P260 An analysis of non-melanoma skin cancer rates in the tofacitinib Ulcerative Colitis clinical programme

Sands, B.E.(1);Cohen, R.D.(2);Ha, C.(3);Reinisch, W.(4);Salese, L.(5);Mundayat, R.(6);Lawendy, N.(5);Panés, J.(7);

(1)Dr. Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York- New York, United States;(2)Inflammatory Bowel Disease Center, University of Chicago Medicine, Chicago- Illinois, United States;(3)Inflammatory Bowel Disease Center, Cedars-Sinai Medical Center, Los Angeles- California, United States;(4)-, Medical University of Vienna, Vienna, Austria;(5)Pfizer Inc, Collegeville, Pennsylvania, United States;(6)Pfizer Inc, New York, New York, United States;(7)Department of Gastroenterology, Hospital Clínic de Barcelona- IDIBAPS- CIBERehd, Barcelona, Spain


Tofacitinib is an oral, small molecule JAK inhibitor for the treatment of ulcerative colitis (UC). We present an updated analysis of non-melanoma skin cancer (NMSC) events in the tofacitinib UC clinical programme, including final data from the open‑label, long-term extension (OLE) study (as of 24 Aug 2020).


NMSC events were evaluated from 3 randomised, placebo (PBO)-controlled studies (2 Phase [P]3 induction studies [NCT01465763; NCT01458951]; 1 P3 maintenance study [NCT01458574]) and an OLE study (NCT01470612), as 3 cohorts: Induction (P3 induction studies [patients (pts) receiving tofacitinib 10 mg twice daily (BID) or PBO]); Maintenance (P3 maintenance study [pts receiving tofacitinib 5 or 10 mg BID or PBO]); Overall (pts receiving tofacitinib 5 or 10 mg BID in P3 or OLE studies). Analysis was by predominant dose (PD) 5 or 10 mg BID, based on average daily dose <15 or ≥15 mg, respectively (82% of pts received PD 10 mg BID). An independent adjudication committee reviewed potential NMSC. Proportions and incidence rates (IRs; unique pts with events per 100 pt-years of exposure) were evaluated for NMSC. A Cox proportional hazards model was used for risk factor analysis.


1124 pts were evaluated for NMSC (2809.4 pt-years of tofacitinib exposure; up to 7.8 years of treatment; median duration 685.5 days). NMSC events in Induction and Maintenance were previously reported (Table 1).1 In Overall, NMSC occurred in 21 pts (IR 0.73 [95% confidence interval (CI) 0.45, 1.12]): PD tofacitinib 5 mg BID n=5, IR 0.63 (0.21, 1.48); PD tofacitinib 10 mg BID n=16, IR 0.77 (0.44, 1.25) (Table 1); 2 new cases since May 2019.1 Eleven pts had squamous cell carcinoma and 15 pts had basal cell carcinoma; 5 pts had both. No NMSC was metastatic or led to discontinuation. IRs by time interval and subgroup are reported (Table 2). Prior NMSC (hazard ratio [HR] 12.08 [95% CI 4.20, 34.76]) and age (per 10-year increase, HR 2.01 [1.38, 2.93]) were significant risk factors for NMSC in the multivariable analysis. Prior immunosuppressant use was not a significant risk factor in either the multivariable or univariate analyses.


In this analysis, NMSC IRs for tofacitinib were similar to those in pts with UC treated with biologics2 and those previously reported in the tofacitinib UC clinical programme.1 NMSC events were more likely to occur in pts with recognised NMSC risk factors: prior NMSC and increasing age.3 Dose dependency of NMSC IR could not be concluded here, as dose changes were permitted. NMSC IRs remained stable over time, up to 7.8 years of exposure.


1. Sands et al. United European Gastroenterol J 2020;8(S):P0498
2. Curtis et al. Inflamm Bowel Dis 2020;Epub ahead of print
3. Marcil, Stern. Arch Dermatol 2000;136:1524-30