LIVER REPLACEMENT FOR ALPHA1-ANTITRYPSIN DEFICIENCY

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  • 1 January 1977
    • journal article
    • research article
    • Vol. 81  (3) , 258-261
Abstract
A 16 yr old girl with advanced cirrhosis and severe .alpha.1-antitrypsin deficiency of the homozygous PiZZ phenotype was treated by orthotopic liver transplantation. After replacement of the liver with a homograft from a donor with the normal PiMM phenotype, the .alpha.1-antitrypsin concentration in the recipient''s serum rose to normal; it had the PiMM phenotype. Two and one third yr later, chronic rejection necessitated retransplantation. Insertion of a homograft from a heterozygous PiMZ donor was followed by the identification of that phenotype in the recipient''s serum. Neither liver graft developed the .alpha.1-antitrypsin glycoprotein deposits seen with the deficiency state. These observations confirm that this hepatic-based inborn error of metabolism is metabolically cured by liver replacement.