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Area of Science:

  • Gastroenterology
  • Immunology
  • Oncology

Background:

  • Refractory celiac disease (RCD) is a rare gastrointestinal disorder with a poor prognosis.
  • Treatment options for RCD are limited, and immunosuppressive medications (IMs) have shown modest success.
  • Open-capsule budesonide (OB) has emerged as a potential therapeutic agent for RCD.

Purpose of the Study:

  • To evaluate the efficacy and outcomes of open-capsule budesonide (OB) in patients with refractory celiac disease (RCD).
  • To assess the response to OB in RCD patients who have failed prior immunosuppressive medication (IM) treatment.

Main Methods:

  • Retrospective analysis of 57 RCD patients treated with OB at Mayo Clinic from 2003 to 2015.
  • Classification of RCD based on T-cell receptor gamma gene rearrangement or intraepithelial lymphocytes (IELs) phenotype.
  • Analysis of demographic, serologic, clinical, and histopathologic variables before and after OB therapy.

Main Results:

  • The majority of patients (69%) were women, with a mean age of 60.5 years.
  • Nearly half of the patients had previously failed IM treatment.
  • Open-capsule budesonide (OB) therapy led to clinical improvement in 92% and histologic improvement in 89% of RCD patients.
  • Absence of clonal T-cell receptor gamma gene rearrangement/aberrant IEL phenotype was observed in 53% of RCD-2 patients post-treatment.

Conclusions:

  • Open-capsule budesonide (OB) therapy is effective in improving clinical and histopathologic outcomes in most patients with refractory celiac disease (RCD).
  • OB is a viable therapeutic option for RCD management, particularly for patients who have not responded to immunosuppressive medications (IMs).
  • Further research is warranted to fully elucidate the long-term benefits and safety profile of OB in RCD.