Rare Endocrinology News

5 Facts you should know about

Paroxysmal Nocturnal Hemoglobinuria

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1

Median age at diagnosis is 36 years

2

In PNH, a deficiency in complement regulatory proteins CD55 and CD59 leaves red blood cells vulnerable to lysis

3

Hemolysis can be intravascular or extravascular. Intravascular hemolysis (IVH) occurs inside blood vessels, while extravascular hemolysis (EVH) occurs in the liver and spleen

4

A meaningful percentage of PNH patients experience low-level hemolysis and anemia despite terminal complement inhibition, with some receiving at least 1 blood transfusion to stabilize Hb levels

5

Infusion therapy can disrupt patients’ lives and can make them feel trapped, frustrated, and anxious

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