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用于难治性克罗恩病的自体造血干细胞

Autologous hematopoietic stem cells for refractory Crohn's disease.

作者信息

DiNicola C A, Zand A, Hommes D W

机构信息

a Department of Medicine , UCLA Center for Inflammatory Bowel Diseases , Los Angeles , CA , USA.

b Vatche & Tamar Manoukian Divison of Digestive Diseases , University of California , Los Angeles , CA , USA.

出版信息

Expert Opin Biol Ther. 2017 May;17(5):555-564. doi: 10.1080/14712598.2017.1305355. Epub 2017 Mar 22.

Abstract

Autologous hematopoietic stem cells are gaining ground as an effective and safe treatment for treating severe refractory Crohn's disease (CD). Autologous hematopoietic stem cell therapy (AHSCT) induces resetting of the immune system by de novo regeneration of T-cell repertoire and repopulation of epithelial cells by bone-marrow derived cells to help patients achieve clinical and endoscopic remission. Areas covered: Herein, the authors discuss the use of AHSCT in treating patients with CD. Improvements in disease activity have been seen in patients with severe autoimmune disease and patients with severe CD who underwent AHSCT for a concomitant malignant hematological disease. Clinical and endoscopic remission has been achieved in patients treated with AHSCT for CD. The only randomized trial published to date, the ASTIC Trial, did not support further use of AHSCT to treat CD. Yet, critics of this trial have deemed AHSCT as a promising treatment for severe refractory CD. Expert opinion: Even with the promising evidence presented for HSCT for refractory CD, protocols need to be refined through the collaboration of GI and hemato-oncology professionals. The goal is to incorporate safe AHSCT and restore tolerance by delivering an effective immune 'cease fire' as a treatment option for severe refractory CD.

摘要

自体造血干细胞作为治疗重度难治性克罗恩病(CD)的一种有效且安全的疗法正逐渐得到认可。自体造血干细胞疗法(AHSCT)通过T细胞库的重新生成以及骨髓来源细胞对上皮细胞的再填充来诱导免疫系统重置,从而帮助患者实现临床和内镜缓解。涵盖领域:在此,作者们讨论了AHSCT在治疗CD患者中的应用。患有严重自身免疫性疾病的患者以及因并发恶性血液病而接受AHSCT的重度CD患者,其疾病活动度均有改善。接受AHSCT治疗的CD患者已实现临床和内镜缓解。迄今为止发表的唯一一项随机试验,即ASTIC试验,并不支持进一步使用AHSCT来治疗CD。然而,该试验的批评者认为AHSCT是重度难治性CD的一种有前景的治疗方法。专家观点:即便有关于HSCT治疗难治性CD的有力证据,但仍需胃肠病学和血液肿瘤学专业人员合作来完善方案。目标是纳入安全的AHSCT,并通过实现有效的免疫“停火”来恢复耐受性,将其作为重度难治性CD的一种治疗选择。

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