Case Report
Copyright ©The Author(s) 2019. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Hepatol. May 27, 2019; 11(5): 483-488
Published online May 27, 2019. doi: 10.4254/wjh.v11.i5.483
Successful treatment of noncirrhotic portal hypertension with eculizumab in paroxysmal nocturnal hemoglobinuria: A case report
Alexandra Alexopoulou, Iliana Mani, Dina G Tiniakos, Flora Kontopidou, Ioanna Tsironi, Marina Noutsou, Helen Pantelidaki, Spyros P Dourakis
Alexandra Alexopoulou, Iliana Mani, Flora Kontopidou, Ioanna Tsironi, Marina Noutsou, Helen Pantelidaki, Spyros P Dourakis, 2nd Department of Medicine, Medical School, National and Kapodistrian University of Athens, Hippokration General Hospital, Athens 11527, Greece
Dina G Tiniakos, Institute of Cellular Medicine, Faculty of Medical Sciences, Newcastle University, Framlington Place, Newcastle NE2 4HH, United Kingdom
Dina G Tiniakos, Department of Pathology, Aretaieion Hospital, National and Kapodistrian University of Athens, Athens 11528, Greece
Author contributions: Alexopoulou A and Mani I reviewed the literature and contributed to manuscript drafting; Tiniakos DG analyzed and interpreted the histological findings and contributed to manuscript drafting; Kontopidou F performed the hematological consultation and contributed to manuscript drafting; Pantelidaki H, Noutsou M, and Tsironi I reviewed the literature and contributed to manuscript drafting; Dourakis SP was responsible for the revision of the manuscript for important intellectual content; all authors issued final approval for the version to be submitted.
Informed consent statement: Informed written consent was obtained from the patient for publication of this report and any accompanying images.
Conflict-of-interest statement: The authors declare that they have no conflict of interest.
CARE Checklist (2016) statement: The authors have read the CARE Checklist (2013), and the manuscript was prepared and revised according to the CARE Checklist (2016).
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See:
Corresponding author: Alexandra Alexopoulou, MD, PhD, Associate Professor, 2nd Department of Medicine, Medical School, National and Kapodistrian University of Athens, Hippokration General Hospital, 114 Vas Sophias St, Athens 11527, Greece.
Telephone: +30-213-2088178 Fax: +30-210-7706871
Received: March 6, 2019
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.

Keywords: Paroxysmal nocturnal hemoglobinuria, Idiopathic non-cirrhotic portal hypertension, Eculizumab, Case report

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.