Diagnosis and management of PNH: Review and recommendations from a Belgian expert panel

European Journal of Haematology
Timothy DevosBérangère Devalet

Abstract

Despite its considerable morbidity and mortality, paroxysmal nocturnal haemoglobinuria (PNH) is still underdiagnosed. Patients with PNH can suffer from cardiovascular, gastrointestinal, neurological or haematological symptoms and refer to several specialists. The aim of this paper is to review the diagnosis and the management of PNH patients, with the primary focus on identifying high-risk groups. Additionally, the implementation and prognostic value of the defined high-risk groups will be commented on and the management of PNH patients is discussed from a Belgian perspective. Finally, based on the available data, recommendations are provided. Eculizumab is a potent C5 complement inhibitor and reduces intravascular haemolysis and thrombosis in PNH patients and improves their quality of life. As thrombosis is the main cause of death in PNH patients, identifying high-risk PNH patients in need of therapy is essential. Currently, novel complement inhibitors are in development and the first data seem promising. Another challenge in PNH is to identify new markers to assess the thrombotic risk to achieve a better risk-based prophylactic anti-thrombotic management. Finally, because of the low prevalence of the disease, PNH patients sho...Continue Reading

References

Jan 1, 1986·AJR. American Journal of Roentgenology·G P MulopulosJ W Clark
Nov 9, 1995·The New England Journal of Medicine·P HillmenJ V Dacie
Apr 29, 1999·Proceedings of the National Academy of Sciences of the United States of America·D J AratenL Luzzatto
Sep 25, 1999·Annals of Internal Medicine·D E DunnN S Young
Oct 1, 2003·Blood Coagulation & Fibrinolysis : an International Journal in Haemostasis and Thrombosis·Martin GrünewaldMartin Griesshammer
Jun 8, 2004·British Journal of Haematology·Jan SimakJaroslav G Vostal
Jun 17, 2004·British Journal of Haematology·Victor M MoyoRobert A Brodsky
Feb 3, 2005·Blood·Rong HuRobert A Brodsky
Jul 30, 2005·Blood·Charles ParkerUNKNOWN International PNH Interest Group
Mar 7, 2006·Journal of Vascular and Interventional Radiology : JVIR·George P KuoHyun S Kim
Aug 22, 2006·Obstetrical & Gynecological Survey·Stefania FieniRoberto Delsignore
Sep 23, 2006·The New England Journal of Medicine·Peter HillmenLucio Luzzatto
Nov 23, 2006·Blood Reviews·Andrea PiccinOwen P Smith
Feb 27, 2007·Journal of Thrombosis and Haemostasis : JTH·P D ZiakasM Voulgarelis
Jun 7, 2008·Blood·Régis Peffault de LatourUNKNOWN French Association of Young Hematologists
Mar 12, 2009·Thrombosis and Haemostasis·Laurent BurnierAnne Angelillo-Scherrer
Aug 19, 2009·European Journal of Haematology·Manisha MadkaikarKanjaksha Ghosh
Jan 19, 2011·British Journal of Haematology·Yukinori KozumaHaruhiko Ninomiya
Apr 2, 2011·Seminars in Thrombosis and Hemostasis·Ilene C Weitz
May 14, 2011·Circulation Research·A Phillip Owens, Nigel Mackman
Jun 29, 2011·Clinical and Translational Science·Jeffrey J Pu, Robert A Brodsky
Apr 14, 2012·Cytometry. Part B, Clinical Cytometry·D Robert SutherlandAndrea Illingworth
Jan 11, 2013·Seminars in Thrombosis and Hemostasis·Romaric Lacroix, Francoise Dignat-George
Apr 24, 2013·Blood·Anita HillPeter Hillmen
Jun 12, 2013·Nephron. Clinical Practice·S HussainJ Kazi

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Citations

Mar 24, 2020·International Journal of Laboratory Hematology·Miriam ArcaviAlberto Lazarowski
Sep 6, 2020·International Journal of Hematology·Yukinori NakamuraTakehiko Mori
Nov 2, 2019·Transfusion Medicine Reviews·Lillemor Skattum
Sep 28, 2021·American Journal of Kidney Diseases : the Official Journal of the National Kidney Foundation·Andrew S BombackJoshua M Thurman

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