Tofacitinib, an Oral Janus Kinase Inhibitor: Analysis of Malignancy (Excluding Nonmelanoma Skin Cancer) Events Across the Ulcerative Colitis Clinical Program

Inflamm Bowel Dis. 2020 Aug 7;izaa199. doi: 10.1093/ibd/izaa199. Online ahead of print.

Gary R Lichtenstein 1, Gerhard Rogler 2, Matthew A Ciorba 3, Chinyu Su 4, Gary Chan 4, Ronald D Pedersen 4, Nervin Lawendy 4, Daniel Quirk 4, Chudy I Nduaka 4, Andrew J Thorpe 4, Julian Panés 5


Author information

  • 1Division of Gastroenterology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA.
  • 2Department of Gastroenterology and Hepatology, University of Zürich, Zürich, Switzerland.
  • 3Division of Gastroenterology, Washington University in Saint Louis, Saint Louis, Missouri, USA.
  • 4Pfizer Inc, Collegeville, Pennsylvania, USA.
  • 5Hospital Clínic de Barcelona, IDIBAPS, CIBERehd, Barcelona, Spain.


Background: Tofacitinib is an oral, small molecule Janus kinase inhibitor for the treatment of ulcerative colitis (UC). Here, we performed an integrated analysis of malignancy events from the tofacitinib phase 3 UC clinical development program (excluding nonmelanoma skin cancer [NMSC]).

Methods: Data (up to May 2019) were pooled from two phase 3 induction studies, a phase 3 maintenance study, and an ongoing, open-label, long-term extension (OLE) study, and analyzed as 3 cohorts: induction (N = 1139), maintenance (N = 592), and overall (induction, maintenance, and ongoing OLE study; N = 1124). Proportions and incidence rates (IRs; unique patients with events per 100 patient-years [PY] of exposure) for malignancies confirmed by adjudication were calculated.

Results: The overall cohort consisted of patients who received at least 1 dose of tofacitinib at 5 or 10 mg twice daily, for up to 6.8 years, with an exposure of 2576.4 PY. Of the 1124 overall cohort tofacitinib-treated patients, 20 developed a malignancy (excluding NMSC; IR, 0.75; 95% confidence interval, 0.46-1.16), of which 17 occurred in patients treated with tofacitinib 10 mg twice daily; importantly, more than 80% of patients predominantly received this dose. Furthermore, there was no apparent clustering of malignancy types, and IRs were stable over time.

Conclusions: In the tofacitinib UC clinical development program, malignancy events were infrequent, and rates were comparable with those in the tofacitinib rheumatoid arthritis and psoriatic arthritis clinical development programs, and for biologic UC treatments. ClinicalTrials.gov: NCT01465763, NCT01458951, NCT01458574, and NCT01470612.

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