TY - JOUR
T1 - The Management of Paroxysmal Nocturnal Hemoglobinuria
T2 - Recent Advances in Diagnosis and Treatment and New Hope for Patients
AU - Young, Neal S.
AU - Meyers, Gabrielle
AU - Schrezenmeier, Hubert
AU - Hillmen, Peter
AU - Hill, Anita
N1 - Funding Information:
While many physicians have little or no experience treating patients with PNH, there have been many recent advances in the management of this disease. These advances were discussed by a panel of international experts at a satellite symposium that preceded the 49th American Society of Hematology Annual Meeting in Atlanta, GA. This symposium was sponsored by an educational grant from Alexion Pharmaceuticals, Inc.
PY - 2009/1
Y1 - 2009/1
N2 - PNH is a rare clonal hematopoietic stem disorder clinically characterized by the triad of chronic complement-mediated hemolysis, thrombosis, and bone marrow failure. While median survival has improved when historical data are compared to more recent data, thrombosis, the major cause of death in PNH, is still observed in approximately 40% of patients. The symptoms associated with this disorder-including fatigue, pain, esophageal spasm, and erectile dysfunction-are often severe and disabling. While PNH may be a curiosity to the physician, it forces the majority of patients to significantly modify their lives. Transplantation represents a curative option; however, the risks associated with this option are not insignificant. Eculizumab has been shown to significantly reduce hemolysis, improve anemia, reduce transfusion requirements, and significantly improve fatigue and other QoL scores. Clearly, targeted complement inhibition by eculizumab has the promise to improve the lives of patients with PNH.
AB - PNH is a rare clonal hematopoietic stem disorder clinically characterized by the triad of chronic complement-mediated hemolysis, thrombosis, and bone marrow failure. While median survival has improved when historical data are compared to more recent data, thrombosis, the major cause of death in PNH, is still observed in approximately 40% of patients. The symptoms associated with this disorder-including fatigue, pain, esophageal spasm, and erectile dysfunction-are often severe and disabling. While PNH may be a curiosity to the physician, it forces the majority of patients to significantly modify their lives. Transplantation represents a curative option; however, the risks associated with this option are not insignificant. Eculizumab has been shown to significantly reduce hemolysis, improve anemia, reduce transfusion requirements, and significantly improve fatigue and other QoL scores. Clearly, targeted complement inhibition by eculizumab has the promise to improve the lives of patients with PNH.
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U2 - 10.1053/j.seminhematol.2008.11.004
DO - 10.1053/j.seminhematol.2008.11.004
M3 - Article
C2 - 19171207
AN - SCOPUS:58549084856
SN - 0037-1963
VL - 46
SP - S1-S16
JO - Seminars in hematology
JF - Seminars in hematology
IS - SUPPL. 1
ER -