P4257 - Disease Characterization and Safety Contextualization in Patients with Ulcerative Colitis Using a Healthcare Administrative Database in the United States

Miguel D Regueiro, MD¹, Meenakshi Bewtra, MD², Marla C. Dubinsky, MD³, Xiang Guo, PharmD, MS⁴, Chunyang Feng, ⁵, Aoibhinn McDonnell, PhD⁶, Milena Gianfrancesco, PhD, MPH^5
1Cleveland Clinic, Cleveland, OH; ²University of Pennsylvania, Philadelphia, PA; ³Icahn School of Medicine at Mount Sinai, New York, NY; ⁴Pfizer Inc., Collegeville, PA; ⁵Pfizer Inc., New York, NY; ⁶Pfizer Ltd., Sandwich, England, United Kingdom

Introduction: Several therapies have been recently approved for ulcerative colitis (UC).¹ Although clinical trials assess drug safety, they should be contextualized with real‑world data (RWD), especially as many rare events are not commonly reported in clinical trials.

Methods: This retrospective, observational study aimed to use RWD to investigate safety outcomes in four cohorts; three for patients with UC: (1) those receiving any treatment (UC overall), (2) those receiving advanced therapies (UC advanced therapy), and (3) those meeting the selection criteria of the etrasimod ELEVATE UC clinical program² (UC trial similar); the fourth cohort comprised individuals without UC (non-UC; Table). Data were extracted (1/1/2016–12/31/2022) from the United States Optum electronic health record database. Demographic data were summarized descriptively. Incidence rates per 1000 patient‑years were calculated (Table).

Results: Data from 32,170 (UC overall), 3,332 (UC advanced therapy), 1,435 (UC trial similar), and 160,795 (non-UC) individuals were included. Cohorts were well balanced regarding sex (female: 48.5% – 54.5%) and were mostly White (86.3% – ‍86.8%) with mean ages of 52.3 (UC overall), 45.0 (UC advanced therapy), 44.0 (UC trial similar), and 52.3 (non-UC) years. The UC overall cohort had numerically the highest rates across nearly all selected safety outcomes (Table). The non-UC and UC trial similar cohorts had generally comparable rates followed by the UC advanced therapy cohort for cardiac, vascular, and circulatory disorders and metabolism and endocrine disorders. These three cohorts had similar rates for rare outcomes: cryptococcal meningitis, progressive multifocal leukoencephalopathy, malignancy (cutaneous), and retinal diseases (Table). Generally, all UC cohorts had numerically higher rates vs the non-UC cohort for most infections and liver injuries (Table).

Discussion: This large-population RWD assessment comprising common and rarely observed safety events found that patients with UC had higher rates for most outcomes vs a matched non-UC population. Overall, the UC advanced therapy and UC trial similar cohorts had lower rates of safety events vs the UC overall cohort and were more comparable to the non-UC cohort. These findings assist in understanding the background risks of safety events in patients with UC, allowing contextualization with clinical trial data and future RWD.

References:

1. Ferretti F et al. J Clin Med 2022; 11: 2302.
2. Sandborn WJ et al. Lancet 2023; 401: 1159–1171.


Disclosures:


Miguel D Regueiro, MD¹, Meenakshi Bewtra, MD², Marla C. Dubinsky, MD³, Xiang Guo, PharmD, MS⁴, Chunyang Feng, ⁵, Aoibhinn McDonnell, PhD⁶, Milena Gianfrancesco, PhD, MPH⁵. P4257 - Disease Characterization and Safety Contextualization in Patients with Ulcerative Colitis Using a Healthcare Administrative Database in the United States, ACG 2024 Annual Scientific Meeting Abstracts. Philadelphia, PA: American College of Gastroenterology.