Sokal Etienne M, Smets Françoise, Bourgois Annick, Van Maldergem Lionel, Buts Jean-Paul, Reding Raymond, Bernard Otte Jean, Evrard Veerle, Latinne Dominique, Vincent Marie Françoise, Moser Anne, Soriano Humberto E
Cliniques St Luc, Université Catholique de Louvain, Brussels, Belgium.
Transplantation. 2003 Aug 27;76(4):735-8. doi: 10.1097/01.TP.0000077420.81365.53.
Hepatocyte transplantation is an investigational alternative to orthotopic liver transplantation to treat liver based inborn errors of metabolism. We report successful hepatocyte transplantation in a 4-year-old girl with infantile Refsum disease. Hepatocytes were isolated from the left liver segment of two male donors using a classic two-step perfusion method. Fresh cells were transplanted first and then cryopreserved cells, for a total of 2 billion cells. Total bile acids and abnormal dihydroxycoprostanoïc acid markedly decreased in the patient's serum, indicating resolution of cholestasis and re-population of liver cells. Pipecholic acid decreased by 40% and c26:c22 fatty acid ratio by 36% after 18 months. Donor chromosomes sequences were detected on biopsy posttransplant, indicating engraftment. Hepatocyte transplantation is a safe and promising technique in the treatment of rare inborn errors of metabolism. Future improvements of cell viability and prevention of apoptosis may increase engraftment and subsequent re-population.
肝细胞移植是一种用于治疗基于肝脏的先天性代谢缺陷的原位肝移植的研究性替代方法。我们报告了在一名患有婴儿型Refsum病的4岁女孩中成功进行肝细胞移植的案例。使用经典的两步灌注法从两名男性供体的左肝段分离肝细胞。首先移植新鲜细胞，然后移植冷冻保存的细胞，总共移植20亿个细胞。患者血清中的总胆汁酸和异常二羟基胆甾烷酸明显降低，表明胆汁淤积得到缓解且肝细胞重新增殖。18个月后，鹅去氧胆酸降低了40%，c26:c22脂肪酸比率降低了36%。移植后活检检测到供体染色体序列，表明植入成功。肝细胞移植是治疗罕见先天性代谢缺陷的一种安全且有前景的技术。未来提高细胞活力和预防细胞凋亡可能会增加植入率及随后的重新增殖。
