沙利度胺在英夫利昔单抗和阿达木单抗治疗抵抗的儿童克罗恩病患者中的应用及结局。
Thalidomide use and outcomes in pediatric patients with Crohn disease refractory to infliximab and adalimumab.
机构信息
Section of Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of Chicago Comer Children's Hospital, Chicago, IL, USA.
出版信息
J Pediatr Gastroenterol Nutr. 2012 Jan;54(1):28-33. doi: 10.1097/MPG.0b013e318228349e.
OBJECTIVE
The aim of the study was to evaluate thalidomide as rescue therapy for pediatric patients with severe refractory Crohn disease (CD) who failed to respond to antitumor necrosis factor (TNF) biologic agents.
PATIENTS AND METHODS
A computerized database was used to identify children with CD who had failed conventional immunosuppression therapy and received thalidomide rescue therapy. Twelve patients, mean age at diagnosis 10 years, were identified. Eight children had disease localized to the ileum and colon and 4 to the gastroduodenal area and colon. Five cases were complicated by strictures and 7 by fistulae. Previous drug therapy included azathioprine/6-mercaptopurine (11/12), methotrexate (7/12), and anti-TNF biologics (12/12). Outcome measures were Harvey-Bradshaw Index, change in prednisone dose, hospitalizations, bowel resections, and incision and drainage procedures. Laboratory evaluations were calculated before and after 1 to 6 months of thalidomide.
RESULTS
Mean Harvey-Bradshaw Index score improved from 11.8 to 3.9 (P = 0.0004), mean prednisone dose decreased from 13.9 to 2.3 mg/day (P = 0.001), mean number of hospitalizations decreased from 6.3 to 1.3 (P = 0.002), and erythrocyte sedimentation rate decreased from 35 to 14 mm/h (P = 0.02). The surgery rate pre-thalidomide was 0.031 and on thalidomide was 0.004. Of the 7 patients with fistulae, 5 had complete fistula closure, 1 had partial closure, and 1 showed no improvement. Adverse reactions that resulted in discontinuation of thalidomide are as follows: 42% peripheral neuropathy, 17% worsening of the CD, 8% dizziness, and 8% allergic reaction. All 5 patients who developed peripheral neuropathy had clinical resolution of the neurologic symptoms within 2 to 3 months after stopping thalidomide.
CONCLUSIONS
Thalidomide is a potentially effective rescue therapy for severe refractory CD in children who fail to respond to anti-TNF medications.
目的
本研究旨在评估沙利度胺作为抗肿瘤坏死因子（TNF）生物制剂治疗失败的重度难治性克罗恩病（CD）患儿的挽救疗法。
患者和方法
使用计算机数据库来确定接受沙利度胺挽救治疗的 CD 患儿。共鉴定出 12 名患儿，诊断时平均年龄为 10 岁。8 例患儿病变局限于回肠和结肠，4 例病变局限于胃十二指肠和结肠。5 例伴有狭窄，7 例伴有瘘管。既往药物治疗包括硫唑嘌呤/6-巯基嘌呤（11/12）、甲氨蝶呤（7/12）和抗-TNF 生物制剂（12/12）。疗效评估指标包括 Harvey-Bradshaw 指数、泼尼松剂量变化、住院次数、肠切除术和切开引流术。在接受沙利度胺治疗 1-6 个月前后进行实验室评估。
结果
平均 Harvey-Bradshaw 指数评分从 11.8 分降至 3.9 分（P=0.0004），平均泼尼松剂量从 13.9 降至 2.3mg/天（P=0.001），平均住院次数从 6.3 降至 1.3（P=0.002），红细胞沉降率从 35 降至 14mm/h（P=0.02）。沙利度胺治疗前手术率为 0.031，治疗后为 0.004。7 例瘘管患者中，5 例完全闭合，1 例部分闭合，1 例无改善。导致沙利度胺停药的不良反应如下：42%周围神经病，17% CD 加重，8%头晕，8%过敏反应。所有 5 例出现周围神经病的患者在停用沙利度胺后 2-3 个月内神经系统症状均得到临床缓解。
结论
沙利度胺是一种潜在有效的挽救疗法，可用于治疗抗肿瘤坏死因子药物治疗失败的重度难治性 CD 患儿。
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