Published online May 27, 2019. doi: 10.4254/wjh.v11.i5.483
Peer-review started: March 7, 2019
First decision: April 11, 2019
Revised: April 25, 2019
Accepted: May 20, 2019
Article in press: May 20, 2019
Published online: May 27, 2019
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.
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.
Eculizumab improved intravascular hemolysis and reversed clinical manifestations of INCPH in a patient with paroxysmal nocturnal hemoglobinuria.
Core tip Idiopathic non-cirrhotic portal hypertension diagnosis is based on liver histology, the clinical signs of portal hypertension, and the exclusion of chronic liver diseases in the absence of portal and hepatic venous thrombosis. We describe a patient with paroxysmal nocturnal hemoglobinuria who presented with intravascular hemolysis, jaundice, and refractory ascites. Abdominal portosystemic collaterals were evident in imaging without varices in endoscopy. Liver histology demonstrated portal venopathy, features of nodular regenerative hyperplasia, and ischemic parenchymal changes without cirrhosis, all indicative of idiopathic non-cirrhotic portal hypertension. Eculizumab treatment achieved improvement of hemolysis and reversion of biochemical and clinical manifestations of liver disease.