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Experimental Hematology and Oncology

, 1:26

First Online: 06 September 2012Received: 17 July 2012Accepted: 23 August 2012

Abstract

Paroxysmal nocturnal hemoglobinuria PNH is a progressive, life-threatening disorder characterized by chronic intravascular hemolysis caused by uncontrolled complement activation. Hepatic vein thrombosis Budd-Chiari syndrome is common in PNH patients. This case report describes the response to eculizumab a humanized monoclonal antibody that inhibits terminal complement activation in a 25-year-old male with progressive liver function deterioration despite standard anticoagulation therapy and transjugular intrahepatic porto-systemic shunt. The patient presented with anemia, severe thrombocytopenia, headache, abdominal pain, and distention. He was diagnosed with PNH, cerebral vein thrombosis, and Budd-Chiari syndrome. Despite adequate anticoagulation, diuretic administration, and placement of a transjugular shunt, additional thrombotic events and progressive liver damage were observed. Eculizumab therapy was initiated, resulting in rapid blockade of intravascular hemolysis, increased platelet counts, ascites resolution, and liver function recovery, all of which are presently sustained. Since starting eculizumab the patient has had no further thrombotic events and his quality of life has dramatically improved. This is the first report to confirm the role of complement-mediated injury in the progression of Budd-Chiari syndrome in a patient with PNH. This case shows that terminal complement blockade with eculizumab can reverse progressive thromboses and hepatic failure that is unresponsive to anticoagulation therapy and suggests that early initiation of eculizumab should be included in the therapeutic regimen of patients with PNH-related Budd-Chiari syndrome.

KeywordsBudd-Chiari syndrome Complement inhibition Eculizumab Paroxysmal nocturnal hemoglobinuria AbbreviationsPNHParoxysmal nocturnal hemoglobinuria

GPIGlycosylphosphatidylinositol

BCSBudd-Chiari syndrome

TIPSTransjugular intrahepatic porto-systemic shunt

LDHLactate dehydrogenase

MRIMagnetic resonance imaging

MRAMagnetic resonance angiography.

Electronic supplementary materialThe online version of this article doi:10.1186-2162-3619-1-26 contains supplementary material, which is available to authorized users.

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Autor: Andrés Brodsky - Octavio Mazzocchi - Fabiana Sánchez - Gus Khursigara - Suneil Malhotra - Mariano Volpacchio

Fuente: https://link.springer.com/







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