Efficacy and safety of TNF-α inhibitors in refractory primary complex aphthosis: a patient series and overview of the literature

Abstract

Background: Otherwise healthy patients with severe recurrent mucocutaneous aphthous ulcerations (complex aphthosis) may require systemic immunomodulatory therapy. However, a subset of patients remain resistant or intolerant to recommended therapeutic agents. Recently, case reports have described that tumor necrosis factor-α (TNF-α) inhibitors may induce remission in these patients. Methods: Data on efficacy and safety of various TNF-α inhibitors used as monotherapy in a case series of 18 patients with refractory primary complex aphthosis are presented. Results: A total of 16 patients (89%) obtained complete or almost clearance of orogenital aphthous ulcerations rapidly after onset of therapy either with etanercept, adalimumab, infliximab or golimumab. Duration of treatment ranged between 3 and 77 months. Nine patients (50%) received more than one TNF-α inhibitor during the course of treatment. Five (28%) patients experienced side effects that could be related to treatment with TNF-α inhibitors. Conclusion: TNF-α inhibitors are an effective and safe treatment option for patients with severe complex aphthosis who do not respond sufficiently to standard therapy as recommended by existing guidelines. However, the final position of TNF-α inhibitors in the therapeutic armamentarium awaits randomized controlled trials.