Pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) in Cystic Fibrosis Related Liver Disease

NCT ID: NCT05037643

Last Updated: 2021-09-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-06-30
• Study Completion Date: 2020-12-31

Brief Summary

Portal hypertension (PHT) and its sequelae are the most clinically important manifestation in cystic fibrosis related liver disease (CFLD), with end-stage liver failure as a late and rare manifestation. The aim is to evaluate the safety and efficacy of a pre-emptive Transjugular Intrahepatic Portosystemic Shunt (TIPS) for the prophylaxis of variceal bleeding in pediatric CFLD patients with subclinical non-cirrhotic portal hypertension (NCPH)

Detailed Description

Cystic fibrosis (CF) is the most frequent autosomal recessive disorder in Caucasians caused by a mutation in the cystic fibrosis transmembrane conductance regulator gene. Cystic Fibrosis associated Liver Disease (CFLD) is a well-known complication and includes a wide range of hepatobiliary diseases. The clinical outcome in CFLD is largely determined by PHT and its sequelae. Variceal bleeding is the most feared complication. Currently, there is no medical therapy to delay or reverse clinically established CFLD.Treatment focuses on screening for and managing the complications of portal hypertension and optimizing nutritional status. Eventually, liver transplantation (LT) is an effective therapeutic option for CF patients with end-stage liver failure, treatment resistant, and complicated portal hypertension.

TIPS placement is a well-established procedure for portal vein decompression in adults. In retrospective case studies of severe CFLD cirrhosis in pediatric populations, TIPS has been proven a feasible option in acute or refractory variceal bleeding and as a bridge to LT. In CFLD patients, little data are available on the long-term outcome of treatments to mitigate PHT. In this trial, the primary aim was to evaluate the safety and efficacy of a pre-emptive TIPS for the prophylaxis of variceal bleeding in non-cirrhotic CFLD with early PHT. The secondary aim was to investigate the long-term clinical outcome of a pre-emptive TIPS and in more particular, whether TIPS might effectively postpone LT.

Conditions

Cystic Fibrosis Liver Disease; Portal Hypertension Due to Cystic Fibrosis (Disorder); Portal Hypertension, Noncirrhotic

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

Pre-emptive TIPS in Cystic Fibrosis Related Liver Disease and non-cirrhotic portal hypertension

Patients with CFLD without cirrhosis were eligible for a pre-emptive TIPS, when early (asymptomatic) signs of portal hypertension. All procedures were performed under general anaesthesia by an experienced interventional radiologist. Depending on the patient's age and physiognomy, TIPS was created following a conventional transjugular technique as for adults or by a dedicated combined percutaneous transhepatic-transjugular (PIPS) approach for small children. Routinely, an expanded polytetrafluoroethylene-covered endoprosthesis was used for shunt creation. If the sheath could not be negotiated into the main portal vein, a self-expandable, non-covered stent was placed. We did not pursue a minimum gradient reduction.

Percutaneous liver biopsy was performed during TIPS procedure to confirm the diagnosis of fibrosis or cirrhosis.

Group Type: EXPERIMENTAL

TIPS

Intervention Type: PROCEDURE

Transjugular Intrahepatic Portosystemic Shunt is performed to prevent / treat symptomatic portal hypertension

Interventions

TIPS

Transjugular Intrahepatic Portosystemic Shunt is performed to prevent / treat symptomatic portal hypertension

Intervention Type: PROCEDURE

Eligibility Criteria

Inclusion Criteria

• Cystic fibrosis
• Liver disease detected by an abnormal physical examination (hepatomegaly or splenomegaly, confirmed on US), abnormalities of liver function tests (increase of AST, ALT, GGT levels above the upper normal limits) or ultrasonographic evidence of liver involvement (US liver score ≥ 5).
• Indirect signs of portal hypertension on Doppler US
• Progressive portal hypertension / liver disease on bi-monthly follow-up, evaluated by physical examination, blood analysis and US

Exclusion Criteria

• Cirrhosis on biopsy
• Symptomatic portal hypertension (Portosytemic pressure gradient > 10 mmHg)

• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Ghent

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Luc Defreyne, M.D., Ph.D

Role: PRINCIPAL_INVESTIGATOR

University Ghent Hospital

Other Identifiers

B67020071504

Identifier Type: -

Identifier Source: org_study_id