Oral budesonide disappoints in first RCT for coeliac disease

Coeliac disease

By Mardi Chapman

1 Jul 2021

An Australian pilot study has failed to demonstrate a benefit on mucosal healing from oral budesonide, in addition to a gluten-free diet, in patients with newly diagnosed coeliac disease.

The RCT compared mucosal healing in biopsy specimens at 8 weeks and 52 weeks in 37 patients treated with either 9 mg/day budesonide for 8 weeks, tapering to 3 mg by week 10, or placebo.

The study, published in Alimentary Pharmacology & Therapeutics, found the proportion of patients achieving a mucosal response (Marsh 0 or 1) at 8 weeks was 37% with budesonide versus 28% with placebo (0=0.73).

Similarly, the response at 52 weeks was numerically but not statistically higher with budesonide (63% v 44%).

Remission (Marsh 0) was also higher but not statistically different with budesonide versus placebo at 8 weeks (32% v 17%) and at 52 weeks (42% v 33%).

There were no differences between the groups in other secondary outcomes measures including GI symptoms, fatigue, or anxiety/ depression.

“The study did, however, recognise that about one in four patients heal their duodenal mucosa within 8 weeks of the institution of a gluten-free diet and enabled identification that this was strongly and inversely associated with the histopathological severity of the duodenal lesion at diagnosis,” the study said.

It found the safety profile of oral effervescent budesonide was acceptable, with no significant differences between the groups in either treatment-related adverse events or post-treatment adverse events.

There was no detectable steroid effect on bone mineral density, blood glucose tolerance or fasting serum cortisol.

The investigators, including Professor Peter Gibson from Alfred Hospital and Monash University, said the size and power of the study and relatively mild patient phenotype may have been limitations of the study.

They also suggested it was possible that insufficient drug was delivered to the duodenum.

“Oral budesonide used in prior case studies of patients with refractory coeliac disease utilised budesonide that was protected from gastric acid by virtue of microcapsule coating in order to enhance delivery to the small intestine and colon.”

“In conclusion, this pilot study is the first of its kind to attempt to institute early and rapid remission in coeliac disease through the use of oral effervescent budesonide as a form of locally active immunosuppression,” they said.

“Although it showed that the intervention and design was both feasible and safe, it failed to provide evidence of a positive effect of budesonide on primary or secondary outcomes.”

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