Efficacy and safety of tofacitinib in patients with rheumatoid arthritis

In recent years, there has been significant progress in the management of rheumatoid arthritis (RA) with the emergence of achieving remission or low disease activity (LDA) as a treatment goal. Now, the findings of a post hoc phase II/III analysis, published in the Arthritis Research and Therapy indicated that tofacitinib provides improved efficacy and clinical benefit in patients with RA, especially in those with fewer prior treatments.

Tesser and colleagues conducted the post-hoc analysis to assess the efficacy and safety of tofacitinib in three distinct groups of RA patients based on their prior therapy response. These groups included those with an inadequate response to non-MTX csDMARDs, those with an inadequate response to MTX, and those with an inadequate response to bDMARDs. The researchers analyzed pooled phase II/III trial data from patients who received tofacitinib or placebo, either as monotherapy or in combination with conventional synthetic (cs) disease-modifying antirheumatic drugs (DMARDs). They evaluated efficacy outcomes separately, including response rates (based on the American College of Rheumatology 20/50/70% response criteria) and remission rates (measured by Disease Activity Score in 28 joints derived from 4 measures, erythrocyte sedimentation rate [DAS28-4(ESR)] < 2.6) at month 3, as well as changes from baseline in DAS28-4(ESR) and Health Assessment Questionnaire–Disability Index scores. Safety assessments were conducted for up to 24 months.

The researchers reported that, at 3rd month, tofacitinib demonstrated improved efficacy outcomes compared to placebo in all the three populations. Patients with an inadequate response to non-MTX csDMARDs showed the most significant improvement in efficacy outcomes with tofacitinib. Over the 24-month period, patients with inadequate responses to non-MTX csDMARDs and MTX experienced fewer adverse events (AEs), serious AEs, and discontinuations due to AEs compared to patients with inadequate responses to bDMARDs. Rates of major adverse events were similar across all populations.

In line with this study, Wollenhaupt et al. reported that tofacitinib 5 mg and 10 mg twice daily showed consistent safety and sustained efficacy in this open-label long-term extension study of patients with RA. The safety data were reported for up to 9.5 years, and efficacy data for up to 8 years, based on adequate patient numbers to support the study.

The researchers emphasized that tofacitinib offers clinical benefits to RA patients across a spectrum of prior treatment experiences. In addition, tofacitinib might exhibit greater efficacy and a more favorable benefit-to-risk profile in a specific subset of patients characterized by a shorter duration of disease and limited prior experience with DMARDs.

The researchers specifically identified patients with intolerance, contraindications, or poor adherence to MTX therapy as potential candidates who may particularly benefit from tofacitinib. This observation underscores the importance of considering individual patient characteristics, such as the duration of disease and prior treatment experiences, in tailoring the therapeutic approach for optimal outcomes with tofacitinib in the management of RA.


  1. Tesser J, Gül A, Olech E, Oelke K, Lukic T, Kwok K, et al. Efficacy and safety of tofacitinib in patients with rheumatoid arthritis by previous treatment: post hoc analysis of phase II/III trials. Arthritis Research & Therapy. 2023 Nov 2;25(1):214.
  2. Wollenhaupt J, Lee EB, Curtis JR, Silverfield J, Terry K, Soma K, et al. Safety and efficacy of tofacitinib for up to 9.5 years in the treatment of rheumatoid arthritis: final results of a global, open-label, long-term extension study. Arthritis Research & Therapy. 2019 Apr 5;21(1):89.