沙利度胺单药维持治疗肠白塞病的长期疗效及安全性分析  

Long term efficacy and safety analysis of thalidomide monotherapy in the maintenance treatment of intestinal Behcet's disease

作　　者：黄钊鹏 晁康[1,2,3] 吴锐彬 高翔 杨庆帆[1,2,3] Huang Zhaopeng;Chao Kang;Wu Ruibin;Gao Xiang;Yang Qingfan(Department of Gastroenterology,the Sixth Affiliated Hospital,Sun Yat-sen University,Guangzhou 510655,China;Guangdong Provincial Key Laboratory of Colorectal and Pelvic Floor Diseases,the Sixth Affiliated Hospital,Sun Yat-sen University,Guangzhou 510655,China;Biomedical Innovation Center,the Sixth Affiliated Hospital,Sun Yat-sen University,Guangzhou 510000,China)

机构地区：[1]中山大学附属第六医院消化内科,广州510655 [2]广东省结直肠盆底疾病重点实验室,广州510655 [3]广州市黄埔区中六生物医学创新研究院,广州510000

出　　处：《中华炎性肠病杂志（中英文）》2023年第4期313-318,共6页Chinese Journal of Inflammatory Bowel Diseases

基　　金：国家临床重点专科;广东省消化系统疾病临床医学研究中心项目(2020B1111170004)。

摘　　要：目的评估沙利度胺单药维持治疗肠白塞病的疗效、预测因素及安全性。方法回顾性纳入2013年1月至2022年3月于中山大学附属第六医院接受沙利度胺单药维持治疗且规律随访的肠白塞病患者,进行疗效、预测因素及安全性分析。主要观察终点为肠白塞病患者治疗第6个月、12个月的无激素维持缓解率;次要终点为治疗后6个月、12个月的黏膜愈合率、沙利度胺长期无激素维持缓解率、沙利度胺疗效的预测因素及安全性。结果共纳入28例患者,沙利度胺治疗第6个月、12个月、第24个月、第36个月无激素维持缓解率分别为85.7%、74.0%、63.5%和50.4%;无激素维持缓解的中位时长为49.5(35.4,63.6)个月。治疗第6个月和12个月的黏膜愈合率为40.0%和35.0%。多因素COX回归分析发现,基线肠白塞病疾病活动指数水平较高(P=0.014,HR=1.057,95%CI 1.011~1.104)是沙利度胺未能维持无激素缓解的独立危险因素。随访期间,14例患者出现不良反应,总体不良反应发生率为50.0%,其中肢体麻木发生率为28.6%,发生中位时长为9.5(5.5,22.5)个月。不良反应导致药物减量率为14.3%,停药率为17.9%,停药中位时长为10.5(23.5,40.3)个月。结论沙利度胺能有效长期维持肠白塞病无激素缓解,但不良反应如肢体麻木的发生率较高值得关注。Objective To evaluate the efficacy,predictive factors,and safety of thalidomide monotherapy in the maintenance treatment of intestinal Behcet's disease.Methods From January 2013 to March 2022,patients with intestinal Behcet's disease received thalidomide in the Sixth Affiliated Hospital of Sun Yat-sen University were retrospectively enrolled for analysis.The primary end points were the maintained steroid-free remission at 6 and 12 months.The secondary end points were the mucosal healing at 6 and 12 months,the long-term maintained steroid-free remission,predictive factors and adverse events.Results A total of 28 patients with active intestinal Behcet's disease were enrolled.The maintained steroid-free remission at month 6,12,24 and 36 were 85.7%,74.0%,63.5%and 50.4%respectively.The median duration of maintained steroid-free remission was 49.5(35.4,63.6)months.Mucosal healing was 40.0%and 35.0%at month 6 and 12 respectively.Multivariate Cox regression analysis indicated that high disease activity index for intestinal Behcet's disease at baseline(P=0.014,HR=1.057,95%CI 1.011-1.104)was independently associated with lower likelihoods of maintaining steroid-free remission.During follow-up,14 patients(50.0%)got adverse reactions and the incidence of limb numbness was 28.6%,with a median duration of 9.5(5.5,22.5)months.The drug dosage reduction rate was 14.3%,and the drug discontinuation rate was 17.9%due to side effects respectively.The median duration of drug discontinuation was 10.5(23.5,40.3)months.Conclusion Thalidomide shows good effectiveness in maintaining long term steroid-free remission in intestinal Behcet′s disease,but the high incidence of limb numbness deserves attention.

关 键 词：肠白塞病 沙利度胺 长期疗效 安全性 

分 类 号：R969[医药卫生—药理学]