Japan's registry transforms outcomes for infants with biliary atresia

Biliary atresia (BA) is the leading cause of liver failure in infants, marked by progressive destruction of the bile ducts. Until the late 1950s, the disease was universally fatal, with no effective treatments. The development of the Kasai portoenterostomy brought new hope, but outcomes varied widely, and many patients still required liver transplantation (LTx). Early diagnosis has remained a pressing challenge, since jaundice can be subtle and late surgery severely compromises outcomes. Differences in healthcare systems and diagnostic practices further complicate survival rates across countries. Because of these challenges, there has been an urgent need to establish a national registry to uncover patterns, refine interventions, and improve long-term survival.

A research team from the Japanese Biliary Atresia Society and Tohoku University has published (DOI: 10.1136/wjps-2025-001024) an in-depth analysis of the JBAR in World Journal of Pediatric Surgery on May 26, 2025. Since its launch in 1989, the registry has tracked 3,951 patients for up to 40 years, providing an unparalleled picture of disease progression, treatment outcomes, and survival. The study highlights how national-level data collection, combined with advances in surgery and liver transplantation, has dramatically improved outcomes for children born with this life-threatening condition.

The JBAR collects detailed information from hospitals nationwide through initial, transplantation, and follow-up surveys. Two-thirds of patients were female, with the average age at surgery around 65 days—though earlier operations produced far better outcomes. To encourage earlier detection, Japan introduced stool color cards in maternal health booklets in 2012, helping families and doctors spot warning signs sooner. By 2023, the average age at surgery had dropped to 60 days, with improved jaundice clearance rates. Between 1989 and 2023, 93% of patients underwent Kasai surgery, and 1,688 received liver transplants. Long-term follow-up revealed native liver survival rates of 50.5%, 44.4%, and 40.9% at 10, 20, and 30 years, respectively, while overall survival remained above 85%. Beyond survival, many patients experienced normal growth and development, with some reaching milestones such as marriage and childbirth. The registry also documented key complications, including cholangitis, portal hypertension, and vitamin K deficiency bleeding, while providing crucial insights into which surgical approaches yield the best outcomes. These findings have directly shaped national treatment guidelines and reinforced the importance of continuous data-driven care.

Nationwide registries like JBAR are essential in rare pediatric diseases. Our results show that timely surgery and access to liver transplantation can turn a once-fatal diagnosis into a survivable condition. Many of these children now live into adulthood, and some even start families, which was unimaginable a few decades ago. Still, challenges remain—especially improving follow-up into adulthood and expanding deceased donor transplantation in Japan. Continuing this registry is vital for advancing care and supporting patients across their lifespan".

Dr. Masaki Nio, corresponding author of the study

Japan's experience underscores the life-saving impact of national registries that integrate early detection, surgical advances, and transplantation strategies. What was once a uniformly fatal disease has been transformed into a manageable chronic condition, with survival rates now exceeding 85% into adulthood. The registry's data have informed clinical guidelines in Japan and beyond, offering a blueprint for countries to adapt within their healthcare systems. As JBAR extends to 40- and potentially 50-year follow-ups, it will provide crucial insights into the adult lives of biliary atresia survivors. This model illustrates how data-driven, collaborative medicine can change the future for rare diseases.