Tofacitinib Treatment Safety in Moderate to Severe Ulcerative Colitis: Comparison of Observational Population Cohort Data From the IBM MarketScan® Administrative Claims Database With Tofacitinib Trial Data

Jeffrey R Curtis; Miguel Regueiro; Huifeng Yun; Chinyu Su; Marco DiBonaventura; Nervin Lawendy; Chudy I Nduaka; Nana Koram; Joseph C Cappelleri; Gary Chan; Irene Modesto; Gary R Lichtenstein

doi:10.1093/ibd/izaa289

Tofacitinib Treatment Safety in Moderate to Severe Ulcerative Colitis: Comparison of Observational Population Cohort Data From the IBM MarketScan® Administrative Claims Database With Tofacitinib Trial Data

Inflamm Bowel Dis. 2021 Aug 19;27(9):1394-1408. doi: 10.1093/ibd/izaa289.

Authors

Affiliations

¹ Division of Clinical Immunology and Rheumatology, Department of Medicine, Department of Epidemiology, University of Alabama at Birmingham, Birmingham, AL, USA.
² Department of Gastroenterology, Hepatology, and Nutrition, Cleveland Clinic, Cleveland, OH, USA.
³ Pfizer Inc, Collegeville, PA, USA.
⁴ Pfizer Inc, New York, NY, USA.
⁵ Pfizer Inc, Groton, CT, USA.
⁶ Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA, USA.

Abstract

Background: Tofacitinib is an oral, small molecule Janus kinase inhibitor for the treatment of ulcerative colitis (UC). We aimed to estimate the overall incidence of safety events in patients with UC in a real-life population cohort for comparison with the tofacitinib UC clinical trial program.

Methods: Clinical trial-like criteria were applied to an IBM MarketScan® claims database population-based cohort (n = 22,967) of patients with UC (October 2010 to September 2015) to identify a UC trial-like cohort treated with tumor necrosis factor inhibitors (TNFi; n = 6366) to compare with the tofacitinib UC clinical trial cohort (n = 1157).

Results: Incidence rates (events per 100 patient-years; [95% confidence interval]) in the UC trial-like cohort were as follows: serious infections, 3.33 (2.73-4.02); opportunistic infections (OIs; excluding herpes zoster [HZ]), 1.45 (1.06-1.93); HZ, 1.77 (1.34-2.29); malignancies (excluding nonmelanoma skin cancer [NMSC]), 0.63 (0.43-0.90); NMSC, 1.69 (1.35-2.10); major adverse cardiovascular events (MACE), 0.51 (0.31-0.79); pulmonary embolism (PE), 0.54 (0.30-0.89); deep vein thrombosis (DVT), 1.41 (1.00-1.93); and gastrointestinal perforations, 0.31 (0.16-0.54). Compared with the UC trial-like cohort, tofacitinib-treated patients had numerically lower incidence rates for serious infections (1.75 [1.27-2.36]), OIs (excluding HZ; 0.16 [0.04-0.42]), NMSC (0.78 [0.47-1.22]), PE (0.16 [0.04-0.41]), and DVT (0.04 [0.00-0.23]), and a higher rate for HZ (3.57 [2.84-4.43]); rates for malignancies (excluding NMSC), MACE, and gastrointestinal perforations were similar.

Conclusions: When acknowledging limitations of comparing claims data with controlled clinical trial data, incidence rates for HZ among TNFi-treated patients in the UC trial-like cohort were lower than in the tofacitinib UC clinical trial cohort; rates for serious infections, OIs, NMSC, PE, and DVT were numerically higher.

Clinicaltrials.gov: NCT00787202, NCT01465763, NCT01458951, NCT01458574, NCT01470612.

Keywords: indirect comparison; safety; ulcerative colitis.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Colitis, Ulcerative* / drug therapy
Colitis, Ulcerative* / epidemiology
Humans
Neoplasms
Opportunistic Infections
Piperidines / therapeutic use*
Product Surveillance, Postmarketing
Pyrimidines / therapeutic use*
Randomized Controlled Trials as Topic

Substances

Piperidines
Pyrimidines
tofacitinib

Associated data

ClinicalTrials.gov/NCT01458574
ClinicalTrials.gov/NCT01465763
ClinicalTrials.gov/NCT01470612
ClinicalTrials.gov/NCT01458951