Outcomes for recipients of liver transplantation for alpha-1-antitrypsin deficiency-related cirrhosis

Elizabeth J. Carey, Vivek N. Iyer, Darlene R. Nelson, Justin H Nguyen, Michael Joseph Krowka

Research output: Contribution to journalArticle

22 Citations (Scopus)

Abstract

Alpha-1-antitrypsin (AAT) deficiency is a rare genetic disease caused by an abnormal production of the serine protease inhibitor AAT. Liver transplantation (LT) cures cirrhosis caused by AAT deficiency and restores the normal production of AAT. There are few reports on the post-LT outcomes of patients with AAT deficiency. The aim of this study was to determine the characteristics and outcomes of patients undergoing LT for AAT deficiency at 3 large transplant centers. All patients undergoing LT at these 3 transplant centers from 1987 to 2012 for AAT deficiency (ZZ or SZ phenotype) were included. The most recent 50 patients with the MZ phenotype were also included for comparison. Data were collected retrospectively from internal databases and medical records. Seventy-three patients (50 with the ZZ phenotype and 23 with the SZ phenotype) underwent LT. The mean age was 52.8 years, and the majority of the patients (75.6%) were men. Before LT, serum AAT levels were lower for the ZZ patients versus the SZ patients (28.3 versus 58.0 mg/dL, P < 0.001). More than 40% of the SZ patients had an additional liver disease, whereas 8% in the ZZ group and 90% in the MZ group did. Before LT, there was no significant difference in pulmonary function between the ZZ and SZ groups. Seventeen patients (all with ZZ phenotype) had pulmonary function tests performed before and after LT. The forced expiratory volume in 1 second (FEV1) continued to decline for the majority. The 1-, 3-, 5-, and 10-year post-LT survival rates were 86%, 83%, 80%, and 72%, respectively, for the ZZ patients and 91%, 86%, 79%, and 79%, respectively, for the SZ patients. In conclusion, survival after LT for patients with ZZ or SZ AAT deficiency is excellent. Despite the normalization of AAT levels after LT, FEV1 continues to decline unexpectedly after LT in some ZZ and SZ patients.

Original languageEnglish (US)
Pages (from-to)1370-1376
Number of pages7
JournalLiver Transplantation
Volume19
Issue number12
DOIs
StatePublished - Dec 2013

Fingerprint

alpha 1-Antitrypsin Deficiency
Liver Transplantation
Fibrosis
alpha 1-Antitrypsin
Phenotype
Forced Expiratory Volume
Autosomal Recessive alpha-1-Antitrypsin Deficiency
Transplants
Inborn Genetic Diseases
Serine Proteinase Inhibitors
Respiratory Function Tests
Rare Diseases

ASJC Scopus subject areas

  • Surgery
  • Transplantation
  • Hepatology

Cite this

Outcomes for recipients of liver transplantation for alpha-1-antitrypsin deficiency-related cirrhosis. / Carey, Elizabeth J.; Iyer, Vivek N.; Nelson, Darlene R.; Nguyen, Justin H; Krowka, Michael Joseph.

In: Liver Transplantation, Vol. 19, No. 12, 12.2013, p. 1370-1376.

Research output: Contribution to journalArticle

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abstract = "Alpha-1-antitrypsin (AAT) deficiency is a rare genetic disease caused by an abnormal production of the serine protease inhibitor AAT. Liver transplantation (LT) cures cirrhosis caused by AAT deficiency and restores the normal production of AAT. There are few reports on the post-LT outcomes of patients with AAT deficiency. The aim of this study was to determine the characteristics and outcomes of patients undergoing LT for AAT deficiency at 3 large transplant centers. All patients undergoing LT at these 3 transplant centers from 1987 to 2012 for AAT deficiency (ZZ or SZ phenotype) were included. The most recent 50 patients with the MZ phenotype were also included for comparison. Data were collected retrospectively from internal databases and medical records. Seventy-three patients (50 with the ZZ phenotype and 23 with the SZ phenotype) underwent LT. The mean age was 52.8 years, and the majority of the patients (75.6{\%}) were men. Before LT, serum AAT levels were lower for the ZZ patients versus the SZ patients (28.3 versus 58.0 mg/dL, P < 0.001). More than 40{\%} of the SZ patients had an additional liver disease, whereas 8{\%} in the ZZ group and 90{\%} in the MZ group did. Before LT, there was no significant difference in pulmonary function between the ZZ and SZ groups. Seventeen patients (all with ZZ phenotype) had pulmonary function tests performed before and after LT. The forced expiratory volume in 1 second (FEV1) continued to decline for the majority. The 1-, 3-, 5-, and 10-year post-LT survival rates were 86{\%}, 83{\%}, 80{\%}, and 72{\%}, respectively, for the ZZ patients and 91{\%}, 86{\%}, 79{\%}, and 79{\%}, respectively, for the SZ patients. In conclusion, survival after LT for patients with ZZ or SZ AAT deficiency is excellent. Despite the normalization of AAT levels after LT, FEV1 continues to decline unexpectedly after LT in some ZZ and SZ patients.",
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