What are potential research topics for a DM Pediatric Hepatology project on biliary atresia?

Potential Research Topics for DM Pediatric Hepatology on Biliary Atresia

The most promising research topics for a DM Pediatric Hepatology project on biliary atresia should focus on improving early diagnosis and optimizing timing of interventions to maximize survival and quality of life outcomes.

Pathophysiology and Early Diagnosis Research

• Investigation of potential infectious or genetic mechanisms in biliary atresia pathogenesis, as no conclusive agent has been identified despite evidence supporting multiple possible causes 1
• Development of improved screening methods for earlier detection of biliary atresia, as diagnosis before 2 months of age significantly improves outcomes with Kasai portoenterostomy 2
• Evaluation of biomarkers that could predict success of Kasai procedure or need for early transplantation 2

Optimizing Kasai Portoenterostomy Outcomes

• Standardization of post-Kasai medication regimens, particularly ursodeoxycholic acid dosing to promote bile flow, which currently lacks standardization 2, 3
• Investigation of optimal prophylactic antibiotic regimens to reduce cholangitis rates post-Kasai 2, 3
• Evaluation of nutritional interventions to improve outcomes in cholestatic children with biliary atresia, as aggressive nutritional support is needed to improve outcomes 2
• Research on factors that predict successful bile drainage after Kasai procedure, as patients with bilirubin below 2 mg/dL within 3 months post-procedure have better transplant-free survival 2, 3

Liver Transplantation Innovations

• Comparison of outcomes between primary living donor liver transplantation versus traditional Kasai-first approach in specific patient populations 4
• Investigation of optimal timing for liver transplantation in biliary atresia patients with failed Kasai procedures 5
• Research on technical innovations in transplantation techniques for infants with biliary atresia, including reduced-size grafts and living-related transplantation 6
• Evaluation of strategies to bridge the gap for patients awaiting transplantation, especially those under 2 years of age 6

Long-term Outcomes and Complications

• Investigation of long-term complications in biliary atresia survivors, particularly manifestations of portal hypertension which affect approximately two-thirds of long-term survivors 7
• Research on quality of life outcomes in biliary atresia patients who survive more than 10 years post-Kasai without transplantation 7
• Evaluation of neurodevelopmental outcomes in biliary atresia patients after both Kasai procedure and liver transplantation 2
• Study of growth patterns and nutritional status in long-term survivors of biliary atresia 2

Management of Complications

• Research on optimal management of portal hypertension in biliary atresia patients, as beta-blocker therapy for primary prophylaxis remains controversial in children 2
• Investigation of strategies to prevent and treat recurrent cholangitis in post-Kasai patients 3
• Evaluation of hepatocellular carcinoma risk and surveillance strategies in biliary atresia patients, as cases have been reported even in children under 1 year 2

Novel Therapeutic Approaches

• Investigation of potential medical therapies that could delay or prevent progression to end-stage liver disease 3
• Research on immunomodulatory approaches, as inflammatory processes appear to play a role in the pathogenesis 1
• Evaluation of biliary diversion techniques as alternatives to transplantation in selected patients 2