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Cx601(达伐司特罗细胞)治疗肛周瘘管型克罗恩病。

Cx601 (darvadstrocel) for the treatment of perianal fistulizing Crohn's disease.

机构信息

a Department of Abdominal Surgery , University Hospitals Leuven, KU Leuven , Leuven , Belgium.

b Department of Gastroenterology and Hepatology , University Hospitals Leuven, KU Leuven , Leuven , Belgium.

出版信息

Expert Opin Biol Ther. 2019 Jul;19(7):607-616. doi: 10.1080/14712598.2019.1623876. Epub 2019 Jun 3.

DOI:10.1080/14712598.2019.1623876
PMID:31121104
Abstract

: Given the well-documented difficulty to treat perianal fistulizing Crohn's disease (pCD), with 40% of patients experiencing recurrence even after reiterative surgery and advanced medical therapy, research in this field has focused on the role of mesenchymal stem cells (MSC). : The aim of this article is to furnish an overview of the pathogenetic mechanisms, clinical applications and evidences for the use of MSC for pCD with particular focus on adipose-derived allogenic MSC including darvadstrocel. : The effect of MSC on fistula healing is probably mediated by their anti-inflammatory properties more than by their ability to engraft and trans-differentiate in the healthy tissue. A holistic treatment of pCD, addressing different pathophysiological factors, may represent the key for an improvement in the healing rate. In this setting, MSC might play a role as 'augmentation' therapy in combination with more conventional treatments. Whether MSC have benefit in non-complex fistula in biological naïve patients, in complex fistula with many tracts and/or in rectovaginal fistulas, are unexplored fields that need further investigation. A central registry of pCD patients undergoing treatment with MSC should be created in order to elucidate the efficacy, safety and costs of stem cells treatment on long term follow up.

摘要

鉴于肛周瘘管克罗恩病(pCD)的治疗难度很大,有 40%的患者即使经过反复手术和先进的药物治疗后仍会复发,因此该领域的研究集中在间充质干细胞(MSC)的作用上。本文旨在概述 MSC 治疗 pCD 的发病机制、临床应用和证据,特别关注脂肪来源的同种异体 MSC,包括 darvadstrocel。MSC 对瘘管愈合的影响可能是通过其抗炎特性介导的,而不是通过其在健康组织中植入和转分化的能力介导的。全面治疗 pCD,解决不同的病理生理因素,可能是提高愈合率的关键。在这种情况下,MSC 可能作为“增强”治疗与更传统的治疗方法联合使用。MSC 是否对生物初治患者的非复杂性瘘管、多瘘管的复杂性瘘管和/或直肠阴道瘘有获益,这些都是尚未探索的领域,需要进一步研究。应该创建一个 pCD 患者接受 MSC 治疗的中央登记处,以便在长期随访中阐明干细胞治疗的疗效、安全性和成本。

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