Thalidomide therapy for patients with refractory Crohn's disease: An open-label trial
Thalidomide therapy for patients with refractory Crohn's disease: An open-label trial
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DOI：
10.1016/s0016-5085(99)70276-3
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发表时间：
1999-12-01
期刊：
影响因子：
29.4
通讯作者：
Hanauer, SB
中科院分区：
文献类型：
--
作者：
Ehrenpreis, ED;Kane, SV;Hanauer, SB
背景和目的：抑制肿瘤坏死因子是沙利度胺抗炎特性的拟议机制。我们进行了沙利度胺治疗难治性克罗恩病的开放标签试验。方法：22 名难治性克罗恩病患者（克罗恩病活动指数 [CDAI] > 200 和/或肛周引流疾病）开始接受沙利度胺治疗，睡前 200 mg（18 名患者）或睡前 300 mg（4 名患者）。在第 0、4 和 12 周评估 CDAI 和目标间隔评分 (GIS)，管腔疾病患者的临床缓解定义为 CDAI 评分降低 > 150 分，瘘管患者 GIS 3 个参数中有 2 次评分大于或等于 1+，临床缓解定义为 GIS 的所有参数 (瘘管患者) 总 CDAI < 150（管腔患者）或大于或等于 2+患者）。结果：9 名管腔疾病患者和 13 名瘘管患者（16 名男性，6 名女性）纳入研究。入组时的中位 CDAI 评分为 371 (95-468)，16 名患者完成了 4 周的治疗（12 名临床缓解，4 名临床缓解）。完成 12 周的所有 14 名患者均符合临床反应标准。 9 名患者达到临床缓解（3 名管腔患者，6 名瘘管患者），中位 CDAI 评分为 175（30-468；与基线相比，P < 0.001）。结论：沙利度胺对某些难治性克罗恩病患者有效。
Background & Aims: Inhibition of tumor necrosis factor is a proposed mechanism for the anti-inflammatory properties of thalidomide. We performed an open-label trial of thalidomide in refractory Crohn's disease. Methods: Twenty-two patients with refractory Crohn's disease (Crohn's Disease Activity Index [CDAI] > 200 and/or draining perianal disease) initiated therapy with thalidomide, 200 mg at bedtime (18 patients), or 300 mg at bedtime (4 patients). CDAI and goal interval scores (GIS) were assessed at weeks 0, 4, and 12, Clinical response for patients with luminal disease was defined as reduction in CDAI score of > 150 points and for fistula patients was 2 scores of greater than or equal to 1+ in 3 parameters of the GIS, Clinical remission was defined as a total CDAI < 150 (luminal patients) or greater than or equal to 2+ for all parameters of the GIS (fistula patients). Results: Nine patients with luminal disease and 13 with fistulas (16 male, 6 female) were enrolled. The median CDAI score at entry was 371 (95-468), Sixteen patients completed 4 weeks of treatment (12 clinical responses, 4 clinical remissions). All 14 patients completing 12 weeks met criteria for clinical response. Nine achieved clinical remission (3 luminal, 6 fistula patients), The median CDAI score was 175 (30-468; P < 0.001 vs. baseline). Conclusions: Thalidomide is efficacious in some patients with refractory Crohn's disease.