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[肝细胞疗法治疗儿童先天性代谢缺陷]

[Liver cell therapy in the treatment of inborn errors of metabolism in children].

作者信息

Pareja E, Ribes C, Gómez-Lechón M J, Cortes M, Vila J J, Dalmau J, Ibáñez V, Polo B, Castell J V, Mir J

机构信息

Unidad de Cirugía y Trasplante Hepático, Unidad de Terapia Celular Hepática, Hospital Universitario La Fe de Valencia, Valencia, España.

出版信息

An Pediatr (Barc). 2013 Dec;79(6):390.e1-8. doi: 10.1016/j.anpedi.2013.05.018. Epub 2013 Jul 27.

Abstract

Liver transplantation has been remarkably effective in the treatment of patients with end-stage liver disease. However, disparity between solid-organ supply and increased demand is the main limitation, resulting in longer waiting times and an increase in the mortality of transplant recipients. This situation creates the need to seek alternatives to orthotopic liver transplantation. Hepatocyte transplantation or liver cell transplantation has been proposed as the best method to support patients, a bridge to restore liver function or liver transplant. The procedure consists in transplanting individual cells in a recipient organ in enough quantity to survive and restore the function. The capacity of hepatic regeneration constitutes the biological basis of hepatocyte transplantation. Liver cell transplantation is carried out by means of the isolation of hepatocytes from donor liver rejected for orthotopic transplantation, to prepare a cell suspension for infusion, cryopreservation and, finally, hepatocytes are implanted into the recipient. This may be an optional therapeutic procedure in some patients with inborn errors of metabolism, fulminant hepatic failure, and acute and chronic liver failure, as a bridge to orthotopic liver transplantation. The first hepatocyte transplantation in Spain was performed in the Cell Therapy Unit of the Hospital La Fe of Valencia, creating a new research line in the transplant program.

摘要

肝移植在治疗终末期肝病患者方面成效显著。然而,实体器官供应与需求增加之间的差距是主要限制因素,导致等待时间延长以及移植受者死亡率上升。这种情况促使人们寻求原位肝移植的替代方案。肝细胞移植或肝细胞移植已被提议作为支持患者、恢复肝功能或作为肝移植桥梁的最佳方法。该手术包括将足够数量的单个细胞移植到受体器官中,使其存活并恢复功能。肝再生能力构成了肝细胞移植的生物学基础。肝细胞移植是通过从因原位移植被拒收的供体肝脏中分离肝细胞来进行的,制备细胞悬液用于输注、冷冻保存,最后将肝细胞植入受体。对于一些患有先天性代谢缺陷、暴发性肝衰竭以及急慢性肝衰竭的患者,这可能是一种可选的治疗方法,作为原位肝移植的桥梁。西班牙的首例肝细胞移植是在巴伦西亚拉费医院的细胞治疗科进行的,这在移植项目中开创了一条新的研究路线。

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