A closer look at paroxysmal nocturnal hemoglobinuria.
Knowledge of the molecular mechanisms leading to the paroxysmal nocturnal hemoglobinuria (PNH) phenotypes has substantially increased in the past two decades. The associated intravascular hemolysis, hypercoagulablilty, and bone marrow failure result in a wide range of clinical sequlae. Although treatment has usually been symptomatic through several modalities and rarely curative through hematopoietic cell transplantation, recent development of the novel targeted therapeutic agent eculizumab has offered new promises for this highly morbid and fatal disease. This review summarizes current knowledge of the pathophysiology, diagnostic modalities, clinical implications, and treatment approaches of patients with PNH.
Rachidi
Saleh
S
Department of Internal Medicine, Division of Hematology & Oncology, American University of Beirut Medical Center, Beirut, Lebanon.
Musallam
Khaled M
KM
Taher
Ali T
AT
D000743:Anemia, Hemolytic / Q000209:etiology / Q000503:physiopathology; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D061067:Antibodies, Monoclonal, Humanized; D001777:Blood Coagulation / Q000502:physiology; D001778:Blood Coagulation Disorders / Q000209:etiology / Q000503:physiopathology; D006457:Hemoglobinuria, Paroxysmal / Q000150:complications / Q000175:diagnosis / Q000235:genetics / Q000503:physiopathology* / Q000628:therapy; D006461:Hemolysis / Q000502:physiology; D006801:Humans; D007674:Kidney Diseases / Q000209:etiology / Q000503:physiopathology; D011379:Prognosis; D013927:Thrombosis / Q000209:etiology / Q000503:physiopathology
D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab