Evolving Trends in Liver Transplant for Metabolic Liver Disease in the United States

Abstract

Indications for liver transplantation (LT) in metabolic disease are evolving. We reviewed the US experience with primary LT for metabolic disease in the Scientific Registry for Transplant Recipients (October 1987 to June 2017) to determine the following: temporal changes in indications, longterm outcomes, and factors predicting survival. Patients were grouped by the presence of structural liver disease (SLD) and whether the defect was confined to the liver. There were 5996 patients who underwent LT for metabolic disease, 2354 (39.3%) being children. LT for metabolic disease increased in children but not in adults. Children experienced a 6‐fold increase in LT for metabolic disease without SLD. Indications for LT remained stable in adults. Living donor liver transplantation increased between era 1 and era 3 from 5.6% to 7.6% in children and 0% to 4.5% in adults. Patient and graft survival improved with time. The latest 5‐year patient survival rates were 94.5% and 81.5% in children and adults, respectively. Outcomes were worse in adults and in those with extrahepatic disease (P < 0.01), whereas SLD did not affect outcomes. Survival improved with younger age at LT until age <2 years. On multivariate analysis, diagnostic category, inpatient status, age at LT, and transplant era significantly predicted outcomes in all ages with male sex predicting survival in childhood only. Children without structural disease were less likely to die awaiting LT and had improved post‐LT survival compared with children with chronic liver disease. In conclusion, LT for metabolic disease is increasingly used for phenotypic correction in children; extrahepatic manifestations significantly impact survival at all ages; where indicated, transplantation should not be unnecessarily delayed; and the development of new allocation models may be required.

Metabolic Indications for Pediatric Liver Transplantation: A Slow Train Coming

“…Living related LT, performed at a low rate both in children and adults, has not affected graft and patient survival rates . This information, although previously often reported from Japan, could be relevant in countries with less readily available sources of cadaveric organs.…”

“…An analysis by McKiernan et al in the current issue of Liver Transplantation provides a good basis to contemplate future metabolic indications for pediatric and adult liver transplantation (LT). The authors divide their 30‐year observation period (1987‐2017) into 3 different eras to detect trends in numbers and outcomes.…”

“…Reassuringly, the authors report very impressive outcomes, including the most recent 5‐year survival rate of 94.5% for children undergoing LT for metabolic conditions. The best outcomes were observed in recipients without systemic disease …”

“…Notably, there was no major era difference in the trends for LT in the adult population, where A1ATD, hereditary hemochromatosis, and Wilson disease remain the principal indications. One of the most significant changes is a 6‐fold increase in pediatric recipients without structural liver disease …”

“…The best outcomes were observed in recipients without systemic disease. (1) Pediatric recipients most commonly had alpha-1-antitrypsin deficiency (A1ATD), tyrosinemia type 1, cystic fibrosis (CF), and Wilson disease, with the more recent additions of urea cycle disorders and organic acidemias. Notably, there was no major era difference in the trends for LT in the adult population, where A1ATD, hereditary hemochromatosis, and Abbreviations: A1ATD, alpha-1-antitrypsin deficiency; CF, cystic fibrosis; LT, liver transplantation.Address reprint requests to Nedim Hadžić, M.…”