Open-capsule budesonide improves refractory celiac disease

Last Updated: 2017-04-10

By Rita Buckley

NEW YORK (Reuters Health) - Open-capsule budesonide appears to be a safe and effective treatment for most patients with refractory celiac disease, according to new research.

“The study describes a novel and safe method for treatment of this severe illness, with the potential to avoid toxic therapy and the future development of malignancy,” senior author Dr. Joseph A. Murray from the Mayo Clinic in Rochester, Minnesota, told Reuters Health.

Dr. Murray and colleagues identified 57 adult patients with refractory celiac disease who had been treated from 2003 to 2015 with open-capsule budesonide or a Mayo-compounded capsule designed for release in the proximal gastrointestinal tract.

Each patient took a 3-mg capsule three times a day. All had persistent or relapsing symptoms and intestinal damage after strict adherence to a gluten-free diet for at least six months; nearly half had treatment failure with immunosuppressive drugs.

After treatment with open-capsule budesonide, the majority of patients showed clinical (92%) and histological (89%) improvement.

Clinical response was complete in 71% of patients and partial in 22%. Histological response was complete in 63% of patients and partial in 26%, with higher rates in those with type 1 refractory celiac disease.

Patients who had previous failure with immunosuppressive agents also had high clinical (93%) and histological (91%) response rates.

Follow-up biopsy in seven of 13 patients with type 2 refractory celiac disease showed an absence of previously seen clonal T-cell receptor gamma gene rearrangement/aberrant intra-epithelial lymphocyte phenotype.

Open-capsule budesonide was well tolerated, with few adverse effects leading to discontinuation of treatment.

Dr. Donald F. Kirby, director of the Center for Human Nutrition at the Cleveland Clinic in Cleveland, Ohio, who was not involved in the research, said it was “very important.”

“The observation of more than a 90% clinical or histological improvement in both refractory celiac disease type 1, and surprisingly, type 2, should not be ignored,” he told Reuters Health.

He added that delivery of the medication to the duodenal bulb and beyond is unique. “This is where 97% of celiac disease begins,” he said.

Dr. Siddhartha Parker from the department of gastroenterology and hepatology at Dartmouth-Hitchcock Medical Center in Lebanon, New Hampshire, said, “Refractory celiac disease is not very common, but its symptoms can be quite severe and it is associated with increased risk of life-threatening T-cell lymphoma of the small intestine.”

“The medication is readily available and quite safe,” he told Reuters Health. “It appears to be a very reasonable treatment option for those with refractory celiac disease.”

Dr. Murray said that the outcomes have the potential to change patient management, with open budesonide considered an initial alternative to systemic steroids.

But he cautioned that the study is limited by its retrospective nature, open-label design and lack of a comparison group. He noted that there is a need for robust testing of open-capsule budesonide with or without failure of conventional immune suppression therapy in patients with refractory celiac disease.

Dr. Kirby also agreed that many clinicians may want to use open budesonide as first-line therapy because it is likely to be less toxic than other commonly used agents.

SOURCE: http://bit.ly/2nTToDf

Am J Gastroenterol 2017.