What is the treatment for Budd-Chiari syndrome?

Treatment of Budd-Chiari Syndrome

The treatment of Budd-Chiari syndrome follows a sequential algorithm starting with anticoagulation therapy, followed by angioplasty if feasible, then TIPS (transjugular intrahepatic portosystemic shunt) if medical treatment fails, and ultimately liver transplantation for those who fail other interventions. 1

Initial Diagnosis and Evaluation

• Diagnosis is established through unequivocal radiological confirmation of hepatic venous outflow obstruction
• Doppler ultrasound is the first-line investigation with >75% sensitivity 1
• If ultrasound is inconclusive, MRI or CT evaluation should be used 1
• Venography is recommended when diagnosis remains uncertain or to characterize anatomy before treatment

Underlying Cause Investigation

• All patients should be investigated for:
  • Inherited thrombophilia (protein S, protein C, antithrombin levels, Factor V Leiden mutation, prothrombin G20210A gene variant)
  • Acquired thrombophilia (antiphospholipid antibodies)
  • Myeloproliferative neoplasms (JAK2V617F mutation, calreticulin mutation)
  • Paroxysmal nocturnal hemoglobinuria
  • Autoimmune disorders
  • Local factors (intra-abdominal inflammatory conditions, malignancies) 1

Treatment Algorithm

Step 1: Medical Treatment

• Immediate anticoagulation therapy with low molecular weight heparin for 5-7 days, followed by oral vitamin K antagonists (target INR 2-3) 1, 2
• Treat the underlying cause (particularly myeloproliferative disorders) 1
• Manage complications of portal hypertension (ascites, varices) using standard protocols for cirrhosis 1

Step 2: Angioplasty/Stenting

• For patients with short, singular stenosis of hepatic veins or IVC 1, 2
• Stent placement reduces re-stenosis rates after angioplasty 2
• Local thrombolysis may be combined with angioplasty in cases of recent thrombosis 2

Step 3: TIPS Procedure

• Indicated when:
  • Medical treatment and angioplasty fail
  • Clinical manifestations persist despite angioplasty
  • Fulminant Budd-Chiari syndrome 1, 2
• TIPS should be performed at specialized centers with expertise in vascular liver diseases 1
• Pre-TIPS MRI with hepatospecific contrast agents is essential to detect and characterize liver nodules 1
• Post-TIPS Doppler ultrasound should be performed early and then every 6 months to detect thrombosis or TIPS dysfunction 1

Step 4: Liver Transplantation

• Indicated for:
  • TIPS failure
  • Fulminant hepatic failure due to Budd-Chiari syndrome 1
• Evaluation for transplantation eligibility should be conducted as soon as TIPS is indicated 1
• Transplantation should be considered only after excluding underlying malignancy 1

Special Considerations

• Approximately 40% of patients with Budd-Chiari syndrome have hypervascular liver nodules (focal nodular hyperplasia, adenomas, and rarely HCC) 1
• The typical radiological criteria for HCC diagnosis may not apply in Budd-Chiari syndrome 1
• Long-term anticoagulation is recommended for patients with underlying myeloproliferative disorders 1

Outcomes and Prognosis

• With the stepwise therapeutic approach, 5-year survival rates approach 70% 3
• Prognosis depends on the severity of liver disease and timely intervention 3
• Early recognition and referral to specialized centers is critical for optimal outcomes 4

Pitfalls to Avoid

• Misdiagnosis is common in resource-limited settings; consider Budd-Chiari in any patient with ascites and liver dysfunction 5
• Do not delay anticoagulation therapy once diagnosis is established 1
• Do not overlook screening for underlying prothrombotic disorders, as identification of one risk factor should not deter from looking for additional factors 1
• Avoid delaying referral to a transplant center for patients with fulminant hepatic failure 1