Vedolizumab treatment in adults with immunodeficiency-associated enteropathy: a case series.

Background: Inflammatory gastrointestinal disease is a common manifestation of inborn errors of immunity (IEI) and may affect any segment of the gastrointestinal tract. Vedolizumab, a gut-selective anti-integrin approved for inflammatory bowel disease, has been scarcely studied in IEI. We describe our experience with vedolizumab in adults with immunodeficiency-associated enteropathy. Methods: We conducted a retrospective case series of adults with humoral or combined immunodeficiency and inflammatory gastrointestinal disease treated with vedolizumab at a single tertiary referral center. Clinical, endoscopic, laboratory and histopathological responses were assessed. Results: Four adult patients with immunodeficiency-associated enteropathy received vedolizumab. Two patients achieved sustained clinical and endoscopic/histological remission, both of whom had predominant villous atrophy (one with TRAF3 haploinsufficiency, and the other had multiple autoimmune manifestations, refractory immune thrombocytopenia and agammaglobulinemia following rituximab). Another patient with combined immunodeficiency and protein-losing enteropathy had marked clinical improvement with mild histologic response, but relapsed at two years in the setting of norovirus and Clostridium difficile infection. In contrast, a woman with common variable immunodeficiency, autoimmune hepatitis, and severe refractory pancolitis failed to respond to vedolizumab and subsequently developed CMV superinfection, requiring treatment escalation. Conclusions: In this case series, vedolizumab was associated with clinical improvement predominantly in patients with villous atrophy rather than refractory colitis. These findings are hypothesis-generating and support further evaluation of vedolizumab as a therapeutic option for selected patients with immunodeficiency-associated enteropathy.