To evaluate the safety and early outcomes of autologous bone marrow mononuclear cell (BMMNC) infusion for liver cirrhosis due to biliary atresia (BA) after Kasai operation. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery.
Biliary atresia (BA) is a progressive fibro-obliterative cholangiopathy and a fatal disease. Without surgery, children with BA rarely survive beyond three years of age. The reported prevalence of BA ranges from 1 in 9640 to 1 in 19,500 live births. In the past, most children with a "non-correctable" type of BA died without adequate treatment. Recently, stem cell administration has been applied in adults with liver cirrhosis and has shown promising outcomes. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery. Serum bilirubin, albumin, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, and prothrombin time were monitored at baseline, three months, six months and 12 months after the transplantation. Esophagoscopies and liver biopsies were performed in patients whose parents provided consent. This study aimed to evaluate both safety and hepatic function after BMMNC administration in these children.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
One administration of autologous bone marrow mononuclear cells via the hepatic artery
Vinmec Research Institute of Stem Cell and Gene Technology
Hanoi, Vietnam
Adverse events and serious adverse events
To assess safety, the number of AEs or SAEs during stem cell administration (72 h) at 1 month, 3 months, 6 months, and 9 months after discharge will be evaluated
Time frame: up to the 12-month period following treatment
The changes in cholestasis
Using Total Bilirubin (units: mg/dL) to measure the changes in cholestasis
Time frame: up to the 12-month period following treatment
The changes in Liver function using Aspart transaminase
Using AST (Aspart transaminase) (units: U/L) to measure the changes in liver function.
Time frame: up to the 12-month period following treatment
The changes in Liver function using Alanine transaminase
Using ALT (Alanine transaminase) (units: U/L) to measure the changes in liver function.
Time frame: up to the 12-month period following treatment
The changes in Liver function using Gamma GT
Using GGT (Gamma GT) (units: U/L) to measure the changes in liver function.
Time frame: up to the 12-month period following treatment
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
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: up to the 12-month period following treatment
The changes in liver biopsy
Liver biopsy is a powerful clinical tool to evaluate the changes in the level of cirrhosis.
Time frame: up to the 12-month period following treatment