Interventions for preventing and managing advanced liver disease in cystic fibrosis

Palaniappan, Senthil K; Than, Nan Nitra; Thein, Aung Win; Moe, Soe; Mourik, Indra van

doi:10.1002/14651858.cd012056.pub2

Cited by 11 publications

(15 citation statements)

References 73 publications

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“…8,11 There are no existing evidence-based guidelines or specific recommendations for the prevention or management of CFLD. 1,11,42 Therefore, once CFLD is diagnosed, therapy is directed towards lowering the impact of complications. 1,11,42…”

Section: Introductionmentioning

confidence: 99%

“…1,11,42 Therefore, once CFLD is diagnosed, therapy is directed towards lowering the impact of complications. 1,11,42…”

Section: Introductionmentioning

confidence: 99%

“…Management of CFLD emphasizes on optimizing nutritional status via adequate caloric intake, fat-soluble vitamin supplementation, pancreatic enzyme replacement therapy, and avoiding hepatotoxic medications. 42 Ursodeoxycholic acid (UDCA) is the only available treatment that may prevent the progression of CFLD. 10 The physiological benefits of UDCA comprise improving bile flow and non-CFTR biliary chloride channels and protecting hepatocytes from the toxicity of accumulating bile acids.…”

Section: Introductionmentioning

confidence: 99%

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Cystic Fibrosis Liver Disease: Know More

et al. 2019

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Cystic fibrosis (CF) is a multisystem disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. CFTR is expressed in the apical surface of cholangiocytes. Homozygous CFTR gene mutation results in viscous and acidic bile secretions secondary to deficient surface fluid and bicarbonate efflux. Viscous, inspissated bile causes ductular obstruction and hepatotoxicity from retained bile components, leading to fibrosis and ultimately cirrhosis, known as CF liver disease (CFLD). CFLD is the third leading cause of death in CF patients. CFLD manifestations can take many forms. They range from asymptomatic elevation of transaminases to cirrhosis and end-stage liver disease. CFLD is diagnosed after excluding other causes of chronic liver disease. To date, there is no effective therapy to prevent or treat CFLD. Management of CFLD emphasizes on optimizing nutritional status. Ursodeoxycholic acid is the only available treatment that may prevent progression of CFLD at present. All CF patients with CFLD need annual investigations and follow-up for early detection of the disease. Liver transplantation should be considered in patients with decompensated cirrhosis and portal hypertension, with acceptable long-term outcomes. Novel therapies of CFLD are promising. This review article aims to summarize the published literature on CFLD, its pathophysiology, clinical features and complications, and management including new therapeutic options.

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Section: Introductionmentioning

confidence: 99%

“…1,11,42 Therefore, once CFLD is diagnosed, therapy is directed towards lowering the impact of complications. 1,11,42…”

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Cystic Fibrosis Liver Disease: Know More

et al. 2019

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“…Portal hypertension and its sequelae represent the most significant risk to patient mortality with variceal bleeding rates reported between 20% and 50% within 10 years of diagnosis . Currently, there is no medical therapy to treat or reverse CFLD . However, LT is an accepted therapy for individuals with advanced liver disease.…”

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confidence: 99%

A Multidisciplinary Approach to Pretransplant and Posttransplant Management of Cystic Fibrosis–Associated Liver Disease

et al. 2019

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Approximately 5%‐10% of patients with cystic fibrosis (CF) will develop advanced liver disease with portal hypertension, representing the third leading cause of death among patients with CF. Cystic fibrosis with advanced liver disease and portal hypertension (CFLD) represents the most significant risk to patient mortality, second only to pulmonary or lung transplant complications in patients with CF. Currently, there is no medical therapy to treat or reverse CFLD. Liver transplantation (LT) in patients with CFLD with portal hypertension confers a significant survival advantage over those who do not receive LT, although the timing in which to optimize this benefit is unclear. Despite the value and efficacy of LT in selected patients with CFLD, established clinical criteria outlining indications and timing for LT as well as disease‐specific transplant considerations are notably absent. The goal of this comprehensive and multidisciplinary report is to present recommendations on the unique CF‐specific pre‐ and post‐LT management issues clinicians should consider and will face.

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“…CF often lead to various comorbidities of multiple systems in the body, such as bronchiectasis, small airways obstruction, progressive respiratory impairment, malabsorption, biliary cirrhosis, and infertility (4). Among these comorbidities, CF-associated liver diseases (CFLD) accounts for around 25% of patients with CF (5). Moreover, ~3.3% of mortality makes the CFLD one of the most cause of death in CF (6).…”

Section: Introductionmentioning

confidence: 99%

Liver Failure in a Chinese Cystic Fibrosis Child With Homozygous R553X Mutation

Lin

et al. 2019

Front. Pediatr.

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Cystic fibrosis (CF) is a relatively rare disease in Asians with various clinical characteristics, including CF-associated liver disease (CFLD), which is a common early non-pulmonary complication. This case report describes a Chinese CF patient harboring a homozygous nonsense mutation (c.1657C>T, p.R553X) who was failure to thrive and had intermittently diarrhea during the first year after birth. Liver function test of the patient showed the mildly and intermittently elevated alanine aminotransferase (ALT) levels ranging from 70 to 92 U/L and aspartate aminotransferase (AST) levels ranging from 80 to 90 U/L, which began at 8 months of age and lasted for 4 years without CF diagnosis. In addition, abdominal computed tomography (CT) revealed diffuse fatty infiltration of the liver at 4 years old and gradually developed hepatic cirrhosis. Subsequently, cirrhosis rapidly progressed with obvious splenomegaly and pancreatic insufficiency and the patient died of liver failure with coagulopathy by the age of 6 years old. Pediatricians should remain vigilant to avoid failure to diagnose CF, the occurrence of which may be underestimated, and pay greater attention to the patients with atypical clinical manifestations in Asian countries.

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Interventions for preventing and managing advanced liver disease in cystic fibrosis

Abstract: In order to develop the best source of evidence, there is a need to undertake randomised controlled trials of interventions for preventing and managing advanced liver disease in adults and children with cystic fibrosis.

Cited by 11 publications

References 73 publications

Cystic Fibrosis Liver Disease: Know More

Cystic Fibrosis Liver Disease: Know More

A Multidisciplinary Approach to Pretransplant and Posttransplant Management of Cystic Fibrosis–Associated Liver Disease

Liver Failure in a Chinese Cystic Fibrosis Child With Homozygous R553X Mutation

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