The Latest Progress in the Application of Tofacitinib in Rheumatic Diseases——A Narrative Review

Tofacitinib is an oral Janus kinase (JAK) inhibitor that modulates cytokine-dependent JAK/STAT signaling and has been extensively investigated in rheumatic diseases. This narrative review provides an updated synthesis of the pharmacological characteristics, mechanisms of action, clinical efficacy, safety, drug–drug interactions and dosing considerations of tofacitinib in special populations. The relevant literature published from January 2013 through December 2025 was identified through searches of MEDLINE, Embase, the Cochrane Library, and Google Scholar, supplemented by manual screening of reference lists. After screening and eligibility assessment, 89 clinical publications were included in the disease-specific evidence synthesis, and the strength of clinical evidence across disease indications was evaluated qualitatively. The most robust evidence is available for rheumatoid arthritis, with randomized trials, long-term extension studies, and real-world investigations demonstrating improvements in disease activity and physical function and inhibition of radiographic progression. Clinical evidence also supports therapeutic benefit in psoriatic arthritis, ankylosing spondylitis, and juvenile idiopathic arthritis, whereas evidence for Takayasu arteritis, polymyalgia rheumatica, systemic lupus erythematosus, and anti-MDA5-positive dermatomyositis-associated interstitial lung disease remains limited or exploratory. Long-term integrated safety analyses in RA have reported incidence rates of serious adverse events of approximately 9.0– 9.4 per 100 patient-years and serious infections of approximately 2.5– 2.7 per 100 patient-years, while herpes zoster, major adverse cardiovascular events, venous thromboembolism, and malignancy remain clinically important safety considerations. Potential drug–drug interactions and dose adjustment in patients with renal or hepatic impairment and other special populations further require individualized assessment. Overall, tofacitinib has a well-established role in RA treatment and potential therapeutic value in several other rheumatic diseases; however, its use should be guided by disease-specific evidence, previous treatment exposure, comorbidities, concomitant medications, and individual safety risks. Further prospective and long-term studies are needed to better define its role in less well-established indications and special populations.