The effect of tamoxifen in intermediate alpha 1-antitrypsin deficiency associated with the phenotype PiSZ.
Three patients, one with lung disease and two with liver disease, with intermediate alpha 1-antitrypsin (AAT) deficiency phenotype PiSZ, (plasma AAT concentration approximately 1/3 of normal) were treated with the anti-oestrogen drug, tamoxifen, for 2-24 months. Patients responded with 50-70% increases in plasma AAT concentrations. Progress of emphysema appeared to be retarded in one case but no improvement in liver function was recorded in the other two patients.
D000368:Aged; D004417:Dyspnea / Q000097:blood / Q000188:drug therapy*; D005260:Female; D006801:Humans; D008107:Liver Diseases / Q000097:blood / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D010641:Phenotype; D013629:Tamoxifen / Q000627:therapeutic use*; D019896:alpha 1-Antitrypsin Deficiency
D013629:Tamoxifen