Long-Term Safety, Efficacy Tofacitinib for Rheumatoid Arthritis

arthritic hand
arthritic hand
Researchers found data that showed tofacitinib 5 mg and 10 mg twice daily is safe and effective.

For patients with rheumatoid arthritis (RA), tofacitinib 5 mg and 10 mg twice daily has a consistent safety profile and sustained efficacy, according to results published in Arthritis Research & Therapy.

The study included participants who had previously completed a phase 1, 2, or 3 qualifying index study of tofacitinib (n=4481). Participants received open-label tofacitinib 5 mg or 10 mg twice daily based on their average total daily dose. Participants were allowed to continue stable background therapy, including conventional synthetic disease-modifying antirheumatic drugs (csDMARDs).

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The primary outcome was the long-term safety and tolerability of tofacitinib, and the secondary outcome was the long-term persistence of efficacy.

The participants contributed 16,291 patient-years of exposure to tofacitinib. The researchers had safety data through month 114. For efficacy data, they had data up to month 96 for tofacitinib 5 mg and up to month 72 for 10 mg.

During follow-up, 52% of participants discontinued treatment, 24% because of adverse events and 4% due to insufficient clinical response. The safety profile of tofacitinib was consistent with that found in prior phase 1, 2, 3, or long-term extension studies.

The incidence rate for adverse events leading to discontinuation was 6.8 per 100 person-years. Herpes zoster had an incidence rate of 3.4, serious infections had an incidence rate of 2.4, malignancies (excluding nonmelanoma cancer) had an incidence rate of 0.8, major adverse cardiovascular events had an incidence rate of 0.4, and all-cause mortality had an incidence rate of 0.3.

The results indicated that clinically meaningful improvements in RA symptoms and physical function were maintained, as seen in index studies.

“The safety profile of tofacitinib in patients who initiated tofacitinib as monotherapy was generally similar to that observed when tofacitinib was initiated in combination with csDMARDs,” the researchers wrote.

Please see the original reference for a full list of disclosures.


Wollenhaupt J, Lee E, Curtis JR, 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 Res Ther. 2019;21(1):89.