Pricey arthritis drug effective in small fraction of ulcerative colitis cases

Reuters Health Information: Pricey arthritis drug effective in small fraction of ulcerative colitis cases

Pricey arthritis drug effective in small fraction of ulcerative colitis cases

Last Updated: 2017-05-08

By Gene Emery

NEW YORK (Reuters Health) - Pfizer's expensive arthritis drug tofacitinib has been shown to produce a remission in nearly 1 in 5 patients with moderate to severe ulcerative colitis, but long-term remission persists in fewer than half of those cases.

In a series of studies published in the May 4 New England Journal of Medicine, researchers reported remission rates of 18.5% and 16.6% at 8 weeks. Rates with placebo were 8.2% (P=0.007) and 3.6% (P<0.001) respectively. Combined, 1,139 patients were treated.

In the long-term test, which only included 593 volunteers who responded to induction therapy, the remission rate at the 52-week mark was 34.4% with 5 mg twice daily, which would cost nearly $50,000 per year according to

At double the dose and an annual cost approaching $100,000, the success rate rose 6 additional percentage points, to 40.6%.

The long-term remission rate in the responders switched to placebo was 11.1% (P<0.001).

When the research team looked at mucosal healing, a dose response was also seen, with a rate of 37.4% with 5 mg of tofacitinib and 45.7% at the 10 mg dose. The rate was only 13.1% with placebo.

Pfizer paid for the study.

While fewer than 1 in 13 of all volunteers showed a long-term response, the patient pool consisted of people for whom conventional therapy, such as glucocorticoids, azathioprine or infliximab, had failed or caused too many side effects.

In the induction studies, known as OCTAVE Induction 1 and OCTAVE Induction 2, more patients receiving the test drug developed an infection. The highest rate was 23.3% versus 18.2% for placebo.

Among patients who responded to the drug in the initial phase, infections were more common in the long-term test, known as OCTAVE Sustain. The rates were 39.8% with the high-dose therapy, 35.9% with lower-dose treatment and 24.2% with placebo. Most infections were ranked mild or moderate.

In the Sustain trial, the rates of herpes zoster infection were 0.5% with placebo, 1.5% with the 5-mg dose and 5.1% with the 10-mg dose.

"As compared with placebo, tofacitinib treatment was associated with increased levels of high-density lipoprotein and low-density lipoprotein cholesterol, more overall infections, and more cases of herpes zoster infection, so the benefit of this therapy comes at a price," Dr. Sonia Friedman of the Brigham and Women̢۪s Hospital Center for Crohn̢۪s and Colitis said in a Journal editorial. "Whether tofacitinib will be an essential therapy for ulcerative colitis remains to be determined in future trials."


N Engl J Med 2017.

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