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纳他珠单抗在受限配给项目中用于克罗恩病患者的临床经验

Clinical experience of natalizumab in Crohn's disease patients in a restricted distribution program.

作者信息

Chen Chien-Huan, Kularatna Gowri, Stone Christian D, Gutierrez Alexandra M, Dassopoulos Themistocles

机构信息

Division of Gastroenterology, Department of Internal Medicine, Washington University School of Medicine, St. Louis, Missouri (Chien-Huan Chen, Gowri Kularatna, Alexandra M. Gutierrez, Themistocles Dassopoulos), U.S.A.

出版信息

Ann Gastroenterol. 2013;26(3):189-190.

Abstract

BACKGROUND

Natalizumab (NAT) is a humanized monoclonal antibody against a4-integrin initially approved for the treatment of multiple sclerosis, and then withdrawn from the market in 2005 due to the risk of progressive multifocal leukoencephalopathy. NAT was approved for the treatment of Crohn's disease in the United States in 2008 under a restricted distribution program. There has been limited data on NAT since then. The purpose of this study was to review the experience with NAT in Crohn's disease patients at a tertiary inflammatory bowel disease center.

METHODS

A retrospective chart review was performed on all patients who received NAT for treatment of refractory Crohn's disease from January 2008 to August 2010 at Washington University Medical Center in St. Louis.

RESULTS

A total of 20 patients were identified and included in our study. Four patients did not complete induction therapy. Seven patients had a clinical response, with 5 patients continuing treatment up to 2012. Four patients had a partial response, 3 had adverse events, and 2 experienced loss of response. Two patients were pregnant while on NAT, and neither had significant adverse pregnancy outcomes. One patient dependent on total parenteral nutrition developed recurrent line sepsis while on NAT. Of the 5 patients on long-term maintenance therapy, 4 have a positive anti-JC virus antibody. No patients developed progressive multifocal leukoencephalopathy or other neurological complications.

CONCLUSION

NAT remains a valuable alternative treatment option for patients with refractory Crohn's disease under a restricted distribution program.

摘要

背景

那他珠单抗(NAT)是一种抗α4整合素的人源化单克隆抗体,最初被批准用于治疗多发性硬化症,后因存在进行性多灶性白质脑病风险于2005年退市。2008年,NAT在美国通过一项受限配给计划被批准用于治疗克罗恩病。自那时起,关于NAT的数据有限。本研究的目的是回顾在一家三级炎症性肠病中心使用NAT治疗克罗恩病患者的经验。

方法

对2008年1月至2010年8月在圣路易斯华盛顿大学医学中心接受NAT治疗难治性克罗恩病的所有患者进行回顾性病历审查。

结果

共确定20例患者纳入我们的研究。4例患者未完成诱导治疗。7例患者有临床反应,其中5例患者持续治疗至2012年。4例患者有部分反应,3例出现不良事件,2例出现反应丧失。2例患者在使用NAT期间怀孕,均未出现重大不良妊娠结局。1例依赖全胃肠外营养的患者在使用NAT期间发生反复的导管败血症。在5例长期维持治疗的患者中,4例抗JC病毒抗体呈阳性。无患者发生进行性多灶性白质脑病或其他神经系统并发症。

结论

在受限配给计划下,NAT仍然是难治性克罗恩病患者的一种有价值的替代治疗选择。

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