Evaluation of Clinical Outcomes in Biliary Atresia Patients Undergoing Kasai Surgery Before 90 Days of Age With Umbilical Cord-Derived Mesenchymal Stem Cell Therapy
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Serum Albumin Level
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 0 Days 至 90 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •Infants with severe malnutrition.
- •Infants with major congenital anomalies other than biliary atresia.
- •Infants with positive tumor markers.
研究组 & 干预措施
Intervention Group (UC-MSC Group)
Participants in this group are infants diagnosed with biliary atresia who undergo Kasai portoenterostomy before 90 days of age. During the operation, they receive intraoperative trans-portal vein injection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) at a dose of 20 million cells diluted in 5 mL autologous plasma.
干预措施: Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs) (Biological)
Intervention Group (UC-MSC Group)
Participants in this group are infants diagnosed with biliary atresia who undergo Kasai portoenterostomy before 90 days of age. During the operation, they receive intraoperative trans-portal vein injection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) at a dose of 20 million cells diluted in 5 mL autologous plasma.
干预措施: Kasai Portoenterostomy (Procedure)
Control Group (Historical Control Group)
Participants in this group are historical patients who previously underwent Kasai portoenterostomy before 90 days of age at the same institution but did not receive UC-MSC therapy. Data for this group is collected retrospectively.
干预措施: Kasai Portoenterostomy (Procedure)
结局指标
主要结局
Serum Albumin Level
时间窗: 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
时间窗: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Measurement of bilirubin levels (total, direct, indirect)
Gamma Glutamyl Transferase (GGT)
时间窗: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Measurement of liver enzyme levels
Aspartate Aminotransferase (AST)
时间窗: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Liver injury marker
Alanine Aminotransferase (ALT)
时间窗: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Liver injury marker
Prothrombin Time (PT), aPTT, INR
时间窗: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Coagulation parameters
Alpha Fetoprotein (AFP)
时间窗: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Tumor marker
Alkaline Phosphatase (ALP)
时间窗: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Liver and bone function marker
Lactate Dehydrogenase (LDH)
时间窗: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Tissue injury marker
次要结局
- Jaundice Status(Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months)
- Anemia Status(Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months)
- Ascites(Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months)
- Pediatric End-Stage Liver Disease (PELD) Score(Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months)
- Overall Survival (OS)(From the surgery date up to the end of the study (12 months maximum follow-up))
研究者
Avriana Pety Wardani
MD, Sp. BA
Dr. Kariadi General Hospital Medical Center