Dave Maneesh, Jaiswal Palashkumar, Cominelli Fabio
aDivision of Gastroenterology and Liver Disease, University Hospitals of ClevelandbDigestive Health Research Institute, Case Western Reserve University, Cleveland, OhiocDivision of Internal Medicine, John H Stroger Jr. Hospital of Cook County, Chicago, Illinois, USA.
Curr Opin Gastroenterol. 2017 Jan;33(1):59-68. doi: 10.1097/MOG.0000000000000327.
There is a need for novel therapies for inflammatory bowel diseases (IBDs) that are well tolerated and effective. Currently, mesenchymal stem/stromal cells (MSCs) are being investigated in clinical trials for treatment of IBD. In this review, we update the recently published studies with an emphasis on the long-term efficacy of MSC therapy for IBD.
A cumulative body of data, including a recent phase III randomized controlled trial demonstrated excellent fistula healing in patients with refractory Crohn's perianal fistulae treated via local injections of MSCs and with a good safety profile. Follow-up studies suggest long-term efficacy of MSC therapy for complex perianal Crohn's disease fistulae; however, the efficacy decreases over time and may necessitate repeat treatment. Systemic (intravenous) therapy for luminal IBD offers a relatively well tolerated alternative but its efficacy remains unclear.
Recent studies demonstrate that MSCs are well tolerated and have an excellent short-term efficacy for the management of refractory fistulizing perianal Crohn's disease. The current data suggest that its influence may 'wear off' over time. More data on larger number of patients with longer duration of follow-up in the setting of a randomized placebo controlled trial are needed to confirm these promising results. For luminal IBD, there is a need for more mechanistic studies in representative preclinical murine models, and the results of an ongoing phase III randomized controlled trial are eagerly awaited.
炎性肠病（IBD）需要耐受性良好且有效的新型疗法。目前，间充质干/基质细胞（MSC）正在进行治疗IBD的临床试验研究。在本综述中，我们更新了最近发表的研究，重点关注MSC治疗IBD的长期疗效。
包括最近一项III期随机对照试验在内的大量累积数据表明，通过局部注射MSC治疗的难治性克罗恩肛周瘘患者瘘管愈合良好，且安全性良好。随访研究表明，MSC治疗复杂性肛周克罗恩病瘘管具有长期疗效；然而，疗效会随时间下降，可能需要重复治疗。腔内IBD的全身（静脉）治疗提供了一种耐受性相对良好的替代方案，但其疗效仍不明确。
最近的研究表明，MSC耐受性良好，对难治性瘘管性肛周克罗恩病的治疗具有出色的短期疗效。目前的数据表明，其影响可能会随着时间推移而“减弱”。需要在随机安慰剂对照试验中对更多患者进行更长时间随访，以证实这些有前景的结果。对于腔内IBD，需要在具有代表性的临床前小鼠模型中进行更多机制研究，并且人们急切期待正在进行的III期随机对照试验的结果。
