Zhong Min, Cui Bota, Xiang Jie, Wu Xia, Wen Quan, Li Qianqian, Zhang Faming
Medical Center for Digestive Diseases, The Second Affiliated Hospital of Nanjing Medical University, Nanjing, China.
Key Lab of Holistic Integrative Enterology, Nanjing Medical University, Nanjing, China.
Front Pharmacol. 2021 Feb 4;11:617535. doi: 10.3389/fphar.2020.617535. eCollection 2020.
Crohn's disease (CD)-related fibrotic stricture remains a clinical challenge because of no effective treatments. This study aimed to evaluate the potential efficacy of rapamycin in patients with CD-related strictures in different locations in gastrointestinal tract. A pilot prospective study on using rapamycin for CD-related stricture was performed from April 2015 to August 2020 in a single center in China. Fifteen patients were enrolled into the study. The clinical efficacy was evaluated by diet score and gastrointestinal obstruction symptoms score. Clinical responses were defined as the ability to tolerate the regular diet with vegetable fiber combined with a reduction of ≥75% in overall target score and a score of less than two points for each item. Three patients discontinued rapamycin for less than 1-month due to intolerance to adverse events, then, 12 patients received ≥1 dose of the rapamycin and provided ≥1 post-baseline target score after baseline were included for intent-to-treat (ITT) analysis. 100% (5/5) of patients with upper gastrointestinal strictures achieved clinical response after using rapamycin. However, no clinical response was observed in those patients with CD lesions in lower gastrointestinal tract. Adverse events occurred in 40% (6/15) of patients. No death or serious opportunistic infections were observed in the present study. This study firstly reported that rapamycin might be effective for CD-related stricture in the upper, but not in lower gastrointestinal tract.
克罗恩病（CD）相关的纤维化狭窄由于缺乏有效的治疗方法，仍然是一个临床挑战。本研究旨在评估雷帕霉素对胃肠道不同部位CD相关狭窄患者的潜在疗效。2015年4月至2020年8月在中国的一个单中心进行了一项关于使用雷帕霉素治疗CD相关狭窄的前瞻性初步研究。15名患者被纳入研究。通过饮食评分和胃肠道梗阻症状评分评估临床疗效。临床反应定义为能够耐受含蔬菜纤维的常规饮食，同时总体目标评分降低≥75%，且每项评分低于2分。3名患者因对不良事件不耐受而停用雷帕霉素不到1个月，随后，12名接受≥1剂雷帕霉素且在基线后提供≥1次基线后目标评分的患者被纳入意向性分析（ITT）。上消化道狭窄患者中有100%（5/5）在使用雷帕霉素后达到临床反应。然而，在下消化道有CD病变的患者中未观察到临床反应。40%（6/15）的患者发生了不良事件。本研究中未观察到死亡或严重的机会性感染。本研究首次报道雷帕霉素可能对上消化道CD相关狭窄有效，但对下消化道无效。
