Celiac.com 05/24/2017 - Refractory celiac disease (RCD) is a rare manifestation of celiac disease that is difficult to treat, and often results in death from enteropathy-associated T-cell lymphoma.
Doctors looking to treat RCD have found very limited success with a number of immunosuppressive medications (IMs), including azathioprine, systemic corticosteroids, or regular budesonide. A team of researchers at the Mayo Clinic recently set out to assess open-capsule budesonide (OB) treatment on RCD patients, including those who saw no improvement with previous IM treatments. The research team included Saurabh S Mukewar, Ayush Sharma, Alberto Rubio-Tapia, Tsung-Teh Wu, Bana Jabri and Joseph A Murray.
Based on clonal T-cell receptor gamma gene rearrangement or aberrant phenotype of intraepithelial lymphocytes (IELs), the team classified 13 patients (23%) as having RCD-2 and 43 (75%) as RCD-1.
The team was unable to determine TCR gene rearrangement status for one patient (2%). Most patients were women (69%), with an average age of 60.5 (+/- 3.5) years, while average body mass index was 28.4 kg/m2.
Nearly 75% of patients suffered from diarrhea, with an average of 6 bowel movements per day (range, 4–25). Nearly half of these patients failed to improve with IM treatment. Twenty-four patients (42%) were anemic, while 12 patients (21%) had hypoalbuminemia. Biopsies showed Marsh 3 lesions in all patients, broken down as follows: 19% were Marsh 3a, 46% were Marsh 3b, and 35% were Marsh 3c.
After OB therapy, 92% showed clinical improvement, while 89% showed histologic improvement. Subsequent biopsies showed that 7 out of 13 patients with RCD-2 (53%) displayed an absence of the previously observed clonal TCR gamma gene rearrangement/aberrant IEL phenotype. During the follow-up period, two patients died of enteropathy-associated T-cell lymphoma.
Most RCD patients show clinical and histopathologic improvement with OB treatment, including those who previously failed to respond to other IMs.
These results show that treatment with open-capsule budesonide is a promising option for patients looking to manage RCD.