PNH is considered chronic. That means it lasts for a long time. The only known cure is a bone marrow transplant bone marrow transplant: A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due… . Other treatments are designed to ease symptoms and prevent problems. A small number of people say their PNH went away on its own over time.
Treating Hemolysis and Anemia
For most people with PNH, the most common problem is anemia caused by hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. . Here are some treatments for hemolysis and anemia.
Blood Transfusions
In a blood transfusion blood transfusion: A procedure in which whole blood or one of its components is given to a person through an intravenous (IV) line into the bloodstream. A red blood cell transfusion or a platelet transfuson can help some patients with low blood counts. , whole blood or parts of blood from a donor are put right into your bloodstream. This can improve your anemia. Your doctor will look at your symptoms to decide if you need a transfusion.
The 2 types of transfusion available for PNH patients are:
- Red blood cell transfusion Red blood cell transfusion: A procedure in which packed red blood cells are given to a person through an intravenous (IV) line into the bloodstream. Transfused red blood cells increase the blood count and help improve symptoms of anemia. Before transfused blood is given, donated blood is typed and crossmatched to the…
- Platelet transfusion Platelet transfusion: A procedure in which platelets are given to a person through an intravenous (IV) line into the bloodstream. Platelets are more likely than red blood cells to cause an immune response, such as chills and fever. The use of platelets from one donor (apheresis) reduces the chance of reaction to…
Learn more about blood transfusions.
Iron Therapy
Hemolysis can lead to a shortage of iron in your body. This can make it hard for your bone marrow to make red blood cells. So, unless you are receiving regular red blood cell red blood cell: The most numerous type of blood cell in healthy people. Red blood cells contain hemoglobin, a protein that picks up oxygen in the lungs and brings it to cells in all parts of the body. Also called erythrocyte, RBC. transfusions, you probably need to take iron pills. Ask your doctor how much iron you need.
Side Effects of Iron Therapy
At first, taking iron pills can cause hemolysis, which leads to dark urine. This happens because your bone marrow is making more red blood cells. Some of them break apart because you have PNH.
Iron pills can also cause an upset stomach. If you have a severe stomach problem, you can receive your iron by injection. There is a very small risk of allergic reaction when iron is given this way.
Folate and Folic Acid
Folate Folate: (FOE-late) A B-vitamin that is found in fresh or lightly cooked green vegetables. It helps the bone marrow make normal blood cells. Most people get enough folate in their diet. Doctors may have people with paroxysmal nocturnal hemaglobinuria (PNH) take a man-made form of folate called folic acid. is a B-vitamin that is found in fresh or lightly cooked green vegetables. It helps your bone marrow make normal blood cells. When your bone marrow has to make more cells, it needs a larger supply of folic acid.
Most people get enough folate in their diet. But if you have PNH, it's a good idea to take 1 mg each day of a man-made form of folate called folic acid.
Growth Factors
Growth factors Growth factors: Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts. Red… are chemicals in your body. They cause your bone marrow to make blood cells. Man-made forms of some growth factors are available. They can reduce the need for red blood cell transfusion. Learn more.
Androgens
Androgens Androgens: Androgens are natural male hormones that can cause your bone marrow to make more red blood cells. This can improve anemia. Androgens are sometimes used to treat aplastic anemia and PNH. Androgens that may be used include: Danazol (Danacrine®) Fluoxymestrone (Halotestin®) Oxymetholone … are natural male hormones that can cause your bone marrow to make more red blood cells. This can improve anemia. Androgens are more likely to be used if you have other bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… problems besides PNH. Learn more.
Treating Bone Marrow Failure
Bone marrow failure is a condition in which your bone marrow does not make enough healthy cells. In people who have PNH, bone marrow failure is most likely to be caused by aplastic anemia. So the treatment for bone marrow failure is the same as that for aplastic anemia. Read more about aplastic anemia.
Bone Marrow Transplantation
If other treatments have not worked to stop hemolysis, clotting, or bone marrow failure, then a bone marrow transplant may be the next step.
In this procedure, healthy bone marrow stem cells stem cells: Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem… and other bone marrow cells are taken from a donor. These cells are given by IV. The donor's cells make their way through your blood and into your bones where they start making healthy blood cells.
Bone marrow transplantation is the only way to cure PNH. But it carries many risks, including death.
Read more about bone marrow and stem cell transplantation.
VACCINATIONS
Patients with PNH should receive vaccinations against certain types of bacteria to prevent infection. Ask your doctor which ones are right for you.
Always ask your hematologist hematologist: (hee-muh-TOL-uh-jist) A doctor who specializes in treating blood diseases and disorders of blood producing organs. about all treatment options available to you.
Order an AA&MDSIF information packet containing detailed information about PNH and its treatment. This packet includes questions that need to be answered by your doctor before you agree to any treatment option.
For more in-depth information on PNH and its treatment, you can also visit our Online Learning Center, and view webinars by some of the leading medical experts on PNH.