An open-label pilot study of low-dose thalidomide in chronically active, steroid-dependent Crohn's disease
An open-label pilot study of low-dose thalidomide in chronically active, steroid-dependent Crohn's disease
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DOI：
10.1016/s0016-5085(99)70277-5
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发表时间：
1999-12-01
期刊：
影响因子：
29.4
通讯作者：
Targan, SR
中科院分区：
文献类型：
--
作者：
Vasiliauskas, EA;Kam, LY;Targan, SR
背景与目的：沙利度胺降低肿瘤坏死因子α的产生，这是一种与克罗恩病（CD）相关的促炎细胞因子。在这项研究中，评估了低剂量沙利度胺治疗中度至重度类固醇依赖性CD的安全性、耐受性和有效性。方法：12名成年男性克罗恩病活动性指数（CDAI）评分大于或等于250，小于或等于500，尽管大于或等于20mg泼尼松/天。前6例患者每晚给予50 mg沙利度胺，后6例每晚给予100 mg沙利度胺。类固醇剂量在治疗的前4周是稳定的，然后在5-12周逐渐减少，CDAI用于评估反应。结果：(1)在第1-4周，所有患者的疾病活动度持续改善：58%缓解，17%缓解；(2)尽管在第5-12周类固醇逐渐减少，但临床改善总体上保持不变，所有患者能够减少大于或等于50%的类固醇，44%的患者完全停止使用类固醇。在5-12周，70%的患者有反应，20%的患者缓解。(3)副作用轻微且大多是短暂的，最常见的是嗜睡、周围神经病变、水肿和皮炎。结论：低剂量沙利度胺在12周内耐受性良好且有效。这项初步研究的结果支持了沙利度胺治疗乳糜泻的多中心对照试验的必要性。
Background & Aims: Thalidomide decreases production of tumor necrosis factor alpha, a proinflammatory cytokine associated with Crohn's disease (CD). In this study the safety, tolerance, and efficacy of low-dose thalidomide were evaluated for treatment of moderate-to-severe, steroid-dependent CD. Methods: Twelve adult male patients with Crohn's Disease Activity Index (CDAI) scores of greater than or equal to 250 and less than or equal to 500 despite greater than or equal to 20 mg prednisone/day were enrolled. The first 6 patients received 50 mg thalidomide every night, the next 6 received 100 mg every night. Steroid doses were stable during the first 4 weeks of treatment, then tapered during weeks 5-12, CDAI was used to assess response. Results: (1) Disease activity improved consistently in all patients during weeks 1-4: 58% response, 17% remission, (2) Clinical improvement was generally maintained despite steroid taper during weeks 5-12, All patients were able to reduce steroids by greater than or equal to 50%, Forty-four percent discontinued steroids entirely. In weeks 5-12, 70% of patients responded and 20% achieved remission, (3) Side effects were mild and mostly transient, with the most common being drowsiness, peripheral neuropathy, edema, and dermatitis, Conclusions: Low-dose thalidomide appears to be well tolerated and effective over a 12-week period. Results of this pilot study support the need for controlled multicenter trials of thalidomide for treatment of CD.