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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease that occurs as a result of a genetic change (mutation) during the creation of red blood cells. This mutation leads to the production of abnormal red blood cells, also known as PNH red blood cells, that break more easily.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease that occurs as a result of a genetic change (mutation) during the creation of red blood cells. This mutation leads to the production of abnormal red blood cells, also known as PNH red blood cells, that break more easily.
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Normal red blood cells have protective proteins that act as a natural shield against the body's complement system that supports the immune system in fighting infections.
PNH red blood cells lack protective proteins due to a mutation, which makes them easier targets for destruction by the complement system through a process called hemolysis.
Hemolysis is the process of destroying red blood cells to help replace older blood cells with new ones. But in people living with PNH, hemolysis occurs much faster than new red blood cells can be made.
This is when red blood cells are destroyed inside the circulatory system.
This is when red blood cells are destroyed outside the circulatory system, such as in the liver and spleen.
An effective treatment response helps you gain full hemolytic control, which means both intravascular and extravascular hemolysis are well controlled. This can help reduce signs of a sub-optimal treatment response, including the main indicators, low hemoglobin (a protein in red blood cells), fatigue and dependence on blood transfusions.
The complement system consists of a group of proteins in the blood. They form a pathway that, when activated, helps support (complement) the immune system by fighting infections.