥9 years), remission rates were numerically higher with BOT 1 mg vs BOT 0.5 mg.
Adverse events occurred with similar frequency in the BOT and placebo groups. Serum cortisol levels did not significantly change in any treatment group, although 4 participants receiving BOT developed asymptomatic low serum cortisol levels. Investigators observed suspected symptomatic candidiasis in 16.2% and 11.8% of patients in the BOT 0.5 mg and BOT 1.0 mg groups, respectively, although all infections resolved with treatment.
Results from this trial suggest that BOT may be an appropriate means to maintain remission in adult patients with EoE. Further study is necessary to identify the minimally effective dose of BOT, as the 0.5-mg and 1-mg groups had comparable remission rates.
Limitations include the fact that researchers did not identify a minimally effective
dose regimen to maintain EoE in remission. Additionally, they allowed concomitant treatment with PPIs, a factor that may have played a role in the lack of evident symptoms at EoT in patients receiving placebo. Finally, measurements of squamous epithelium thickness at baseline and after long-term exposure to STCs are necessary.
“[T]his trial confirmed that EoE requires a therapeutic long-term management and demonstrated that BOT is effective and safe as maintenance therapy for adult patients with EoE who achieved disease remission with the same compound,” investigators concluded.
Disclosure: Several study authors declared affiliations with the pharmaceutical industry.
Please see the original reference for a full list of authors’ disclosures.
Straumann A, Lucendo AJ, Miehlke S, et al. Budesonide orodispersible tablets maintain remission in a randomized, placebo-controlled trial of patients with eosinophilic esophagitis [published online July 25, 2020]. Gastroenterology. doi: 10.1053/j.gastro.2020.07.039