Liver transplantation in children with metabolic diseases: The studies of pediatric liver transplantation experience

Arnon, Ronen; Kerkar, Nanda; Davis, Michael K.; Anand, Ravinder; Yin, Wanrong; González‐Peralta, Regino P.

doi:10.1111/j.1399-3046.2010.01339.x

Cited by 73 publications

(75 citation statements)

References 17 publications

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“…We have confirmed previous findings that survival was largely similar in recipients with and without liver disease . Why this should occur is unclear given that those with liver disease were measurably more ill, being more likely to be hospitalized or to receive intensive care at the time of transplant.…”

Section: Discussionsupporting

confidence: 86%

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

et al. 2019

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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.

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Section: Discussionsupporting

confidence: 86%

“…Those without liver diseases will be more vulnerable to acute metabolic decompensation due to the transplant procedure itself, which, in turn, may impact early allograft function. Higher rates of early graft dysfunction, hepatic artery thrombosis, and perioperative systemic complications are recognized in those with nonstructural disease …”

Section: Discussionmentioning

confidence: 99%

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

et al. 2019

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“…Recent political and economic upheaval has introduced a large number of migrants into Western countries who often may not have had screening for a variety of metabolic or genetic disorders. In addition, children receiving transplantation for metabolic liver disease have improved outcomes compared to children transplanted for other disorders, such as biliary atresia, with 1- and 5-year survival of 95% and 89%[12]. Finally, the presence of severe extrahepatic disease such as severe neuromuscular disease in our patient should not deter consideration for transplantation since liver transplantation can result in remarkable improvement and reversal in neurologic and cognitive dysfunction, particularly in children.…”

Section: Discussionmentioning

confidence: 99%

Case of hepatocellular carcinoma in a patient with hereditary tyrosinemia in the post-newborn screening era

Imseis¹,

Bynon²,

Thornhill³

2017

WJH

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Hereditary tyrosinemia type 1 (HT-1) is a metabolic disorder caused by a defect in tyrosine degradation. Without treatment, symptoms of hepatomegaly, renal tubular dysfunction, growth failure, neurologic crises resembling porphyrias, rickets and possible hepatocellular carcinoma can develop. The use of 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione and early diagnosis through newborn screening initiatives have resulted in a sharp decline in morbidity and mortality associated with this disease. We present a case report of a 7-year-old patient with HT-1 who was born prior to the addition of tyrosinemia to the newborn screening in her birth area. At her time of diagnosis, the patient had developed many of the symptoms associated with her disease, including chronic kidney disease, rickets, and myopathy that left her non-ambulatory. During her initial evaluation, she was also noted to have hepatocellular carcinoma. With cadaveric liver transplantation and nutritional support, her symptoms all either resolved or stabilized. Her case illustrates the severity of the disease if left untreated, the need for vigilance in populations who do not routinely receive newborn screens, and the markedly improved outcomes in patients following transplant.

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“…Fifteen percent of children enrolled in the studies in the pediatric liver transplantation (SPLIT) registry underwent liver transplantation for metabolic diseases and 7.6% of them represented WD. 75 …”

Section: Metabolic Liver Diseasesmentioning

confidence: 99%

Current and Future Burden of Chronic Nonmalignant Liver Disease

Udompap

Kim

2015

Clinical Gastroenterology and Hepatology

117

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Disease burden is an important indicator of the state of health of a population. It can be measured as the frequency (e.g. incidence and prevalence) of a condition or its effects including: fatal and non-fatal health loss from disease (e.g. disability-adjusted life years (DALYs)) as well as the financial costs (e.g. direct health care costs and indirect health care expenditures related to lost income due to premature death). Accurate disease burden information is essential for policy-making such as prioritization of health interventions and allocation of resources. Chronic Liver Disease (CLD) causes substantial health and economic burden in the US, where nearly two million deaths annually are attributable to CLD. In the recent past, overall mortality rate of CLD has been increasing. Viral hepatitis and alcoholic liver disease are thought to be the most common etiologies of chronic liver diseases. More recently, the prevalence of non-alcoholic steatohepatitis (NAFLD) is rapidly increasing and non-alcoholic steatohepatitis (NASH) has become a leading indication for liver transplantation. In this work, we assemble available data on the burden of CLD in the United States, focusing on non-malignant complications, whereas the impact on mortality and healthcare expenses of hepatocellular carcinoma (HCC), an important consequence of CLD is discussed elsewhere.

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Liver transplantation in children with metabolic diseases: The studies of pediatric liver transplantation experience

Cited by 73 publications

References 17 publications

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

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

Case of hepatocellular carcinoma in a patient with hereditary tyrosinemia in the post-newborn screening era

Current and Future Burden of Chronic Nonmalignant Liver Disease

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