Liver-directed gene therapy: molecular tools and current preclinical and clinical studies
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Key Points 1 How do physicians decide which patients with pulmonary vascular disease will benefit from liver transplantation? 2 Studies on patients with pulmonary vascular disease are limited and the findings and recommendations may not apply to all practice sites. 3 All patients with hypoxemia, liver disease, and pulmonary vasodilation do not have hepatopulmonary syndrome (HPS). 4 Not all patients with hepatopulmonary syndrome will benefit from liver transplantation. 5 The mean pulmonary artery pressure (mPAP) may not be an accurate predictor of mortality in patients with portopulmonary hypertension. 6 The effects of pulmonary vasodilators on the outcome of patients with portopulmonary hypertension (PPHTN) is still unconfirmed but promising. (Liver Transpl 2004;10:S54–S58.)
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Hepatopulmonary syndrome (HPS) is associated with increased waitlist mortality in liver transplantation (LT) candidates. Children with HPS are granted Model for End-Stage Liver Disease (MELD)/Pediatric End-Stage Liver Disease (PELD) exception points for waitlist prioritization in the United States based on criterion developed for adults. In this study, the impact of this MELD/PELD exception policy on post-LT survival in children was examined. A retrospective cohort of patients aged younger than 18 years with a MELD/PELD exception request who underwent LT between 2007 and 2018 were identified in the Scientific Registry of Transplant Recipients. Patients were stratified by waitlist partial pressure of arterial oxygen (PaO 2 ) to assess risk factors for waitlist mortality and post-LT survival. Among 3082 pediatric LT recipients included in the study, 124 patients (4%) received MELD/PELD exception points for HPS. Patients with HPS were a median age of 9 years (interquartile range: 6, 12 years), 54.8% were girls, and 54% were White. Most patients (87.9%) were listed with laboratory MELD/PELD scores <15. Waitlist mortality for patients with HPS exception points was rare and not different from patients without HPS. When stratified by pre-LT PaO 2 , hypoxemia severity was not associated with differences in 1-, 3-, or 5-year survival rates after LT ( p = 0.13). However, patients with HPS showed a slightly lower survival rate at 5 years compared with patients without HPS (88.7% vs. 93.4%; p = 0.04). MELD/PELD exceptions for children with HPS mitigated waitlist mortality, and recipients with HPS experienced excellent 5-year survival after LT, although slightly lower than in patients without HPS. Unlike adults with HPS, the severity of pre-LT hypoxemia in children does not impact post-LT survival. These data suggest that adult criteria for granting MELD/PELD exception points may not appropriately capture HPS severity in pediatric patients. Further prospective multicenter studies to examine the risk factors predicting negative survival outcomes in children with HPS are warranted.
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Liver disease
Interquartile range
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Liver disease
Alcoholic Hepatitis
Hepatitis C
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As the mean Model for End-Stage Liver Disease (MELD) score at time of liver transplantation continues to increase, it is crucial to implement preemptive strategies to reduce wait-list mortality. We review the most common complications that arise in patients with a high MELD score in an effort to highlight strategies that can maximize survival and successful transplantation.
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With an advance in liver transplantation, patients should be managed with a thorough knowledge of the pathophysiology and pharmacology of liver disease The action of anesthetic drugs on the diseased liver or the special circumstances of this operation have been under investigation. This article focuses on recent observations in advanced liver disease and liver transplantation.
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Urea cycle disorders (UCDs) are rare causes of hyperammonemic encephalopathy in adults. Most UCDs present in childhood and, if unrecognized, are rapidly fatal. Affected individuals who survive to adulthood may remain undiagnosed because of clinicians' unawareness of the condition or atypical presentations. We describe the case of a 49-year-old man who initially presented with a stroke and developed hyperammonemic encephalopathy over a period of 8 months. A diagnosis of carbamoyl phosphate synthetase type 1 deficiency was made, and the patient was referred for liver transplantation. One year after liver transplantation, the patient had normal plasma ammonia concentrations and had returned to work.
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The classical form of alpha-1-antitrypsin deficiency (A1ATD) is known to cause liver disease in children and adults, but there is relatively little information about the risk of severe, progressive liver disease and the need for liver transplantation. To better understand how newly evolving pharmacological, genetic, and cellular therapies may be targeted according to risk for progressive liver disease, we sought to determine the age distribution of A1ATD as a cause of severe liver disease, as defined by the need for liver transplantation. Using 3 US liver transplantation databases for the period 1991-2012, we found 77.2% of 1677 liver transplants with a reported diagnosis of A1ATD were adults. The peak age range was 50-64 years. Using 2 of the databases which included specific A1AT phenotypes, we found that many of these adults who undergo liver transplantation with A1ATD as the diagnosis are heterozygotes and have other potential causes of liver disease, most notably obesity and ethanol abuse. However, even when these cases are excluded and only ZZ and SZ phenotypes are considered, severe liver disease requiring transplantation is more than 2.5 times as likely in adults. The analysis also showed a markedly increased risk for males. In the pediatric group, almost all of the transplants are done in children less than 5 years of age. In conclusion, A1ATD causes progressive liver disease most commonly in adults with males in the highest risk category. In the pediatric group, children less than 5 years of age are highest in risk. These results suggest that A1ATD most commonly causes liver disease by mechanisms similar to age-dependent degenerative diseases and more rarely in children by powerful modifiers. Liver Transplantation 22 886-894 2016 AASLD.
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Liver disease
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The model for end-stage liver disease (MELD) is considered to be a good predictor of disease status in patients with end-stage liver disease and has been used for organ distribution in liver transplantation. This article briefly describes the relationship between MELD, MELD-Na score and the mortality of patients waiting for liver transplantation, the intraoperative blood transfusion, the survival, complications, and re-transplantation after liver transplantation.
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End stage liver disease; Liver transplantation; MELD-Na score; Postoperative complications
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The heart and liver are organs that are closely related in both health and disease. Patients who undergo liver transplantation may suffer from heart disease that is: (a) related to the original cause of the liver disease such as hemochromatosis, (b) related to the liver disease itself, or (c) related to other associated conditions. Furthermore, liver transplantation is one of the most cardiovascular stressful events that a patient with cirrhosis may undergo. After liver transplantation, the progression of pre-existing or the development of new-onset cardiac disease may occur. This article reviews the relationship between the heart and liver transplantation in the pre-transplant, intra-operative, and post-transplant periods.
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Hereditary hemochromatosis
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Background data: Liver transplantation is the best therapeutic option for patients with end- stage liver disease due to its excellent long term survival results. The demand for deceased donor liver transplantation vastly exceeds the supply. In the U.S. the Model for End Stage Liver Disease (MELD) score is now used for allocation in liver transplantation waiting lists, replacing the Child- Turcotte- Pugh (CTP) score. The MELD system is based on the risk of death without transplantation and was originally developed for survival estimation in patients after TIPS. The majority of the European countries still use the CTP score for liver organ allocation. However, there is a debate whether the MELD score is superior CTP to predict mortality in patients with cirrhosis on waiting list and after liver transplantation.
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