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Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired disorder that leads to the premature death and impaired production of blood cells
Prevalence
1-9 / 100 000
Age of Onset
ICD-10
D59.5
Inheritance
This condition does not appear to have a clear pattern of inheritance.
5 Facts you should know
FACT
Median age at diagnosis is 36 years
FACT
In PNH, a deficiency in complement regulatory proteins CD55 and CD59 leaves red blood cells vulnerable to lysis
FACT
Hemolysis can be intravascular or extravascular. Intravascular hemolysis (IVH) occurs inside blood vessels, while extravascular hemolysis (EVH) occurs in the liver and spleen
FACT
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
FACT
Infusion therapy can disrupt patients’ lives and can make them feel trapped, frustrated, and anxious
Interest over time
Google searches
Common signs & symptoms
Fatigue
Hemolytic anemia
Bone marrow hypocellularity
Hypercoagulability
Thromboembolism
Current treatments
The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.
Eculizumab(Brand name: Soliris) Manufactured by Alexion Pharmaceuticals, Inc.
FDA-approved indication: March 2007, eculizumab (Soliris) was approved for the treatment of paroxysmal nocturnal hemoglobinuria to reduce hemolysis.
National Library of Medicine Drug Information Portal
Medline Plus Health Information
Ravulizumab-cwvz(Brand name: Ultomiris) Manufactured by Alexion Pharmaceuticals, Inc.
FDA-approved indication: December 2018, ravulizumab-cwvz (Ultomiris) was approved for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
National Library of Medicine Drug Information Portal