Vedolizumab Treatment in Adults with Inflammatory Gastrointestinal Disorders and Underlying Immunodeficiency

Noninfectious gut inflammation is a common manifestation of inborn errors of immunity (IEI) and can affect various segments of the gastrointestinal tract. Vedolizumab, which selectively blocks leukocyte trafficking to the gut, is approved for treatment of inflammatory bowel disease; however, there is lack of data regarding safety and efficacy in IEI. We aim to describe the experience of patients with gut inflammation related to IEI treated with vedolizumab.

4 patients >18 years old with humoral or combined immunodeficiency and gut inflammation treated with vedolizumab (weeks 0, 2, and 6, and then every 8 weeks) at a tertiary center. Clinical improvement was defined as a reduction in diarrhea reported by patients with weight gain and/or improvement of malabsorption.

Case 1: 37-year-old man, combined immunodeficiency with dysregulation due to TRAF3 haploinsufficiency. He had a long-standing enteropathy with duodenal, ileal, and colonic involvement and was corticosteroids dependent. He started vedolizumab, achieving good symptomatic control. After one year of treatment, mild endoscopic activity was observed, so he added a low dose of corticosteroids, resulting in clinical improvement.

23-year-old man, combined immunodeficiency with dysregulation, WES negative. He presented chronic diarrhea, weight loss, and malabsorption requiring hospitalizations in context of complete villous atrophy with poor response to corticosteroids. A clear clinical response was observed after induction with 3 doses of vedolizumab.

49-year-old woman, CVID with autoimmune hepatitis and chronic severe colitis refractory to topical and oral corticosteroids and azathioprine. She received four doses of vedolizumab without clinical improvement, eventually developing extensive pancolitis due to CMV. She is being considered for a subsequent treatment line with ustekinumab.

51-year-old woman, persistent agammaglobulinemia after rituximab treatment for immune thrombocytopenia and multiple other autoimmune manifestations, WES pending. Chronic diarrhea with complete villous atrophy refractory to corticosteroids. After 5 doses of vedolizumab, she exhibited clinical and endoscopic improvement and remained free of atrophy for two years. After this period, partial villous atrophy recurred.

We observed a more favorable and sustained effect of vedolizumab on duodenal atrophy compared with colitis, emerging as a safe option for patients with immunodeficiency and enteropathy.