DAY 3 - Real World Comparison of Effectiveness, Durability, and Safety of First-Line Advanced Therapies for Ulcerative Proctitis

Ulcerative proctitis, a subtype of ulcerative colitis, is typically treated with topical 5-aminosalicylic acid and steroid therapies. Many patients with refractory ulcerative proctitis require advanced therapies. However, data on the effectiveness of these advanced therapies in ulcerative proctitis patients have been limited, creating a significant knowledge gap in the field.

At the American College of Gastroenterology (ACG) 2024 Annual Scientific Meeting in Philadelphia, Pennsylvania, Rahul Dalal, MD, a gastroenterologist from the Brigham and Women's Hospital, discussed the challenges faced by ulcerative proctitis patients, who commonly experience severe symptoms despite the localized nature of their condition. 

To address this issue, Dr. Dalal and his team conducted a retrospective cohort study comparing real-world outcomes of advanced therapies in bio-naïve patients with ulcerative proctitis. The research method involved analyzing adults who initiated advanced therapies for ulcerative proctitis between 2014 and 2023; diagnoses were confirmed through endoscopic reports. The primary outcome of the study was steroid-free clinical remission (SFCR) at 52 weeks. Secondary outcomes included treatment persistence at 52 weeks and adverse events.

Dr. Dalal shared the results of their study, which included 72 adults who initiated various advanced therapies for ulcerative proctitis: 47% vedolizumab, 44% anti-TNFs (of which 69% was adalimumab), 4% ustekinumab, 3% tofacitinib, and 1% ozanimod. His team found that vedolizumab was associated with significantly higher adjusted odds of SFCR at 52 weeks compared to anti-TNFs. Furthermore, they observed lower treatment persistence and a higher hazard of discontinuation for anti-TNFs compared to vedolizumab, although these findings were not statistically significant. Interestingly, a subgroup analysis revealed that adalimumab was associated with a significantly higher discontinuation hazard than vedolizumab.

The strength of this study was that it included a uniform population of bio-naïve patients with ulcerative proctitis and verified disease extent via manual review of endoscopy reports. The key finding of high odds of SFCR and treatment persistence in patients treated with vedolizumab for ulcerative proctitis can guide clinicians in making more informed decisions when selecting advanced therapies for ulcerative proctitis patients who have not responded to conventional therapies.

However, Dr. Dalal also acknowledged the limitations of their research. The study's retrospective nature and the relatively small sample size may limit the generalizability of the results. Additionally, the study predominantly included representation of adalimumab among anti-TNFs, and it did not include newer advanced therapies that have recently entered the market, which may offer additional treatment options for ulcerative proctitis patients.

Dr. Dalal concluded his presentation by shedding light on the implications of his research and future directions: "In bio-naïve patients with ulcerative proctitis, vedolizumab may be more effective at achieving SFCR and treatment persistence at one year compared to anti-TNFs, primarily adalimumab. We need larger cohorts to improve our understanding of the comparative effectiveness of these therapies, as well as assess long-term outcomes for newer therapies that have recently come out."