Paroxysmal nocturnal hemoglobinuria PNH

Soliris is for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). PNH is a chronic disease where a patient s oxygen-carrying red blood cells are missing the normally present complement inhibitors. The cells are therefore abnormally fragile and inadvertently destroyed by normal complement activation. 

Indications Paroxysmal nocturnal hemoglobinuria (PNH) Category Monoclonal antibody Half-life 272 Hours... 

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hemolytic anemia resulting from a somatic mutation in a multipotent hemopoietic stem cell (reviewed in refs. [115,116]). The abnormal stem cells produce subpopulations of blood cell types that lack all GPI-anchored proteins. The absence of decay-accelerating factor (DAF) and... 

An unoommon sign, which cannot be detected from the smear, but can be elicited by a sucrose water test" of peripheral blood, is transient paroxysmal nocturnal hemoglobinuria (PNH), which may first occur insidiously during a period of established aplastic anemia, and may be followed within one to a few years by the appearanoe of rapidly fatal aoute myelogenous leukemia. Clinioal detection of PNH, whioh ooours in only one or two peroent of those destined to have aoute myelogenous leukemia, may be difficult if the sucrose water test is positive, the somewhat more definitive Ham test, also known as the acid-serum hemolysis test, may provide confirmation. 

Macromolecules that are GPI anchored rely on the anchor for cell surface expression. Thus, defects in GPI anchoring can have severe consequences if they cause non-expression of functionally critical macromolecules. A well documented example is paroxysmal nocturnal hemoglobinuria (PNH), a human disease caused by a GPI biosynthesis defect in affected blood cells leading to the non-expression of complement regulatory proteins [4]. 

Paroxysmal Nocturnal Hemoglobinuria (PNH) Disease and Defects in GPI-Anchoring of Proteins... 

AIP, acute intermittent porphyria PNH, paroxysmal nocturnal hemoglobinuria. 

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