Introduction: We examined the long-term outcome of transplantation for alpha 1-antitrypsin deficiency (A1ATD). Method: Data were reviewed on 42 transplants in 35 children with A1ATD over 42yr and compared with 129 transplants in 116 children with biliary atresia (BA). Results: Over 50% of patients were followed up for >10yr. A1ATD were older than BA at transplantation, median age, 6.0 vs. 1.0yr (p<0.0001), and transplanted earlier in the course of liver failure (total bilirubin, 2.7mg/dL [1.4-6.9] vs. 9.7mg/dL [2.9-15.4], p=0.005). Patient survival was greater in A1ATD than BA: one-yr post-transplant, 82.7% vs. 67.9%; fiveyr, 76.5% vs. 60.2%; and 10yr, 76.5% vs. 55.9% (p=0.03). Death-censored graft survival was similar: one-yr post-transplant, 68.4% vs. 66.2%; fiveyr, 68.4% vs. 55.8%; and 10yr, 68.4% vs. 52.5% (p=0.2). Deaths were from infection, hemorrhage, and graft failure <6months post-transplant. Patient survival improved at fiveyr from 33.3% pre-cyclosporine (CSA) (1969-1984) (n=6) to 76.5% in the CSA era (1985-1994) (n=17) and 100% with tacrolimus (1995-2006) (n=12) (p=0.007). Conclusions: The age at transplantation and the degree of liver dysfunction were related to the differences in graft and patient survival between A1AT and BA.
- Alpha 1-antitrypsin deficiency
- Biliary atresia
- Liver transplantation
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