Tofacitinib in refractory non-infectious anterior uveitis: a retrospective observational study

Abstract Purpose To evaluate the efficacy, corticosteroid-sparing effect and short-term safety of tofacitinib in adults with non-infectious anterior uveitis refractory to conventional corticosteroid and immunosuppressive therapy. Methods Retrospective observational study of 29 consecutive adults (31 eyes) treated at two tertiary care centres in New Delhi between January 2022 and June 2024. Refractory disease was defined as failure to maintain remission on ≤ 7.5 mg/day of oral prednisolone after at least three months of combined corticosteroid and at least one conventional immunosuppressant. All patients received tofacitinib 5 mg twice daily and were followed for 12 months. The primary outcome was sustained clinical remission, defined as Standardization of Uveitis Nomenclature (SUN) grade 0 anterior chamber cells on two consecutive visits at least three months apart together with complete discontinuation of topical and systemic corticosteroids. Secondary outcomes were time to remission, best-corrected visual acuity (BCVA), relapse during follow-up, adverse events, and a comparison of responders with non-responders. Results Mean age was 34.6 ± 8.2 years and 16 of 29 patients (55.2%) were women. Ankylosing spondylitis was the commonest systemic association (9 patients, 31.0%). Remission was achieved in 26 of 29 patients (89.7%) at a mean of 4.5 ± 1.6 weeks. Among these 26 responders, 19 (73.1%) discontinued topical and oral corticosteroids by 4 weeks and the remaining 7 (26.9%) by 6 weeks. Remission was maintained without relapse through 12 months in 23 /26 responders (88.4%). Three patients who relapsed on tofacitinib were controlled by short course of topical steroids only. Mean BCVA improved from 0.74 to 0.37 logMAR ( p < 0.001). Corticosteroid (topical and oral) was withdrawn by 4 weeks in all 19 responders previously treated with methotrexate or azathioprine and by 6 weeks in the 7 previously treated with mycophenolate mofetil. The three non-responders (10.3%) had systemic lupus erythematosus, Behçet’s disease and inflammatory bowel disease-associated arthritis. No serious adverse events occurred; two patients (6.9%) developed transient transaminitis, two (6.9%) an uncomplicated urinary tract infection and one (3.4%) localised herpes zoster reactivation. Conclusions In this uncontrolled retrospective cohort, tofacitinib was associated with high rates of remission and rapid corticosteroid withdrawal in refractory non-infectious anterior uveitis, with no serious adverse events over 12 months. These findings are hypothesis-generating and require confirmation in controlled prospective studies before tofacitinib can be positioned relative to established biologic therapy.