Therapeutic effect of tofacitinib combined with leflunomide for refractory Takayasu arteritis: pilot study

Takayasu arteritis (TAK) is a refractory disease with high incidence of disease relapse and glucocorticoids dependence. The objective of this study is to evaluate the effect and safety of tofacitinib (TOF) combined with leflunomide (LEF) treatment regimen in patients with refractory TAK, thereby providing a potential new therapeutic option for managing this condition.

Twenty-four patients with refractory TAK treated with tofacitinib and leflunomide between May 2020 to August 2024 were enrolled retrospectively from the East China Takayasu Arteritis (ECTA) cohort established by the Department of Rheumatology of Zhongshan Hospital. Most (91.7%) patients received glucocorticoids (GCs) simultaneously. Remission rate, tapering of GCs, reduction in inflammatory parameters, improvement in vascular imaging, and adverse events were analyzed in a 12-month follow-up period.

Remission rate upon combined treatment was 87.5% (95CI: 69.0%-95.7%) at 6 months and 79.2% (95CI: 59.5%-90.8%) at 12 months respectively. Relapse rate was 8.3% (95CI: 2.3%-25.8%). GCs were successfully tapered from 15.2±12.9 mg/day at baseline to 7.8±4.5 mg/d at 6 months (p = 0.02) and 6.5±4.5 mg/day at 12 months (p = 0.001). The decrease in the erythrocyte sedimentation rate (ESR) was also significant (36.3±33.8 mm/h at baseline vs. 17.1±17.6 mm/h at 12 months, p = 0.02). Stability or improvements in imaging were observed in 5/7 (71.4%) patients who received magnetic resonance angiography (MRA) follow-up. The regimen was well-tolerated with only few and mild adverse events observed.

The regimen of TOF combined with LEF might be a promising novel option in treating refractory TAK patients.