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

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.

Study Overview

• Status: Recruiting
• Conditions: Cirrhosis; Primary Sclerosing Cholangitis; Autoimmune Hepatitis; HBV; HCV; Biliary Atresia; Choledochal Cyst
• Intervention / Treatment: Other: Prediction model

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 Type: Observational
• Enrollment (Anticipated): 400

Contacts and Locations

Study Locations

• China
  • Sichuan
    • Chengdu, Sichuan, China, 6100000
      • Recruiting
      • West China Hospital, Sichuan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second to 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Pediatric patients with cirrhosis who were diagnosed histologically with definite pathologic specimens.

Description

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 Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Retrospective cohort; 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.	Other: Prediction model; Clinical models were developed and validated to predict liver-related complications and survival outcomes in pediatric patients with biopsy-proven with cirrhosis.
Prospective cohort; The same inclusion/exclusion criteria were applied for the same center prospectively. It is a external validation cohort.	Other: Prediction model; Clinical models were developed and validated to predict liver-related complications and survival outcomes in pediatric patients with biopsy-proven with cirrhosis.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hepatic decompensation	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)	At least 5-year follow up

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival	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.	At least 5-year follow up

Collaborators and Investigators

• Sponsor: West China Hospital

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 1, 2021
• Primary Completion (ANTICIPATED): December 31, 2024
• Study Completion (ANTICIPATED): December 31, 2024

Study Registration Dates

• First Submitted: December 17, 2021
• First Submitted That Met QC Criteria: December 17, 2021
• First Posted (ACTUAL): January 6, 2022

Study Record Updates

• Last Update Posted (ACTUAL): January 20, 2022
• Last Update Submitted That Met QC Criteria: January 5, 2022
• Last Verified: January 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Immune System Diseases; Neoplasms; Autoimmune Diseases; Cysts; Congenital Abnormalities; Liver Diseases; Hepatitis, Chronic; Biliary Tract Diseases; Hepatitis; Bile Duct Diseases; Digestive System Abnormalities; Fibrosis; Liver Cirrhosis; Cholangitis; Cholangitis, Sclerosing; Hepatitis, Autoimmune; Biliary Atresia; Choledochal Cyst
• Other Study ID Numbers: HX-2021523

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No