Interventions for managing advanced liver disease in cystic fibrosis

Review question

We aimed to find the best evidence about preventing and managing advanced liver disease in adults and children with cystic fibrosis by comparing different treatment options.


In advanced liver disease in cystic fibrosis, the normal liver tissue is replaced by scar tissue. As the disease progresses, the liver becomes hard and the blood cannot easily flow through the organ leading to increased pressure in an important blood vessel in the liver called the portal vein (portal hypertension). Later on, the veins around the lower part of the oesophagus (gullet) become swollen and torn, resulting in life-threatening bleeding (variceal bleeding). There are several treatments currently available for variceal bleeding and portal hypertension; drug treatments (non-selective beta blockers), endoscopic therapy (e.g. band ligation where tiny elastic bands are placed around the enlarged veins to tie them off so they can't bleed), or sclerotherapy (where a blood-clotting solution is injected directly into a vein and irritates the lining of the blood vessel so it swells and sticks together). The insertion of a transjugular intrahepatic porto-systemic shunt (also known as TIPSS) (an artificial channel within the liver that allows movement between the inflowing and out flowing veins) has been employed in recurrent bleeding or as a bridge to liver transplantation. Surgical porto-systemic shunts have also been used in selected patients with preserved liver function. Liver transplantation is performed in cystic fibrosis patients with decompensated cirrhosis or end-stage liver disease.

Guidelines for screening and managing portal hypertension have been available for the general population (without cystic fibrosis). However, the optimal treatment for advanced liver disease in cystic fibrosis has not yet been defined, leading to a wide variety of practice among different centres.

Search date

The evidence is current to: 04 January 2017.

Study characteristics

We searched for high quality trials comparing the treatments described above in children and adults with advanced liver disease in cystic fibrosis. Unfortunately, we did not find any trials to include in this review.

Key results

Since we did not find any trials for this review, it is not possible to make any specific recommendations or to develop best-practice guidelines at this stage. Our review highlighted a clear need for randomised controlled trials of treatments for the prevention and management of advanced liver disease in adults and children with cystic fibrosis.

Authors' conclusions: 

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.

Read the full abstract...

Cystic fibrosis is an autosomal recessive inherited defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene resulting in abnormal regulation of salt and water movement across the membranes. In the liver this leads to focal biliary fibrosis resulting in progressive portal hypertension and end-stage liver disease in some individuals. This can be asymptomatic, but may lead to splenomegaly and hypersplenism, development of varices and variceal bleeding, and ascites; it has negative impact on overall nutritional status and respiratory function in this population. Prognosis is poor once significant portal hypertension is established. The role and outcome of various interventions for managing advanced liver disease (non-malignant end stage disease) in people with cystic fibrosis is currently unidentified.


To review and assess the efficacy of currently available treatment options for preventing and managing advanced liver disease in children and adults with cystic fibrosis.

Search strategy: 

We searched the Cochrane Cystic Fibrosis Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books.

Date of last search: 06 April 2017.

We also searched the reference lists of relevant articles and reviews and online trials registries. Date of last search: 04 January 2017.

Selection criteria: 

Any published and unpublished randomised controlled trials and quasi-randomised controlled trials of advanced liver disease in cystic fibrosis with cirrhosis or liver failure, portal hypertension or variceal bleeding (or both).

Data collection and analysis: 

Authors independently examined titles and abstracts to identify potentially relevant trials, but none were eligible for inclusion in this review.

Main results: 

A comprehensive search of the literature did not identify any published eligible randomised controlled trials.