Predictive Models of Hepatic Decompensation and Survival Outcomes in Pediatric Patients With Cirrhosis

Recruiting

• Conditions: Biliary Atresia; Autoimmune Hepatitis; HCV; Primary Sclerosing Cholangitis; Cirrhosis; Choledochal Cyst; HBV
• Interventions: Other: Prediction model

• Registration Number: NCT05181332
• Lead Sponsor: West China Hospital

Brief Summary

The aim of this study was to developed and validated models to predict hepatic decompensation and survivals in pediatric patients with cirrhosis and compared these models with currently available models.

Detailed Description

Noninvasive liver fibrosis tests such as the NAFLD fibrosis score (NFS), Hepascore, and transient elastography were specifically developed to predict fibrosis and can help predict patients with NAFLD at the highest risk of developing liver-related complications. These tests have been widely applied in adult cirrhosis. The accuracy of these models, however, may be influenced by patient factors including age, body mass index, and diabetes, potentially limiting their prognostic accuracy and clinical practicability in children. Therefore, it is currently unknown how to best predict hepatic decompensation and survival outcomes among pediatric patients with cirrhosis. To fill this knowledge gap, the investigators performed a retrospective-prospective cohort study with the aim of developing and validating a clinical model to predict liver-related complications and survival outcomes in pediatric patients with biopsy-proven with cirrhosis. Secondly, the investigators aimed to compare the predictive accuracy with currently available noninvasive model.

Study Timeline

• Study Start: 2021-01-01
• Study First Submitted: 2021-12-17
• Study First Submitted QC: 2021-12-17
• Status Verified: 2022-01
• Last Update Submitted: 2022-01-05
• Study First Posted: 2022-01-06
• Last Update Posted: 2022-01-20
• Primary Completion: 2024-12-31
• Study Completion: 2024-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 400

Inclusion Criteria

• Age up to 18 years old
• No previous episodes of clinical decompensation
• With written informed consent

Exclusion Criteria

• Clinical data missing
• Without written informed consent

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Retrospective cohort	Prediction model	The internal cohort was retrospectively enrolled in West China Hospital, Sichuan University from June 2010 and December 2020. It is a training and internal validation cohort.
Prospective cohort	Prediction model	The same inclusion/exclusion criteria were applied for the same center prospectively. It is a external validation cohort.

Primary Outcome Measures

Name	Time	Method
Hepatic decompensation	At least 5-year follow up	The primary outcome was the first event of hepatic decompensation, defined by the occurrence of any of the following: ascites (identified or confirmed by abdominal ultrasound), upper gastrointestinal bleeding secondary to portal hypertension (confirmed by endoscopy in the presence of gastroesophageal varices or portal hypertensive gastropathy), or hepatic encephalopathy (established by clinical parameters, neuropsychological tests, or electroencephalogram)

Secondary Outcome Measures

Name	Time	Method
Overall survival	At least 5-year follow up	The endpoint was defined as the occurrence of death or the last follow-up. Patients were followed from the day of liver biopsy until the occurrence of death, liver transplant, or last visit. The outcome was evaluated by an experienced hepatologist in each center every 3-6 months. At each visit, a medical history, physical examination, and standard laboratory tests were performed.

Trial Locations

Locations (1)

West China Hospital, Sichuan University; 🇨🇳 Chengdu, Sichuan, China