Liver transplantation as a definitive treatment for familial hypercholesterolemia: A series of 36 cases

Mansoorian, Mohsenreza; Kazemi, Kourosh; Nikeghbalian, Saman; Shamsaeefar, Alireza; Mokhtari, Maral; Dehghani, Seyed Mohsen; Salahi, Heshmatollah; Amoozgar, Hamid; Malek-Hosseini, Seyed Ali

doi:10.1111/petr.12562

Cited by 23 publications

(27 citation statements)

References 14 publications

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“…193,295 In contrast, an HLCM comprised of hepatocytes of HFHC well recapitulated the disease presentation. 193 To date, liver transplantation is the only definitive therapeutic intervention for HFHC 296 ; however, reservation exists for a variety of reasons. First, the hepatocytes of HFHC remain normal lifelong and thus maintain normal liver function except for LDL uptake.…”

Section: Genetic Liver Diseasesmentioning

confidence: 99%

Art of Making Artificial Liver: Depicting Human Liver Biology and Diseases in Mice

et al. 2020

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Advancement in both bioengineering and cell biology of the liver led to the establishment of the first-generation humanized liver chimeric mouse (HLCM) model in 2001. The HLCM system was initially developed to satisfy the necessity for a convenient and physiologically representative small animal model for studies of hepatitis B virus and hepatitis C virus infection. Over the last two decades, the HLCM system has substantially evolved in quality, production capacity, and utility, thereby growing its versatility beyond the study of viral hepatitis. Hence, it has been increasingly employed for a variety of applications including, but not limited to, the investigation of drug metabolism and pharmacokinetics and stem cell biology. To date, more than a dozen distinctive HLCM systems have been established, and each model system has similarities as well as unique characteristics, which are often perplexing for end-users. Thus, this review aims to summarize the history, evolution, advantages, and pitfalls of each model system with the goal of providing comprehensive information that is necessary for researchers to implement the ideal HLCM system for their purposes. Furthermore, this review article summarizes the contribution of HLCM and its derivatives to our mechanistic understanding of various human liver diseases, its potential for novel applications, and its current limitations.

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Section: Genetic Liver Diseasesmentioning

confidence: 99%

Art of Making Artificial Liver: Depicting Human Liver Biology and Diseases in Mice

et al. 2020

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“…The need for greater availability of apheresis, in support of this strategy, is highlighted. A premise of the HEART UK clinical strategy for managing HoFH is that the early use of lomitapide and/or evolocumab, beyond their current licensed age restriction, in severely affected children and young adults should be balanced against the risks of liver transplantation [24].…”

Section: Introductionmentioning

confidence: 99%

HEART UK statement on the management of homozygous familial hypercholesterolaemia in the United Kingdom

et al. 2016

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This consensus statement addresses the current three main modalities of treatment of homozygous familial hypercholesterolaemia (HoFH): pharmacotherapy, lipoprotein (Lp) apheresis and liver transplantation. HoFH may cause very premature atheromatous arterial disease and death, despite treatment with Lp apheresis combined with statin, ezetimibe and bile acid sequestrants. Two new classes of drug, effective in lowering cholesterol in HoFH, are now licensed in the United Kingdom. Lomitapide is restricted to use in HoFH but, may cause fatty liver and is very expensive. PCSK9 inhibitors are quite effective in receptor defective HoFH, are safe and are less expensive. Lower treatment targets for lipid lowering in HoFH, in line with those for the general FH population, have been proposed to improve cardiovascular outcomes. HEART UK presents a strategy combining Lp apheresis with pharmacological treatment to achieve these targets in the United Kingdom (UK). Improved provision of Lp apheresis by use of existing infrastructure for extracorporeal treatments such as renal dialysis is promoted. The clinical management of adults and children with HoFH including advice on pregnancy and contraception are addressed. A premise of the HEART UK strategy is that the risk of early use of drug treatments beyond their licensed age restriction may be balanced against risks of liver transplantation or ineffective treatment in severely affected patients. This may be of interest beyond the UK.

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“…To help answer these questions, Mansoorian now reports a cohort of children transplanted for HoFH (36 cases) with a mean follow‐up of 24 months . To determine when to transplant patients with HoFH, it is critical to pay particular attention to the local center's resources.…”

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confidence: 99%

Familial homozygous hypercholesterolemia: When to turn to transplant?

Asai

Kohli

2015

Pediatric Transplantation

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Liver transplantation as a definitive treatment for familial hypercholesterolemia: A series of 36 cases

Cited by 23 publications

References 14 publications

Art of Making Artificial Liver: Depicting Human Liver Biology and Diseases in Mice

Art of Making Artificial Liver: Depicting Human Liver Biology and Diseases in Mice

HEART UK statement on the management of homozygous familial hypercholesterolaemia in the United Kingdom

Familial homozygous hypercholesterolemia: When to turn to transplant?

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