Paroxysmal nocturnal hemoglobinuria and liver transplantation, a new paradigm

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a type of hemolytic anemia acquired by the PIG-A gene mutation. This causes a deficiency of a complement regulatory protein, CD59, which results in hemolysis, hemoglobinuria and thrombosis (due to the release of procoagulant factors). Budd-Chiari syndrome is characteristic in these patients and has classically been considered as a contraindication for liver transplantation (LT) due to post-transplant recurrence. Since the approval of eculizumab for the treatment of PHN, disease control is possible and therefore the post-transplant recurrence of thrombotic phenomena involving the liver is avoided.