Dear Editors,Eculizumab is a therapeutic complement C5 inhibitor approved for the treatment of atypical hemolytic uremic syndrome(aHUS),paroxysmal nocturnal proteinuria(PNH),generalized myasthenia gravis and neuromyelitis optica spectrum disorder.Correct dosing of eculizumab is important to ensure complete complement blockade.Complement activity is often monitored by measuring either formation of the C5 activation product C5b-9 in vitro using an ELISA,independent of red blood cells,or by hemolytic assays measuring hemoglobin release from erythrocytes.Both approaches allow analysis of complement activation via the classical(CP)and alternative(AP)pathways.
Lambertus P.van den HeuvelNicole C.A.J.van de KarCaroline DuineveldAndrei SarleaThea J.A.M.van der VeldenWilhelmus T.B.LiebrandSanne van KraaijCamilla SchjalmRomy BouwmeesterJack F.M.WetzelsTom E.MollnesElena B.Volokhina
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.
Alexandra AlexopoulouIliana ManiDina G TiniakosFlora KontopidouIoanna TsironiMarina NoutsouHelen PantelidakiSpyros P Dourakis
