Thalidomide induces clinical remission and mucosal healing in adults with active Crohn's disease: a prospective open-label study.
Thalidomide induces clinical remission and mucosal healing in adults with active Crohn's disease: a prospective open-label study.
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沙利度胺可诱导患有活动性克罗恩病的成人的临床缓解和粘膜愈合：一项前瞻性开放标签研究。
DOI：
10.1177/1756283x17698910
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发表时间：
2017-05
影响因子：
4.2
通讯作者：
Chen MH
中科院分区：
文献类型：
--
作者：
He Y;Mao R;Chen F;Xu PP;Chen BL;Wu Y;Qiu Y;Zhang SH;Feng R;Zeng ZR;Ben-Horin S;Chen MH
沙利度胺可有效诱导和维持难治性克罗恩病（CD）儿童和青少年的临床缓解。然而，沙利度胺对成年CD患者的疗效和安全性知之甚少。我们在2013年1月至2015年4月进行了一项前瞻性开放标签队列研究。共有47名对皮质类固醇和/或免疫调节剂或生物制剂依赖/耐药或不耐受的成年活动性CD患者每天接受50 - 100mg沙利度胺治疗。主要结局是在第8周评估临床缓解。在第24周进行内镜评估，并定义为内镜下反应（克罗恩病内镜严重程度指数[CDEIS]评分从基线CDEIS 6或更高降低bbb50分），内镜下完全缓解（CDEIS评分< 3）和粘膜愈合（MH）（无溃疡）。共有47名患有活动性乳糜泻的成年人被纳入研究。在所有纳入的47例患者中，临床缓解率在第4周和第8周分别为14.9%和23.4%，但在第12周和第24周分别增加到46.8%和53.2%（意向治疗分析）。共有32名患者同意并在第24周接受回肠结肠镜检查。内镜下缓解率和完全缓解率分别为68.4%和43.8%。28.1%的患者达到了MH（无溃疡）。不良事件发生在27/47（57.4%）的患者中，但只有5/47（10.6%）的患者需要停药。对于成年活动性乳糜泻患者，低剂量沙利度胺诱导和维持临床缓解是有效且耐受的，但沙利度胺诱导临床缓解的最佳时间可能比之前认为的要长，最佳时间可能是12周。沙利度胺可以合理地实现MH。
Thalidomide is effective in inducing and maintaining clinical remission in children and adolescents with refractory Crohn’s disease (CD). However, little is known about the efficacy and safety of thalidomide for adult patients with CD. We conducted a prospective open-label cohort study between January 2013 and April 2015. A total of 47 adult patients with active CD who were dependent/resistant or intolerant to corticosteroids and/or immunomodulators or biologics received 50–100 mg of thalidomide daily. Primary outcome was clinical remission evaluated at week 8. Endoscopic assessment was performed at week 24 and defined as endoscopic response (decrease in Crohn’s Disease Endoscopic Index of Severity [CDEIS] score > 5 points from baseline CDEIS of 6 or more), complete endoscopic remission (CDEIS score < 3), and mucosal healing (MH) (no ulceration). A total of 47 adults with active CD were enrolled. The clinical remission rate was 14.9% and 23.4% at week 4 and week 8, but increased to 46.8% at week 12 and 53.2% at week 24 out of all the 47 patients included (intention-to-treat analysis). Altogether 32 patients consented and underwent ileocolonoscopy at week 24. The rate of endoscopic response and complete endoscopic remission were 68.4% and 43.8%. MH (no ulceration) was achieved in 28.1% of patients. Adverse events occurred in 27/47 (57.4%) patients but necessitated therapy discontinuation in only 5/47 (10.6%) of patients. Low-dose thalidomide was effective and tolerated for inducing and maintaining clinical remission in adult patients with active CD, but the optimal time frame for thalidomide to induce clinical remission may be longer than previously appreciated and is probably optimal at 12 weeks. MH could reasonably be achievable with thalidomide.