Liver Transplantation for alpha-One-Antitrypsin Deficiency

Abstract

Purpose: We report the outcomes of liver transplantation (LT) for alpha-1-antitrypsin deficiency from a single transplant program. Methods: A retrospective review of the Organ Transplant Database at the University of Nebraska Medical Center was performed to identify all patients (pts) transplanted for AAT deficiency between 1990–2005. Pts were diagnosed with AAT deficiency if 3 of the following 4 criteria were present: depressed serum levels of AA; ZZ phenotype; liver biopsy and/or explant histopathology confirming the presence of diastase resistant AAT globules. The following outcomes were recorded: age; sex; prevalence of hepatoma; hepatic artery thrombosis (HAT); biliary complications; episodes of rejection and infection; post transplant lymphoproliferative disease (PTLD); graft and patient survival. Results: 35 pts were transplanted for AAT deficiency with a median age of 8 mths. The majority of AAT pts were male (77%) under the age of 18 yrs (76%) and received a cadaver liver (74%). Incidental hepatoma was present in 2 explants and recurred in 1 pt. HAT developed in 4 pts (8.3%) and biliary complications (leaks, strictures and /or necrosis) occurred in 9 pts (26%). Acute rejection occurred in 21 pts (60%) and chronic rejection occurred in 7 pts (20%). PTLD occurred in 7 pts (20%). Eight pts died, with sepsis accounting for cause of death in 6 pts (75%). Infection was seen in 77% of pts. Mean follow up was 7.5 years. Estimates of overall survival were based on the Kaplan Meier Method. Cumulative incidence methods were used to estimate the incidence of graft failure (GF). Outcome information was available for 32 of 35 patients. 1 year Overall Survival (OAS) was 77% (95% CI 57–88%). 3 year OAS is 72% (95% CI 52–85%). There was no difference in OAS when the patients were categorized by the number of complications (p = 0.7). Pts < 18 years tend to have improved estimates of OAS compared to pts > than 18 yrs (p = 0.087). Estimated cumulative incidence of GF at 1 year is 6% (95% CI 0–17%) and at 7 years 13% (95% CI 0- 34%). Conclusions: In this predominantly male, pediatric cohort of LT pts with AAT, long term survival is approximately 72%. Pediatric pts tend to have a better OAS than adult pts. The number of post transplant complications does not appear to be a predictor of outcome. Sepsis was the most common cause of death. Long term results of LT for AAT deficiency are otherwise encouraging with outcomes comparable to pts undergoing LT for other indications