Rheumatoid arthritis: long-term safety and efficacy of tofacitinib

  • Wollenhaupt J & al.
  • Arthritis Res Ther
  • 5 Apr 2019

  • curated by Sarfaroj Khan
  • UK Clinical Digest
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  • Tofacitinib 5 and 10 mg twice daily (BID) demonstrated consistent long-term safety (as monotherapy or combination therapy) and sustained efficacy in patients with rheumatoid arthritis (RA).

Why this matters

  • Safety and efficacy data are consistent with findings from prior tofacitinib phase 2, phase 3, and long-term extension studies.

Study design

  • Patients with RA (n=4481) who had previously completed a phase 1, 2 or 3 tofacitinib qualifying index study and received tofacitinib 5 (n=1123) and 10 mg (n=3358) BID were evaluated for long-term safety and efficacy profile.
  • Funding: Pfizer Inc.

Key results

  • Total tofacitinib exposure was 16,291 patient-years.
  • Safety data were reported up to 114 months for tofacitinib and efficacy data up to 96 months for tofacitinib 5 mg and up to 72 months for tofacitinib 10 mg.
  • Overall, 52% of patients discontinued by 114 months and the safety profile remained consistent with prior phase 1, 2, 3 studies or long-term extension studies.
  • Incidence rate (IR) for adverse events (AEs) leading to discontinuation was 6.8 (95% CI, 6.39-7.20) for all tofacitinib and IR for serious AEs was 9.03 (95% CI, 8.55-9.53).
  • For all-cause AEs, IRs were 3.4 (95% CI, 3.2-3.7), 2.4 (95% CI, 2.2-2.6), 0.8 (95% CI, 0.7-1.0), 0.4 (95% CI, 0.3-0.5) and 0.3 (95% CI, 0.19-0.36) for herpes zoster, serious infections, malignancies excluding non-melanoma skin cancer, major adverse cardiovascular events and for all-cause mortality, respectively.
  • Clinically meaningful improvements in the signs and symptoms and physical functioning were maintained over time in patients who continued tofacitinib treatment.


  • Tofacitinib dose assignment using average total daily dose does not account for cumulative treatment exposure or dose changes over time.