Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder where the immune system mistakenly attacks and destroys red blood cells — a process called hemolysis. It affects roughly 13 people per million in the United States.
The root cause is a mutation in the PIGA gene within bone marrow stem cells. This mutation Show Full Answer
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder where the immune system mistakenly attacks and destroys red blood cells — a process called hemolysis. It affects roughly 13 people per million in the United States.
The root cause is a mutation in the PIGA gene within bone marrow stem cells. This mutation means red blood cells are produced without a protective shield, leaving them vulnerable to attack by a part of the immune system called the complement system. PNH doesn't just affect red blood cells — it impacts all three types of blood cells:
- Red blood cells — which carry oxygen throughout the body
- White blood cells — which help fight infections
- Platelets — which help blood clot properly
Because red blood cells are destroyed faster than the body can replace them, this leads to low red blood cell levels, known as hemolytic anemia. There are three main types of PNH:
- Classic (hemolytic) PNH — the most common form, with clear signs of red blood cell breakdown
- Subclinical PNH — fewer or no obvious symptoms
- PNH with a primary bone marrow disorder — occurs alongside another bone marrow condition
Without treatment, PNH can lead to serious complications, but with the right care and monitoring, many people manage to maintain a good quality of life.
April 26
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