BACKGROUND & AIMS It is a challenge to manage patients with ulcerative proctitis (UP) refractory to standard therapy. We investigated the effectiveness of tumor necrosis factor (TNF) antagonists in a large cohort of patients with refractory UP. METHODS We conducted a nationwide retrospective cohort study of 104 consecutive patients with active UP refractory to conventional therapies, treated at 1 of 15 centers in France or 1 center in Belgium (the GETAID cohort). Patients received at least 1 injection of anti-TNF (infliximab, adalimumab, golimumab) from October 2006 through February 2017. Clinical response was defined as significant improvement in UC-related symptoms, and remission as complete disappearance of UC-related symptoms, each determined by treating physicians. We collected demographic, clinical, and treatment data. The median duration of follow-up was 24 months (interquartile range, 13-51 months). The primary outcome was clinical response of UP to anti-TNF treatment. RESULTS Overall, 80 patients (77%) had a clinical response to anti-TNF therapy and 52 patients (50%) achieved clinical remission. Extra-intestinal manifestations (odds ratio OR, 0.24; 95% CI, 0.08-0.7), ongoing treatment with topical steroids (OR, 0.14; 95% CI, 0.03-0.73), and ongoing treatment with topical 5-aminosalycilates (OR, 0.21; 95% CI, 0.07-0.62) were significantly associated with the absence of clinical remission. Sixty percent (38/63) of the patients who had endoscopic assessment during follow up had mucosal healing. Among the overall population (n = 104), the cumulative probabilities of sustained clinical remission were 87.6% ± 3.4% at 1 year and 74.7% ± 4.8% at 2 years. CONCLUSIONS In a retrospective study of 104 patients with refractory UP, anti-TNF therapy induced clinical remission in 50% and mucosal healing in 60%. About two thirds of the patients were still receiving anti-TNF therapy at 2 years.

中文翻译：

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肿瘤坏死因子拮抗剂治疗难治性溃疡性直肠炎患者的疗效。

背景和目的 管理对标准治疗无效的溃疡性直肠炎 (UP) 患者是一项挑战。我们在一大群难治性 UP 患者中研究了肿瘤坏死因子 (TNF) 拮抗剂的有效性。方法 我们对 104 名在法国 15 个中心中的 1 个中心或比利时的 1 个中心（GETAID 队列）接受治疗的传统疗法难治性活动性 UP 连续患者进行了一项全国性回顾性队列研究。从 2006 年 10 月到 2017 年 2 月，患者至少接受了 1 次抗 TNF（英夫利昔单抗、阿达木单抗、戈利木单抗）注射。 临床反应定义为 UC 相关症状的显着改善，缓解定义为 UC 相关症状完全消失，每个通过治疗医生。我们收集了人口统计学、临床和治疗数据。中位随访时间为 24 个月（四分位距，13-51 个月）。主要结果是 UP 对抗 TNF 治疗的临床反应。结果 总体而言，80 名患者 (77%) 对抗 TNF 治疗有临床反应，52 名患者 (50%) 达到临床缓解。肠外表现（比值比 OR，0.24；95% CI，0.08-0.7），外用类固醇持续治疗（OR，0.14；95% CI，0.03-0.73），以及外用 5-氨基水杨酸酯持续治疗（OR， 0.21；95% CI，0.07-0.62）与临床缓解的缺失显着相关。在随访期间进行内窥镜评估的患者中有 60% (38/63) 有粘膜愈合。在总人群（n = 104）中，持续临床缓解的累积概率在 1 年时为 87.6% ± 3.4%，在 2 年时为 74.7% ± 4.8%。结论 在一项对 104 名难治性 UP 患者的回顾性研究中，抗 TNF 治疗诱导了 50% 的临床缓解和 60% 的粘膜愈合。大约三分之二的患者在 2 年时仍在接受抗 TNF 治疗。