Biliary atresia (BA) is a rare congenital condition in children, occurring in approximately 1 in 8,000 to 1 in 18,000 live births. However, it is the most common cause of cholestasis (bile flow obstruction) in infants. The disease leads to complications such as biliary cirrhosis and end-stage liver disease, with about 60–80% of children with BA eventually requiring liver transplantation. In Vietnam, liver transplantation remains a major challenge—not only due to technical complexity, but also because of limited organ donors and high costs, making access difficult for many patients.
Combining stem cell therapy with Kasai surgery is considered a modern and advanced treatment approach for BA. This method aims to support the treatment of the disease, improve quality of life, and extend the survival time with the child’s native liver.
Although BA is rare, it is the leading cause of neonatal cholestasis, especially in children in Asia. It is characterized by progressive, spontaneous inflammation of the bile ducts, resulting in biliary cirrhosis and eventually liver failure. Common symptoms include prolonged jaundice after birth and pale-colored stools. These signs are easily mistaken for other liver and biliary diseases, often leading to delayed diagnosis and reduced treatment success.
To date, the only standard treatment for BA is the Kasai portoenterostomy, a surgical procedure designed to restore bile flow from the liver to the intestines. However, this is typically a temporary solution. Statistics show that 60–80% of affected children still require a liver transplant after surgery due to continued liver fibrosis and declining liver function. While liver transplantation is a highly effective treatment and has been routinely performed at the Vietnam National Children’s Hospital, it still faces numerous barriers: shortage of organ donors, high costs, and complex post-operative care.
Liver transplantation has become a routine procedure at the Vietnam National Children’s Hospital
Using stem cell therapy in treatment aligns with the development trends of modern medicine. Research has shown that stem cells can promote liver regeneration through various mechanisms:
- Replacing damaged and non-functional liver cells
- Restructuring the liver microenvironment
- Anti-fibrotic and anti-inflammatory effects
- Promoting cell proliferation
- Stimulating liver regeneration and reducing fibrosis
- Enhancing angiogenesis (formation of new blood vessels), improving circulation and liver function
Illustration: Regenerative capacity of autologous bone marrow stem cells
Between 2020 and 2025, the National-Level Research Project titled “Application of Autologous Bone Marrow Stem Cells to Support the Treatment of Biliary Atresia in Children” was implemented at the Vietnam National Children’s Hospital. This project opened promising prospects for improving survival outcomes in children with BA. The research team developed and standardized protocols for early diagnosis, as well as procedures for isolation, storage, and use of bone marrow stem cells—ensuring safety and effectiveness.
Follow-up results showed that combining stem cell therapy with the Kasai procedure improved post-surgical bile drainage, enhanced liver function, reduced the rate of biliary tract infections and early liver failure. Children who received the combined therapy had a significantly longer survival time with their native liver compared to those who did not.
This research marks a breakthrough in the application of stem cell therapy, bringing new hope to children suffering from complex hepatobiliary diseases.
