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Medical, ethical, and legal aspects of hematopoietic stem cell transplantation for Crohn's disease in Brazil.
World Journal of Stem Cells ( IF 4.1 ) Pub Date : 2020-11-13 , DOI: 10.4252/wjsc.v12.i10.1113
Milton Artur Ruiz , Roberto Luiz Kaiser Junior , Lilian Piron-Ruiz , Priscila Samara Saran , Lilian Castiglioni , Luiz Gustavo de Quadros , Tainara Souza Pinho , Richard K Burt

Crohn's disease (CD) is a chronic inflammatory bowel disease that can affect any part of the gastrointestinal tract. The etiology of CD is unknown; however, genetic, epigenetic, environmental, and lifestyle factors could play an essential role in the onset and establishment of the disease. CD results from immune dysregulation due to loss of the healthy symbiotic relationship between host and intestinal flora and or its antigens. It affects both sexes equally with a male to female ratio of 1.0, and its onset can occur at any age, but the diagnosis is most commonly observed in the range of 20 to 40 years of age. CD diminishes quality of life, interferes with social activities, traumatizes due to the stigma of incontinence, fistulae, strictures, and colostomies, and in severe cases, affects survival when compared to the general population. Symptoms fluctuate between periods of remission and activity in which complications such as fistulas, strictures, and the need for bowel resection, surgery, and colostomy implantation make up the most severe aspects of the disease. CD can be progressive and the complications recurrent despite treatment with anti-inflammatory drugs, corticosteroids, immunosuppressants, and biological agents. However, over time many patients become refractory without treatment alternatives, and in this scenario, hematopoietic stem cell transplantation (HSCT) has emerged as a potential treatment option. The rationale for the use of HSCT for CD is anchored in animal studies and human clinical trials where HSCT could reset a patient's immune system by eliminating disease-causing effector cells and upon immune recovery increase regulatory and suppressive immune cells. Autologous HSCT using a non-myeloablative regimen of cyclophosphamide and anti-thymocyte globulin without CD34+ selection has been to date the most common transplant conditioning regimen adopted. In this review we will address the current situation regarding CD treatment with HSCT and emphasize the medical, ethical, and legal aspects that permeate the procedure in Brazil.

中文翻译:

巴西克罗恩病造血干细胞移植的医学,伦理和法律方面。

克罗恩氏病(CD)是一种慢性炎症性肠病,可能会影响胃肠道的任何部分。CD的病因尚不清楚。然而,遗传,表观遗传,环境和生活方式因素可能在该疾病的发作和建立中起重要作用。CD是由于宿主和肠道菌群及其抗原之间健康的共生关系丧失而导致的免疫失调所致。男女之比为1.0时,它对男女的影响均相同,其发作可发生在任何年龄,但最常见的诊断是20至40岁。CD会降低生活质量,干扰社交活动,因大小便失禁,瘘管,狭窄和口交而蒙受创伤,在严重的情况下,与普通人群相比会影响生存。症状在缓解期和活动期之间波动,其中并发症如瘘管,狭窄,需要肠切除,手术和结肠造口植入术构成该病最严重的方面。尽管使用抗炎药,皮质类固醇,免疫抑制剂和生物制剂进行治疗,但CD可能是进行性的,并发症会复发。但是,随着时间的流逝,许多患者在没有其他治疗方法的情况下变得难治,在这种情况下,造血干细胞移植(HSCT)已成为一种潜在的治疗选择。将HSCT用于CD的基本原理扎根于动物研究和人类临床试验中,其中HSCT可以使患者 通过消除引起疾病的效应细胞和免疫恢复后的免疫系统,可增加调节性和抑制性免疫细胞。迄今为止,使用环磷酰胺的非清髓性方案和未选择CD34 +的抗胸腺细胞球蛋白的自体HSCT已成为最常用的移植条件疗法。在这篇综述中,我们将介绍HSCT治疗CD的当前情况,并强调渗透到巴西手术中的医学,道德和法律方面。
更新日期:2020-11-14
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