Effectiveness and safety of leflunomide compared with cyclophosphamide as induction therapy in Takayasu's arteritis: an observational study.

AIMS

The objective of this study was to investigate the outcomes of leflunomide (LEF) compared with those of cyclophosphamide (CYC) as induction against active Takayasu's arteritis (TA) in Chinese patients.

METHODS

This was an observational study based on a prospective cohort that included TA patients diagnosed in large third-level first-class general hospitals in East China from January 2009 to September 2018. LEF- or CYC-induced active patients were enrolled for comparative effectiveness analysis. One-to-more paired cohorts of LEF CYC were derived by propensity-score matching (PSM). The primary outcome was complete remission (CR) at 9-month follow up, and secondary endpoints included partial remission (PR) and effectiveness rate (ER). Multivariable logistic regression was used to identify statistical significance.

RESULTS

A total of 131 enrolled patients with at least 3-months treatment included 53 receiving a regimen of glucocorticoid (GC) and LEF and 78 receiving GC and CYC. Compared with the CYC group, the LEF group showed higher CR rate {LEF CYC: 84.6% [95% confidence interval (CI) 74.5-94.8%] 59.0% (47.8-70.1%); relative risk (RR) = 0.3 (0.1-0.6),  = 0.002} and lower daily GC dose [10.0 (5.0-12.5) 12.5 (10.0-15.0) mg,  = 0.043] at the end of the 9-month induction. In the matched analysis, the LEF group ( = 23) still indicated a higher CR rate than the CYC group ( = 54) after PSM [RR = 0.1 (0.0-0.6),  = 0.003]. Four LEF-treated patients had mild side effects, and one died unrelated to LEF.

CONCLUSION

LEF could be an alternative induction therapy against TA, showing good effectiveness and tolerance compared with CYC.