Biliary Atresia, Kasai Portoenterostomy, Liver Fibrosis, Liver Function Disorders, Mesenchymal Stem Cell Transplantation, Survival Rate
Conditions
Brief summary
The goal of this clinical trial with historical control is to evaluate whether umbilical cord-derived mesenchymal stem cell (UC-MSC) therapy can improve clinical outcomes in infants with biliary atresia undergoing Kasai surgery before 90 days of age. The main questions it aims to answer are: Does UC-MSC therapy improve liver function parameters (bilirubin, albumin, liver enzymes, coagulation profile)? Does UC-MSC therapy reduce complications such as anemia, ascites, jaundice, and improve PELD scores? Does UC-MSC therapy improve overall survival compared to standard Kasai surgery alone? Researchers will compare the group receiving UC-MSC in 2025-2027 with a historical control group of patients who previously underwent Kasai surgery without UC-MSC therapy. Participants will: Undergo preoperative evaluation, including laboratory and imaging tests. Receive Kasai surgery combined with intraoperative trans-portal vein injection of 20 million UC-MSCs. Be monitored postoperatively through serial laboratory tests, imaging (Fibroscan), and clinical assessments at scheduled intervals. Be followed up for potential serious adverse events and survival outcomes.
Interventions
Intraoperative trans-portal vein injection of 20 million mesenchymal stem cells derived from umbilical cord tissue, suspended in 5 mL autologous plasma.
PROCEDUREKasai Portoenterostomy
Surgical procedure to create a portoenterostomy for biliary drainage in infants with biliary atresia.
Sponsors
Universitas Diponegoro
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
0 Days to 90 Days
Healthy volunteers
No
Inclusion criteria
* Infants diagnosed with biliary atresia confirmed by intraoperative findings. * Underwent Kasai portoenterostomy at an age less than 90 days. * Parents or legal guardians have provided informed consent to participate in the study and follow all study procedures.
Exclusion criteria
* Infants with severe malnutrition. * Infants with major congenital anomalies other than biliary atresia. * Infants with positive tumor markers.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Lactate Dehydrogenase (LDH) | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Tissue injury marker |
| Alanine Aminotransferase (ALT) | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Liver injury marker |
| Aspartate Aminotransferase (AST) | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Liver injury marker |
| Prothrombin Time (PT), aPTT, INR | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Coagulation parameters |
| Alpha Fetoprotein (AFP) | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Tumor marker |
| Alkaline Phosphatase (ALP) | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Liver and bone function marker |
| Serum Albumin Level | Baseline (Pre-op), 4 weeks, 6 weeks, 12 weeks, 24 weeks, and 12 months post-operation | Measurement of serum albumin as marker of liver synthetic function |
| Total Bilirubin Level | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Measurement of bilirubin levels (total, direct, indirect) |
| Gamma Glutamyl Transferase (GGT) | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Measurement of liver enzyme levels |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Ascites | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Clinical evaluation via physical exam and ultrasound |
| Jaundice Status | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Clinical and laboratory bilirubin assessment |
| Pediatric End-Stage Liver Disease (PELD) Score | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Disease severity scoring system |
| Overall Survival (OS) | From the surgery date up to the end of the study (12 months maximum follow-up) | Survival rate since intervention |
| Anemia Status | Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months | Clinical and laboratory evaluation of anemia |
Countries
Indonesia
Contacts
Primary ContactAvriana Pety Wardani, MD, Sp. BA
Backup ContactKevin Christian Tjandra, MD
Outcome results
None listed