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Successful liver transplantation for Budd‐Chiari syndrome in a patient with paroxysmal nocturnal hemoglobinuria treated with the anti‐complement antibody eculizumab
Author(s) -
Singer Andrew L.,
Locke Jamye E.,
Stewart Zoe A.,
Lonze Bonnie E.,
Hamilton James P.,
Scudiere Jennifer R.,
Anders Robert A.,
Rother Russell P.,
Brodsky Robert A.,
Cameron Andrew M.
Publication year - 2009
Publication title -
liver transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.814
H-Index - 150
eISSN - 1527-6473
pISSN - 1527-6465
DOI - 10.1002/lt.21714
Subject(s) - paroxysmal nocturnal hemoglobinuria , medicine , eculizumab , liver transplantation , hemoglobinuria , cd59 , gastroenterology , transplantation , aplastic anemia , portal vein thrombosis , immunology , cirrhosis , complement system , anemia , antibody , bone marrow
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hemolytic anemia caused by somatic mutations in the phosphatidylinositol glycan‐complementation class A gene and the resulting absence of a key complement regulatory protein, CD59. Affected red blood cells in patients with PNH undergo intravascular complement‐mediated lysis with resulting anemia, hemoglobinuria, and venous thromboses. Hepatic venous outflow thrombosis [Budd‐Chiari syndrome (BCS)] is especially common in PNH patients and often fatal. The few case reports of outcomes in patients undergoing liver transplant for BCS secondary to PNH detail instances of recurrent BCS as well as early thrombotic portal vein occlusion and hepatic artery thrombosis requiring retransplantation. PNH is therefore generally considered a contraindication to liver transplantation. Here we present the first report of a patient with PNH and BCS undergoing successful liver transplantation while receiving eculizumab, a humanized monoclonal antibody that blocks the activation of the terminal complement at C5. Liver Transpl 15:540–543, 2009. © 2009 AASLD.