Recently, mesenchymal stem cell (MSC) transplantation has emerged as a promising treatment for liver cirrhosis in adults. Additionally, bone marrow-derived stem cell transplantation has shown success in treating children with biliary atresia (BA). This study aims to evaluate the efficacy of Umbilical Cord-Derived Mesenchymal Stem Cell (UC-MSC) therapy in BA through a multicentric randomized controlled trial.
Biliary atresia (BA) is the most common cause of chronic cholestasis in neonates and accounts for at least 50% of pediatric liver transplants. The incidence of BA is estimated to range from 1:5000 to 1:19000 live births. If the operation is not performed, all patients will die due to complications of liver cirrhosis. Recently, mesenchymal stem cell (MSC) transplantation has been found to be a promising treatment for liver cirrhosis in adults. Stem cell transplantation derived from bone marrow has also been successfully applied to children with BA. The aim of this study is to demonstrate the efficacy of Umbilical Cord-Derived Mesenchymal Stem Cell (UC-MSC) therapy in BA by planning a multicentric randomized controlled trial.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
64
UC-MSC transplantation will be administered twice to each patient in the study group via the hepatic artery: the first transplantation will be performed post-surgery at the beginning, and the second one will be performed 6 months later, with a dose of 1 million UC-MSC/kg.
In this group, UC-MSC will not be administered. This group serves as a passive control. The standard treatments that are routinely provided to these patients will continue to be administered.
Esenyurt State Hospital
Istanbul, Istanbul, Turkey (Türkiye)
RECRUITINGAdverse events
Adverse events will be assessed during the stem cell applications, as well as at 1 week, 1 month, 3 months, 6 months, 1 year, and 2 years after the application.
Time frame: 2 years
Pediatric End-Stage Liver Disease (PELD) score
Using PELD score (according to the suggestion of The Liver and Intestinal Organ Transplantation Committee in 2009). PELD is calculated based on three indicators: albumin (g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio). Formula: PELD = 10 \* (0.48 \* ln(Serum Bilirubin) + 1.857 \* ln(INR) - 0.687 \* ln(Albumin) + (0.436 if patient is less than 1 year old) + (0.667 if patient has growth failure)). Evaluate the result: If PELD \<10: good results If 10 \<PELD \<15: average results If PELD\> 15: bad results Albumin (Unit: g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio).
Time frame: 2 years
ALT levels (Alanine transaminase)
The levels of ALT will be closely monitored in both groups. The values for this parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
AST levels (Aspartate trasnaminase)
The levels of AST will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
Direct bilirubin levels
The levels of direct bilirubin will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
Total bilirubin levels
The levels of total bilirubin will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
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Time frame: 2 years
GGT levels (Gama glutamil transferase)
The levels of GGT will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
Liver biopsy
Liver biopsy is a crucial clinical tool for assessing the progression and severity of cirrhosis
Time frame: 2 years
Level of cirrhosis
Level of cirrhosis will be assesed with PELD score. Using PELD score (according to the suggestion of The Liver and Intestinal Organ Transplantation Committee in 2009). PELD is calculated based on three indicators: albumin (g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio). Formula: PELD = 10 \* (0.48 \* ln(Serum Bilirubin) + 1.857 \* ln(INR) - 0.687 \* ln(Albumin) + (0.436 if patient is less than 1 year old) + (0.667 if patient has growth failure)). Evaluate the result: If PELD \<10: good results If 10 \<PELD \<15: average results If PELD\> 15: bad results Albumin (Unit: g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio).
Time frame: 2 years
Re-operation rate
The patients who need further surgical intervention will be noted.
Time frame: 2 years
Liver transplantation
Liver transplantation will be evaluated. The number of patients requiring a liver transplant, along with the timing of the need, will be recorded.
Time frame: 2 years
Albumin levels
The levels of albumin will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
INR (international normalized ratio)
The levels of INR (international normalized ratio) will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.
Time frame: 2 years
Cholangitis
The number of cholangitis for each cases will be closely monitored in both groups. The frequency of cholangitis experienced by each patient will be recorded and evaluated.
Time frame: 2 years