Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Tofacitinib as Induction & Maintenance Therapy

N Engl J Med; 2017 May 4; Sandborn, et al

Tofacitinib was more effective as induction and maintenance therapy than placebo in patients with moderately to severely active ulcerative colitis, according to 3 phase 3, randomized, double-blind, placebo-controlled trials of tofacitinib therapy in adults with ulcerative colitis. In the OCTAVE Induction 1 and 2 trials, 598 and 541 patients, respectively, were randomly assigned to receive induction therapy with tofacitinib (10 mg twice daily) or placebo for 8 weeks. In the OCTAVE Sustain trial, 593 patients who had a clinical response to induction therapy were randomly assigned to receive maintenance therapy with tofacitinib (either 5 mg or 10 mg twice daily) or placebo for 52 weeks. Researchers found:

  • In the OCTAVE Induction 1 trial, remission at 8 weeks occurred in 18.5% of the patients in the tofacitinib group vs 8.2% in the placebo group; in the OCTAVE Induction 2 trial, remission occurred in 16.6% vs 3.6%.
  • Remission at 52 weeks occurred in 34.3% of the patients in the 5-mg tofacitinib group and 40.6% in the 10-mg tofacitinib group vs 11.1% in the placebo group in the OCTAVE Sustain trial.


Sandborn WJ, Su C, Sands BE, et al. Tofacitinib as induction and maintenance therapy for ulcerative colitis. N Engl J Med. 2017;376:1723-1736. doi:10.1056/NEJMoa1606910.


Ulcerative colitis (UC) is a chronic inflammatory condition of the large colon that can cause chronic bloody diarrhea and abdominal pain. The goal of treatment is to induce remission of active disease and prevent relapse. For mild disease, remission can often be induced using 5-aminosalicylic acid (5-ASA) topically (suppository or enema) or orally. If 5-ASA is not sufficient, then often an oral course of corticosteroids is used.1 For corticosteroid-refractory disease, monoclonal antibody therapies, particularly anti–tumor necrosis factor (TNF) medications, are used (infliximab, adalimumab, and golimumab). A newer monoclonal antibody, vedolizumab, targets gut-selective immunosuppression and may have fewer side effects than the other anti-TNF medications. Tofacitinib, described above in the OCTAVE trials, is in a new class of medications of small-molecule drugs called janus kinases (JAKs) that block or modulate inflammatory pathways. Tofacitinib inhibits multiple cytokine receptors and has been previously approved for the treatment of rheumatoid arthritis.2 This class of medications offers the promise of efficacy at a much lower cost than monoclonal antibodies, and hopefully with fewer side effects. —Neil Skolnik, MD

  1. Adams SM, Bornemann S. Ulcerative colitis. Am Fam Physician. 2013;87(10):699-705.
  2. Freidman S. Tofacitinib for ulcerative colitis—A promising step forward. N Engl J Med. 2017;376:1792-1793. doi:10.1056/NEJMe1701505.

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