Autologous Bone Marrow Mononuclear Stem Cell for Children Suffering From Liver Cirrhosis Due to Biliary Atresia

Study Overview
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Brief Summary
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The aim of this study was to evaluate the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation for Children Suffering from Liver Cirrhosis Due to Biliary Atresia

Detailed Description
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This study is ferformed to evaluate the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation in the management of Liver Cirrhosis Due to Biliary Atresia after Kasai's operation of 20 patients at Vinmec Research Institute of Stem Cell and Gene Technology, Hanoi, Vietnam from January 2017 to December 2018

Official Title
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Outcomes of Autologous Bone Marrow Mononuclear Stem Cell (BMMC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia After Kasai's Operation: An Open Label Uncontrolled Clinical Trial

Conditions
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Liver Cirrhosis, Biliary

Intervention / Treatment
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* Combination Product: Autologous BMMC transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children were diagnosed with Liver Cirrhosis Due to biliary atresia after Kasai's operation 1 year old or older Patients with manifestation of cirrhosis after Kasai's operation: hepatomegaly, congestive splenomegaly, elevated liver enzymes, Esophageal Varices (based on Endoscopic Diagnosis), cirrhosis (based on liver biopsy) Exclusion Criteria: Under 1 year old patients Epilepsy Coagulation disorders Allergy to anesthetic agents Severe health conditions such as cancer, failure of heart, lung, liver or kidney Active infections Severe psychiatric disorders

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 15 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Autologous BMMC transplantation<br>Stem cell transplantations include 2 administrations of autologous bone marrow mononuclear cells via the hepatic artery at baseline and 6 months afterward | Combination Product: Autologous BMMC transplantation<br>* Stem cell transplantation<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assess the changes in cholestasis | Using Bilirubin (total, direct and indirect) (units: mg/dL) to measure the changes in cholestasis | Baseline, 3, 6 months and 12 months after transplantation | 
| Assess the changes in Liver function | Using AST (Aspart transaminase) and ALT (Alanin transaminase) (units: U/L), GGT (Gamma GT) (units: UI/L), and Bilirubin (units: mg/dL) to measure the changes in Liver function | Baseline, 3, 6 months and 12 months after transplantation | 
| Assess the changes in level of cirrhosis | 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). | Baseline, 3, 6 months and 12 months after transplantation | 
| Assess the changes in liver biopsy | Liver biopsy, in combination with history and physical examination data, is a powerful clinical tool for diagnosing and treating liver disease to evaluate the changes in liver biopsy | Baseline, 3, 6 months and 12 months after transplantation | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Autologous bone marrow Transplantation, biliary atresia, Kasai's operation, liver cirrhosis