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Safety of Tofacitinib for Treatment of Ulcerative Colitis, Based on 4.4 Years of Data From Global Clinical Trials
William J Sandborn1, Julián Panés2, Geert R D'Haens3
1Division of Gastroenterology, University of California, San Diego, La Jolla, California.
Background & Aims:
Tofacitinib is an oral, small-molecule inhibitor of JAK approved in several countries for the treatment of ulcerative colitis (UC). We report integrated safety analyses of tofacitinib-treated patients with moderate to severe UC.
Methods:
Patients receiving placebo or tofacitinib (5 or 10 mg) twice daily were analyzed as 3 cohorts: induction (phase 2 and 3 induction studies, n = 1220), maintenance (phase 3 maintenance study, n = 592), and overall (patients receiving tofacitinib 5 or 10 mg twice daily in phase 2, phase 3, or open-label, long-term extension studies, n = 1157; 1613 patient-years' exposure). Incidence rates (IRs; patients with events per 100 patient-years of exposure) were evaluated for select adverse events.
Results:
In the maintenance cohort, IRs for select adverse events were similar among treatment groups, except for a numerically higher IR of herpes zoster infection among patients who received tofacitinib 5 mg twice daily (2.1; 95% CI, 0.4-6.0) and statistically higher IR among patients who received tofacitinib 10 mg twice daily (IR, 6.6; 95% CI, 3.2-12.2) vs placebo (IR, 1.0, 95% CI, 0.0-5.4). For the overall cohort (84% received average dose of tofacitinib 10 mg twice daily), IRs were: death, 0.2 (95% CI, 0.1-0.6); serious infections, 2.0 (95% CI, 1.4-2.8); opportunistic infections, 1.3 (95% CI, 0.8-2.0); herpes zoster infection, 4.1 (95% CI, 3.1-5.2); malignancy (excluding non-melanoma skin cancer), 0.7 (95% CI, 0.3-1.2); non-melanoma skin cancer, 0.7 (95% CI, 0.3-1.2); major adverse cardiovascular events, 0.2 (95% CI, 0.1-0.6); and gastrointestinal perforations, 0.2 (95% CI, 0.0-0.5).
Conclusions:
In safety analyses of patients with moderate to severe UC treated with tofacitinib, we observed a dose relationship with herpes zoster infection. Although follow-up time was relatively short, the safety profile of tofacitinib for patients with UC appeared similar to that reported for patients with rheumatoid arthritis and for patients with UC treated with biologic agents, except for the higher IR of herpes zoster infection. ClinicalTrials.gov, no: NCT00787202, NCT01465763, NCT01458951, NCT01458574, and NCT01470612.
Insights
Tofacitinib, a JAK inhibitor for ulcerative colitis (UC), showed a dose-dependent increase in herpes zoster infections. Overall safety in UC patients was similar to other conditions, with notable adverse event rates reported.
Area of Science:
- Gastroenterology
- Immunology
- Pharmacology
Background:
- Tofacitinib is an oral Janus kinase (JAK) inhibitor approved for ulcerative colitis (UC).
- Integrated safety analyses were conducted on patients with moderate to severe UC treated with tofacitinib.
Purpose of the Study:
- To evaluate the safety profile of tofacitinib in patients with moderate to severe UC.
- To assess the incidence rates of select adverse events associated with tofacitinib treatment.
Main Methods:
- Patients were analyzed in induction, maintenance, and overall cohorts, receiving placebo or tofacitinib (5 or 10 mg twice daily).
- Incidence rates (IRs) per 100 patient-years were calculated for adverse events.
Main Results:
- Herpes zoster infection IRs were higher with tofacitinib, particularly at the 10 mg dose, compared to placebo.
- In the overall cohort, IRs were reported for death, serious infections, opportunistic infections, herpes zoster, malignancies, MACE, and GI perforations.
Conclusions:
- A dose relationship was observed between tofacitinib and herpes zoster infection in UC patients.
- The safety profile in UC patients was comparable to rheumatoid arthritis patients, except for a higher incidence of herpes zoster.
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