Narrative Review: Paroxysmal Nocturnal Hemoglobinuria: The Physiology of Complement-Related Hemolytic Anemia

Reviews15 April 2008Narrative Review: Paroxysmal Nocturnal Hemoglobinuria: The Physiology of Complement-Related Hemolytic AnemiaRobert A. Brodsky, MDRobert A. Brodsky, MDFrom Johns Hopkins Medical Institutions, Baltimore, Maryland.Search for more papers by this authorAuthor, Article, and Disclosure Informationhttps://doi.org/10.7326/0003-4819-148-8-200804150-00003 SectionsAboutFull TextPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareFacebookTwitterLinkedInRedditEmail Clinical PrinciplesParoxysmal nocturnal hemoglobinuria (PNH) is a rare hematopoietic stem-cell disorder caused by a somatic mutation in a gene known as phosphatidylinositol glycan class A (PIGA). It may arise de novo or in the setting of acquired aplastic anemia.Clinical presentation can include hemolytic anemia, hemoglobinuria, thrombosis, severe fatigue, abdominal pain, and esophageal spasm.Thrombosis, the leading cause of death from PNH, most commonly occurs in abdominal and cerebral veins.Therapeutic options include supportive care, bone marrow transplantation, and monoclonal antibody therapy with the terminal complement inhibitor eculizumab.Pathophysiologic PrinciplesThe product of the PIGA gene is required for the biosynthesis of a glycolipid anchor ...References1. Brodsky RA. Paroxysmal nocturnal hemoglobinuria.. In: Hoffman R, Benz EJ Jr, Shattil SJ, Furie B, Cohen HJ, Silberstein LE, et al., eds. Hematology: Basic Principles and Practice. 4 ed. Philadelphia: Elsevier Churchill Livingston; 2005:419-27. Google Scholar2. Moyo VM, Mukhina GL, Garrett ES, Brodsky RA. Natural history of paroxysmal nocturnal haemoglobinuria using modern diagnostic assays. 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Honoraria: Alexion Pharmaceuticals. Grants received: National Institutes of Health. Patents received: U.S. patent number 6 393 095 B1 for detection of GPI-anchored proteins.Corresponding Author: Robert A. Brodsky, MD, Division of Hematology, Johns Hopkins Medicine, Ross Research Building Room 1025, 720 Rutland Avenue, Baltimore, MD 21205-2196; e-mail, [email protected]edu. 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