Welcome to the NORD Physician Guide to Paroxysmal Nocturnal Hemoglobinuria (PNH). The NORD Online Physician Guides are written for physicians by physicians with expertise on specific rare disorders. This guide was written by Carlos M. De Castro, III, MD, Professor of Medicine, Duke University School of Medicine and Wendell Rosse, MD, Professor Emeritus of Medicine, Florence McAlister Professor Emeritus of Medicine, Duke University School of Medicine. (see acknowledgements for additional information).
NORD is a nonprofit organization representing all patients and families affected by rare diseases. The information NORD provides to medical professionals is intended to facilitate timely diagnosis and treatment for patients.
PNH is an acquired hematological disorder that can lead to debilitating and life-threatening complications due to chronic intravascular hemolysis and thrombophilia.
PNH patients are anemic due to intravascular hemolysis and an underlying marrow dysfunction, usually characteristic of aplastic anemia.
Eculizumab, an antibody to the fifth component of complement, can reduce or stop the intravascular destruction of red cells and decrease anemia in PNH patients, but has no effect on the marrow dysfunction.
PNH occurs in all age groups, especially young adults.
There are reports on the following related topics in NORD’s online Rare Disease Database for patients, families and the public at: