Foetal liver transplant in Fabry's disease.
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.
D000328:Adult; D002470:Cell Survival; D002518:Ceramides / Q000097:blood; D003404:Creatinine / Q000097:blood; D000795:Fabry Disease / Q000201:enzymology / Q000473:pathology / Q000628:therapy*; D005260:Female; D005333:Fetus; D006801:Humans; D016031:Liver Transplantation; D008297:Male; D011247:Pregnancy; D011507:Proteinuria / Q000209:etiology; D013546:Sweating; D000509:alpha-Fetoproteins; D000519:alpha-Galactosidase / Q000097:blood
D002518:Ceramides; D000509:alpha-Fetoproteins; D003404:Creatinine; D000519:alpha-Galactosidase