Background Real-world data on the effectiveness and safety of ustekinumab (UST) in ulcerative colitis (UC) are lacking in Latin America. In this study, we aimed to describe the effectiveness and safety of UST in a real-world multicentre cohort of Brazilian patients with UC. Methods We conducted a multicentre retrospective observational cohort study, including patients with moderate to severe UC (Total Mayo score 6-12, with an endoscopic subscore of 2 or 3) who received UST. The co-primary endpoints were clinical remission, defined as a total Mayo score ≤2 at 1 year, with a combined rectal bleeding and stool frequency subscore of ≤1, and endoscopic remission (endoscopic Mayo subscore of zero) within one year from baseline. Secondary endpoints included clinical response between weeks 12-16, endoscopic response within one year of starting UST, steroid-free clinical remission at week 52, and biochemical remission at week 52. We also evaluated UST treatment persistence and safety. Results A total of 50 patients were included (female, n=36, 72.0%), with a median disease duration of 9.2 years (1-27). Most patients had extensive colitis (n=38, 76.0%), and 43 (86.0%) were steroid-dependent at baseline. Forty patients (80.0%) were previously exposed to biologics (anti-TNF drugs, n=31; vedolizumab [VDZ], n=27). The co-primary endpoints of clinical remission and endoscopic remission at 1 year were achieved by 50.0% and 36.0% of patients, respectively. Clinical response at weeks 12-16 was 56.0%, endoscopic response, steroid-free clinical remission, and biochemical remission at week 52 were 68.0%, 67.4%, and 50.0%, respectively. The UST treatment persistence rates at 24 months was 73.7%. During the follow-up, 10 patients (20.0%) were hospitalized, mostly due to disease progression, and three patients required colectomy. Nine patients (18.0%) discontinued the drug mainly due to a lack of effectiveness. Twenty-six adverse events (AEs) were reported, 15 of which were considered as serious AEs. Conclusion This is the first real-world experience study to report the effectiveness and safety of UST specifically in a Latin American population. In this real-world cohort of difficult-to-treat UC patients, UST was associated with improvements in clinical, biochemical, and endoscopic outcomes. The safety profile was favorable, consistent with the known profile of UST.

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P698 乌司奴单抗治疗溃疡性结肠炎的有效性和安全性：巴西多中心真实世界观察研究

背景 拉丁美洲缺乏乌特克单抗 (UST) 治疗溃疡性结肠炎 (UC) 有效性和安全性的真实世界数据。在这项研究中，我们旨在描述 UST 在巴西 UC 患者的真实多中心队列中的有效性和安全性。方法 我们进行了一项多中心回顾性观察队列研究，包括接受 UST 的中度至重度 UC 患者（Mayo 总评分 6-12，内镜分项评分为 2 或 3）。共同主要终点是临床缓解，定义为 1 年时 Mayo 总评分≤2，直肠出血和大便频率总分≤1，以及基线一年内内镜缓解（内镜 Mayo 评分为零）。次要终点包括第12-16周之间的临床反应、开始UST后一年内的内镜反应、第52周时的无类固醇临床缓解以及第52周时的生化缓解。我们还评估了UST治疗的持久性和安全性。结果共纳入50例患者（女性，n=36，72.0%），中位病程为9.2年（1-27）。大多数患者患有广泛性结肠炎（n=38，76.0%），43 例（86.0%）在基线时有类固醇依赖性。40 名患者 (80.0%) 之前曾接触过生物制剂（抗 TNF 药物，n=31；维多珠单抗 [VDZ]，n=27）。1 年时临床缓解和内镜缓解的共同主要终点分别由 50.0% 和 36.0% 的患者实现。第12-16周的临床缓解率为56.0%，第52周的内镜缓解、无类固醇临床缓解和生化缓解分别为68.0%、67.4%和50.0%。24 个月时 UST 治疗持续率为 73.7%。随访期间，10名患者（20.0%）因疾病进展而住院，其中3名患者需要结肠切除术。9 名患者（18.0%）主要由于缺乏疗效而停药。报告了 26 起不良事件 (AE)，其中 15 起被视为严重 AE。结论 这是第一项真实世界经验研究，专门报告 UST 在拉丁美洲人群中的有效性和安全性。在这个现实世界中难以治疗的 UC 患者队列中，UST 与临床、生化和内窥镜结果的改善相关。安全性良好，与 UST 的已知情况一致。