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• W2147251029 abstract "Paroxysmal nocturnal hemoglobinuria (PNH) is a complex hematological disorder probably first described three centuries ago, and regarded as a mystery until the 1980s, when most of its pathophysiology was elucidated, followed by the 1990s, when the underlying molecular defect was finally unraveled. The original denomination, PNH, stresses only one component of the disease (i.e., a hyperhemolytic state). A more complete contemporary definition could read as follows: an acquired blood disorder characterized by the expansion of one or a few hematopoietic cell clones that are unable to produce the glycosyl-phosphatidyl inositol (GPI) anchor, against the background of a reduced bone marrow activity. Some landmarks in the history of understanding this disorder are listed in Table 25-1. Because of its complex pathophysiology, this disorder has been variously classified among hemolytic anemias, myelodysplasia, myeloproliferative disorders, or bone marrow failure syndromes; indeed, PNH has some features of each of these. Here we will briefly summarize clinical, diagnostic, and therapeutic features, and we will then review in some detail recent progress on the genetic and pathophysiological aspects of PNH." @default.
• W2147251029 created "2016-06-24" @default.
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• W2147251029 date "2007-11-17" @default.
• W2147251029 modified "2023-10-14" @default.
• W2147251029 title "Paroxysmal Nocturnal Hemoglobinuria" @default.
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• W2147251029 doi "https://doi.org/10.1007/978-1-59259-963-9_86" @default.
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