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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Stem Cell Application Group | Experimental | In this group, 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. The applications will be applied after kasai portoenterostomy. |
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| Control | Sham Comparator | In this group, UC-MSC will not be administered. This group serves as a passive control. The standard treatments that are routinely provided to Biliary Atresia patients will continue to be administered. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Stem Cell | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse 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. | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| 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). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Mustafa Azizoglu, MD, PhD | Contact | +905447448244 | mdmazizoglu@gmail.com |
| Name | Affiliation | Role |
|---|---|---|
| Mustafa Azizoglu, MD, PhD | Esenyurt State Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Esenyurt State Hospital | Recruiting | Istanbul | Istanbul | 34340 | Turkey (Türkiye) |
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| ID | Term |
|---|---|
| D001656 | Biliary Atresia |
| D008103 | Liver Cirrhosis |
| ID | Term |
|---|---|
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
| D004065 | Digestive System Abnormalities |
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This study is designed as a multicentric randomized controlled trial (RCT). Patients will be randomly divided into two groups. Treatment Group: 30 Kasai-operated BA patients who will receive UC-MSC transplantation, and Control Group: 30 BA patients who will only undergo Kasai operation. 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.
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| Control | Other | 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. |
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| 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. | 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. | 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. | 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. | 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. | 2 years |
| Liver biopsy | Liver biopsy is a crucial clinical tool for assessing the progression and severity of cirrhosis | 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). | 2 years |
| Re-operation rate | The patients who need further surgical intervention will be noted. | 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. | 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. | 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. | 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. | 2 years |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008107 | Liver Diseases |
| D005355 | Fibrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |