ABSTRACT

Background

Efficacy and safety of ustekinumab for the treatment of ulcerative colitis (UC) has been demonstrated in clinical trials, but few real-world data are available so far. The aim of this study was to assess effectiveness and safety of ustekinumab in a cohort of refractory UC patients.

Methods

Data of patients with moderate to severe UC treated with ustekinumab were retrospectively collected. Primary endpoint was steroid-free clinical remission at weeks 24 and 52 of therapy. Secondary endpoints were treatment response, endoscopic remission, treatment persistence at 12 months and safety.

Results

A total of 68 patients [males 63%; median (range) age 42 (16–72) years] were included. Almost all patients (97%) were biologics experienced. At weeks 24 and 52, 31% and 50% of patients achieved steroid-free clinical remission, 84% and 82% had clinical response, respectively. At the end of follow-up, there was a significant reduction of pMS from baseline (p < 0.001) and of steroid use (p < 0.001). At week 52, 22% of the available endoscopies (18/38) showed mucosal healing. The probability to persist in therapy at week 52 was 87%. Only one adverse event occurred.

Conclusions

Data from our real-life cohort of refractory UC patients suggest satisfactory effectiveness and a good safety of ustekinumab.

中文翻译：

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乌司奴单抗治疗溃疡性结肠炎的一年有效性和安全性：来自意大利的多中心真实世界研究

摘要

背景

乌司奴单抗治疗溃疡性结肠炎 (UC) 的疗效和安全性已在临床试验中得到证实，但迄今为止鲜有可用的真实数据。本研究的目的是评估优特克单抗在难治性 UC 患者队列中的有效性和安全性。

方法

回顾性收集使用优特克单抗治疗的中度至重度 UC 患者的数据。主要终点是治疗第 24 周和第 52 周时无类固醇临床缓解。次要终点是治疗反应、内镜缓解、12 个月的治疗持续性和安全性。

结果

共68例[男性63%；中位（范围）年龄42（16-72）岁]被包括在内。几乎所有患者 (97%) 都有过生物制剂经验。在第 24 周和第 52 周，31% 和 50% 的患者达到无类固醇临床缓解，分别有 84% 和 82% 有临床反应。在随访结束时，pMS 与基线相比显着降低（p < 0.001）和类固醇使用（p < 0.001）。在第 52 周，22% 的可用内镜检查 (18/38) 显示粘膜愈合。在第 52 周继续治疗的概率为 87%。仅发生了一项不良事件。

结论

来自我们真实生活中难治性 UC 患者队列的数据表明，乌司奴单抗具有令人满意的有效性和良好的安全性。