Successful treatment of noncirrhotic portal hypertension with eculizumab in paroxysmal nocturnal hemoglobinuria: A case report  

作　　者：Alexandra Alexopoulou Iliana Mani Dina G Tiniakos Flora Kontopidou Ioanna Tsironi Marina Noutsou Helen Pantelidaki Spyros P Dourakis 

机构地区：[1]2nd Department of Medicine,Medical School,National and Kapodistrian University of Athens,Hippokration General Hospital,Athens 11527,Greece [2]Institute of Cellular Medicine,Faculty of Medical Sciences,Newcastle University,Framlington Place,Newcastle NE2 4HH,United Kingdom [3]Department of Pathology,Aretaieion Hospital,National and Kapodistrian University of Athens,Athens 11528,Greece

出　　处：《World Journal of Hepatology》2019年第5期483-488,共6页世界肝病学杂志（英文版）（电子版）

摘　　要：BACKGROUND Idiopathic non-cirrhotic portal hypertension(INCPH)is mainly associated with thrombophilia in Western countries.Paroxysmal nocturnal hemoglobinuria(PNH)is a rare hematologic disease that manifests with hemolytic anemia,thrombosis,and peripheral blood cytopenias.Portal and hepatic venous thrombosis were reported in PNH.A rare case of INCPH complicating PNH is described.CASE SUMMARY A 63-year old woman with a 2-year past medical history of PNH without treatment was admitted because of jaundice and refractory ascites requiring large volume paracentesis.Liver histology revealed portal venopathy with portal fibrosis and sclerosis,nodular regenerative hyperplasia,parenchymal ischemic changes,and focal sinusoidal and perivenular fibrosis without bridging fibrosis or cirrhosis,all indicative of INCPH.The flow cytometry confirmed PNH diagnosis and eculizumab treatment was initiated.Her condition was improved gradually,bilirubin was normalized 6 months following initiation of eculizumab,and 1 year later diuretics were stopped.CONCLUSION Eculizumab improved intravascular hemolysis and reversed clinical manifestations of INCPH in a patient with paroxysmal nocturnal hemoglobinuria.BACKGROUND Idiopathic non-cirrhotic portal hypertension(INCPH) is mainly associated with thrombophilia in Western countries. Paroxysmal nocturnal hemoglobinuria(PNH) is a rare hematologic disease that manifests with hemolytic anemia,thrombosis, and peripheral blood cytopenias. Portal and hepatic venous thrombosis were reported in PNH. A rare case of INCPH complicating PNH is described.CASE SUMMARY A 63-year old woman with a 2-year past medical history of PNH without treatment was admitted because of jaundice and refractory ascites requiring large volume paracentesis. Liver histology revealed portal venopathy with portal fibrosis and sclerosis, nodular regenerative hyperplasia, parenchymal ischemic changes, and focal sinusoidal and perivenular fibrosis without bridging fibrosis or cirrhosis, all indicative of INCPH. The flow cytometry confirmed PNH diagnosis and eculizumab treatment was initiated. Her condition was improved gradually, bilirubin was normalized 6 months following initiation of eculizumab,and 1 year later diuretics were stopped.CONCLUSION Eculizumab improved intravascular hemolysis and reversed clinical manifestations of INCPH in a patient with paroxysmal nocturnal hemoglobinuria.

关 键 词：PAROXYSMAL NOCTURNAL HEMOGLOBINURIA IDIOPATHIC non-cirrhotic portal hypertension ECULIZUMAB Case report 

分 类 号：R[医药卫生]