A Disease That Causes Ongoing Red Blood Cell Destruction (Hemolysis)
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired mutation that causes some
or all of your body’s red blood cells (RBCs) to be destroyed by a process called
hemolysis.1-3 PNH is a complex disease with signs and symptoms that are
nonspecific, unpredictable, and often similar to those of other diseases. In addition,
PNH presents uniquely in each person. If you have PNH, some or all of your RBCs
may be missing an important protective protein. Without this protein, RBCs are prone
to destruction by a part of your body’s defense system called complement.1-3
Even though you can’t feel it, hemolysis is constant, silent, and can be life threatening.4
Like other chronic diseases, such as diabetes or hypertension, PNH can result in
serious health problems if not treated. Common symptoms associated with PNH include
stomach pain, difficulty swallowing, anemia, shortness of breath, and tiredness
(fatigue). More serious complications can include blood clots, kidney failure, and
damage to vital organs.5 People with PNH may have different symptoms
that can unpredictably get worse (such as during times of stress) or better from
time to time. However, all people with PNH experience chronic hemolysis.
People with PNH may have other medical conditions that affect the function of their
bone marrow such as aplastic anemia (AA) or myelodysplastic syndromes (MDS). Unlike
PNH, which destroys RBCs, these diseases may reduce the production of blood cells
and further complicate PNH.6 If you have PNH in combination with AA or
MDS, talk to your doctor — it’s important to effectively treat all the conditions
you may have.
