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World J Orthop. Sep 18, 2014; 5(4): 504-511
Published online Sep 18, 2014. doi: 10.5312/wjo.v5.i4.504
Efficacy and safety of tofacitinib for treatment of rheumatoid arthritis
Lisa M Lundquist, Sabrina W Cole, Martha L Sikes
Lisa M Lundquist, College of Health Professions, Mercer University, Atlanta, GA 30341, United States
Sabrina W Cole, School of Pharmacy, Wingate University, Wingate, NC 28174, United States
Martha L Sikes, College of Health Professions, Mercer University, Atlanta, GA 30341, United States
Author contributions: Lundquist LM, Cole SW and Sikes ML wrote and edited the manuscript.
Correspondence to: Lisa M Lundquist, PharmD, BCPS, College of Health Professions, Mercer University, 3001 Mercer University Drive, Atlanta, GA 30341, United States. lundquist_lm@mercer.edu
Telephone: +1-678-5476208 Fax: +1-678-5476231
Received: December 29, 2013
Revised: March 26, 2014
Accepted: May 31, 2014
Published online: September 18, 2014
Processing time: 234 Days and 10.2 Hours
Abstract

Tofacitinib is the first in a new class of nonbiologic disease-modifying antirheumatic drugs (DMARDs), a targeted, synthetic DMARD, approved for the treatment of rheumatoid arthritis (RA) as monotherapy or in combination with methotrexate or other non-biologic DMARD. Tofacitinib, an orally administered Janus kinase (JAK) inhibitor, decreases T-cell activation, pro-inflammatory cytokine production, and cytokine signaling by inhibiting binding of type I cytokine receptors family and γ-chain cytokines to paired JAK1/JAK3 receptors. The net effect of tofacitinb’s mechanism of action is decreased synovial inflammation and structural joint damage in RA patients. To date, six phase 3 trials have been conducted to evaluate the safety and efficacy of tofacitinib under the oral rheumatoid arthritis triaLs (ORAL) series. This review describes the pharmacology of the novel agent, tofacitinib, and details the safety and efficacy data of the ORAL trials.

Keywords: Tofacitinib; Rheumatoid arthritis; Janus kinase inhibitor

Core tip: Tofacitinib, a Janus kinase inhibitor, is a targeted, synthetic, disease-modifying antirheumatic drug (DMARD) approved for the treatment of moderately to severely active rheumatoid arthritis in patients who have had an inadequate response to methotrexate. In numerous phase 2 and 3 trials, tofacitinib has proven to be safe and effective as monotherapy or in combination with methotrexate or other non-biologic DMARDs.