An interventional study of TIPS in Cystic Fibrosis Liver Disease, Portal Hypertension Due to Cystic Fibrosis (Disorder) and Portal Hypertension, Noncirrhotic, sponsored by University Hospital, Ghent. Completed. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2021-09-08.
Sponsored by University Hospital, Ghent · Not applicable, Interventional, and Prevention
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)
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.
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Exclusion Criteria:
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.
Procedure: TIPS
Transjugular Intrahepatic Portosystemic Shunt is performed to prevent / treat symptomatic portal hypertension
Variceal bleeding
Variceal bleeding, diagnosed on endoscopy, is regarded as the main complication of portal hypertension from cystic fibrosis liver disease
Time frame: Through study completion, an average of 10 year
Hypersplenism
Thrombocytopenia causes an increased bleeding tendency. Increasing splenomegaly causes earlier concerns with abdominal distension and decreased appetite by gastric compression. In symptomatic hypersplenism, a surgical splenorenal shunt or splenectomy might be indicated.
Time frame: Through study completion, an average of 10 year
Liver transplant/splenectomy-free survival
Transplant/splenectomy-free survival time was calculated from date of TIPS to the following event: transplant, splenectomy and death from any cause.
Time frame: Through study completion, an average of 10 year
Model for end-stage liver disease (MELD) score
The Model for End-Stage Liver Disease, or MELD, is a scoring system for assessing the severity of chronic liver disease.
Time frame: Through study completion, an average of 10 year
Body Mass Index (BMI) for age Z-score
BMI-for age Z-scores are measures of relative weight adjusted for child age and sex defined by the Flemish growth charts
Time frame: Through study completion, an average of 10 year
Length for age Z-score
Length-for age Z-scores are measures of relative length adjusted for child age and sex defined by the Flemish growth charts
Time frame: Through study completion, an average of 10 year
No study locations are listed for this record.
Plan to share: No
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University Hospital, Ghent