Therapy with Budesonide in Patients with Refractory Sprue

INTRODUCTION: Refractory sprue (RS) is a rare malabsorption syndrome, which often requires long-term corticosteroid treatment. Locally acting budesonide could replace systemic corticosteroid therapy and reduce toxicity in patients with RS. AIMS: To evaluate the efficacy and toxicity of budesonide in patients with RS. PATIENTS AND METHODS: Clinical and histological data from patients with RS who received budesonide were analyzed. RS was defined as villous atrophy and malabsorption in spite of a strict gluten-free diet persisting for >6 months or requiring earlier therapeutic intervention. RESULTS: We identified 9 patients (1 with autoimmune enteropathy, 4 with RS type I without and 3 with RS type II with signs of early T cell lymphoma and 1 with CD4-positive sprue-like intestinal T cell lymphoma), who received 9 mg/day of budesonide (range 6-12) for 24 months (1-60), and 7 of whom had an initial treatment with 40 mg/day of prednisolone (30-60) for 4 months (1-144). The initial body mass index was 18 (13.1-22.8) and increased similarly under prednisolone [21.5 (14.9-26.7), p < 0.05] and budesonide therapy [21 (18-27.2), p < 0.05]. The stool frequency per day also decreased similarly from 6 (2-8) to 2 (1-3) and 2 (1-5), each p < 0.05, under prednisolone and budesonide therapy, respectively. Two patients with RS type II did not respond and 7, including all 4 with RS type I, were clinically stable with budesonide therapy. Skin fragility in 1 patient was the only adverse effect of budesonide therapy. CONCLUSIONS: Budesonide may be an effective treatment option in patients with RS type I, which can stabilize the clinical condition similar to prednisolone.