沙利度胺治疗成人难治性溃疡性结肠炎11例疗效分析

Efficacy and safety of thalidomide in the treatment of adult refractory ulcerative colitis

目的评价沙利度胺对成人难治性溃疡性结肠炎(UC)的有效性和安全性。方法对2009年11月至2017年11月间北京协和医院消化内科接受沙利度胺治疗的所有成人UC患者进行回顾性分析,对患者的临床特点,沙利度胺的使用剂量、疗程、合并用药、临床疗效及不良反应进行评估。结果 11例难治性UC患者中,2例为重度活动,3例为中度活动,5例为轻度活动,1例为缓解期。10例活动期患者经沙利度胺治疗后,5例临床缓解,1例临床有效,3例无效,1例因不良反应停药。7例缓解期的患者中,6例为沙利度胺诱导缓解,1例为前期其他方案诱导缓解,除1例失访、1例维持缓解失败,其余5例均能维持缓解,且内镜评估均达到黏膜愈合。8例患者出现不良反应,包括手足麻木、便秘、嗜睡、头晕、皮疹、肝功能异常及多汗。1例患者因无法耐受嗜睡和头晕症状而停药。结论沙利度胺对难治性UC具有一定疗效,可用于诱导和维持临床缓解和黏膜愈合,但治疗时应密切监测和随访不良反应。

Objective To evaluate the efficacy and safety of thalidomide in the treatment of refractory ulcerative colitis （UC）. Methods Adult patients with refractory UC and treated with thalidomide from Nov. 2009 to Nov. 2017 were included. Their clinical records were reviewed. Results Among the 11 refractory UC patients, 2 were severely active, 3 were moderately active, 5 were mildly active, and 1 was in remission. After treatment with thalidomide, the clinical remission, effective and ineffective rates were 50.0%（5/10）, 10.0%（1/10）, and 30.0%（3/10） respectively. One patient had thalidomide stopped because of adverse reactions （ADR）. Among the 7 patients in remission, one lost to follow up, 5 out of 6 patients achieved clinical remission could maintain remission during the observation period of 30 to 56 months, and all of them achieved mucosal healing. Eight out of 11 （72.7%） patients had ADRs, including numbness of hands and feet, conspitation, somnolence, dizziness, rash, liver function abnormity, and sweatiness. Conclution Thalidomide can induce and maintain clinical remission and also achieve mucosal healing in adult refractory UC. However, its ADRs should be dosely monitored during treatment.