Systematic review of tofacitinib: a new drug for the management of rheumatoid arthritis.

PURPOSE: The goal of this study was to review and summarize the efficacy and safety of use of tofacitinib for treating rheumatoid arthritis (RA).

METHODS: A systematic literature review was conducted to identify English-language articles published through May 2013 within PubMed, ClinicalTrials.gov, and Cochrane Library reporting results from Phase II and Phase III tofacitinib randomized clinical trials. Tofacitinib must have been used as monotherapy or in combination therapy with disease-modifying antirheumatic drugs (DMARDs) in the treatment of RA. Study outcomes had to include at least 1 of the following: American College of Rheumatology (ACR) 20%, 50%, or 70% response rates; tender/swollen joint count; health assessment questionnaire of disability; radiographic outcomes; and drug persistence.

FINDINGS: Eight studies (4 Phase II and 4 Phase III trials) were included in the review. Patients with active RA and who were nonresponders to a biologic agent or the nonbiologic DMARD methotrexate were included in these studies. The results of the Phase II trials show that tofacitinib at doses ≥3 mg BID was efficacious among the nonresponders. The results of the Phase III trials, comparing tofacitinib 5 and 10 mg with placebo, show that tofacitinib led to a significant improvement in ACR20 response (P < 0.0001), Health Assessment Questionnaire-Disability Index (P < 0.0001) scores, and ACR50 response (P < 0.0001) after 3 months. The efficacy of tofacitinib was numerically similar to adalimumab. The most common adverse events were infections, infestations, increases in LDL-C and HDL-C levels, and a decrease in neutrophil counts.

IMPLICATIONS: Tofacitinib is an efficacious drug for the management of moderate to severe RA among patients with an inadequate response to methotrexate and tumor necrosis factor inhibitors. Long-term studies can help in understanding the risk/benefit profile of tofacitinib.