Hematopoietic Stem Cell Transplantation in Crohn's Disease: State-of-the-Art Treatment

Hematopoietic Stem Cell Transplantation in Crohn's Disease: State-of-the-Art Treatment
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DOI:
10.1159/000449090
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发表时间:
2017-01-01
期刊:
影响因子:
2.3
通讯作者:
Hawkey, C. J.
Hawkey, C. J.
中科院分区:
医学3区
文献类型:
--
作者:
Hawkey, C. J.

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自体和异基因造血干细胞移植(HSCT)已尝试在克罗恩病(CD)。在同种异体HSCT中,宿主骨髓被消融并被来自供体的骨髓替代。这种遗传上不同的骨髓的替代在许多情况下是有效的,包括那些具有免疫学基础的情况,如CD。虽然同种异体HSCT的毒性已经排除了其在特发性CD中的吸收,但人们对其在治疗早发性婴儿(炎性肠病)中的效用感兴趣,其表现为单基因疾病,其中白细胞介素10信号传导系统的异常是最好的识别。在自体HSCT中,在进行淋巴消融和移植患者自身的未定型干细胞之前收获患者自身的干细胞,其产生具有改变的T细胞库的免疫系统。在少数情况下,这导致了实质性和长期缓解,相当于可能治愈CD。然而,来自Autumn Stem Cell International Crohn's Disease研究的病例系列和对照数据表明,尽管这种方法有其自身的优势,但大多数患者仍有再次发生CD的风险,尽管对常规治疗的反应有所改善。CD的新治疗方法的可用性意味着HSCT由于其更大的毒性而不是大多数患者的合适治疗方法,即使疗效可能是上级。更广泛的使用将取决于更安全和更有针对性的协议的发展。(C)2017 S. Karger AG,巴塞尔
Both autologous and allogeneic haemopoietic stem cell transplantation (HSCT) have been tried in Crohn's disease (CD). In allogeneic HSCT, the host bone marrow is ablated and replaced by bone marrow from a donor. This substitution of a genetically different bone marrow is effective in a number of conditions including those with an immunological basis such as CD. While the toxicity of allogeneic HSCT has precluded its uptake in idiopathic CD, there is interest in its utility in the management of early onset infantile (inflammatory bowel disease), which behaves as a monogenic disorder, with abnormalities of the interleukin 10 signalling system as the best recognized. In autologous HSCT, the patient's own stem cells are harvested before proceeding to lymphoablation and transplantation of the patient's own uncommitted stem cells, which generate an immune system with an altered T-cell repertoire. In a limited number of cases, this has led to substantial and prolonged remission tantamount to possible cure of CD. However, case series and controlled data from the Autologous Stem Cell International Crohn's Disease study suggest that although this method has its own advantages, most patients are still at risk of redeveloping CD, albeit with an arguably improved response to conventional treatment. The availability of new treatments for CD means that an HSCT is not a suitable treatment method for a majority of patients because of its greater toxicity, even though efficacy may be superior. Wider usage would depend upon the development of protocols that are safer and better targeted. (C) 2017 S. Karger AG, Basel