A pilot study of tofacitinib for refractory Behçet’s syndrome

Behcet’s syndrome (BS) is a chronic inflammatory disease characterised by recurrent oral and genital ulcers, frequently complicated with uveitis gastrointestinal, neurological, major vessel and cardiac involvement. For severe and (or) refractory BS, tumour necrosis factor alpha (TNF-α) inhibitors are indicated beyond glucocorticoids (GCs) and immunosuppressants,1 which are still responded inadequately in a small portion of refractory patients. Tofacitinib, a JAK1/3 inhibitor targeting T cell signalling, was approved for autoinflammatory diseases including rheumatoid arthritis, psoriatic arthritis and ulcerative colitis (UC).2 Given T cells are implicated in BS,3 tofacitinib could be a promising approach for BS. In this study, we explored the efficacy and safety of tofacitinib in refractory BS. We enrolled seven male and six female patients with BS fulfilling the 2013 International Criteria for BS,4 presented with active vascular/cardiac (n=5), gastrointestinal (n=6) and articular (n=2) involvements between May 2018 and January 2020 (table 1), with a mean age of 42.2±15.5 years and a median disease duration of 102 (IQR 72–240) months. At enrolment, all patients had active disease (Behcet's Disease Current …