Malik M C, Touraine J L, Perrot H, Traeger J
Proc Eur Dial Transplant Assoc. 1979;16:413-8.
Two patients, 33 and 26 years old, presented with Fabry's disease and minimal renal involvement. They were treated with a transplant of foetal liver cells exhibiting normal enzymatic activities, after plasma transfusions and symptomatic therapies had proved ineffective. In the first patient, objective and subjective clinical symptoms were significantly improved: sweating appeared, cutaneous lesions seemed slightly decreased and pains disappeared. In the second patient, pains were also seemingly decreased. The mechanism which may be held responsible for improvement of our patients, as of recipients of a kidney transplant, is not completely elucidated. The cells, rather than steroids or azathioprine, seemed to be responsible for the improvement.
两名患者，年龄分别为33岁和26岁，患有法布里病且肾脏受累程度轻微。在输血和对症治疗均无效后，他们接受了具有正常酶活性的胎儿肝细胞移植。在第一名患者中，客观和主观临床症状均有显著改善：出汗出现，皮肤病变似乎略有减轻，疼痛消失。在第二名患者中，疼痛似乎也有所减轻。与肾移植受者一样，可能导致我们患者病情改善的机制尚未完全阐明。似乎是这些细胞而非类固醇或硫唑嘌呤导致了病情改善。
