PNH - Treatment (OLD) | Aplastic Anemia and MDS International Foundation

PNH - Treatment (OLD)

PNH is considered chronic. That means it lasts for a long time. The only known cure is a bone marrow transplant. 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.

 

    Blood Transfusions

    A blood transfusion is a safe and common procedure. When you receive a transfusion, the cell parts of blood from a donor are put into your bloodstream. This increases your blood counts and reduces symptoms. Most people who have PNH will receive at least one blood transfusion.

    The 2 types of transfusion available for PNH patients are:

    A transfusion for red blood cells takes 2 to 4 hours. A platelet transfusion takes 30 to 60 minutes.

    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 transfusions, you probably need to take iron pills. Ask your doctor how much iron you need. 

    At first, taking iron pills can cause hemolysis, which can lead 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.

    Folic Acid

    Folic acid, also called folate or vitamin B-12, 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 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.

    Red cell growth factor is called erythropoietin or EPO for short. It is a protein made by your kidneys in response to low oxygen levels in the body caused by low red cell counts and anemia. EPO causes your bone marrow to make more red blood cells.

    If you don't have enough natural EPO or if you have anemia, your doctor may prescribe a pharmaceutical form of EPO, such as:

    • Epoetin Alpha (Procrit®, Epogen®):  Epoetin alpha is typically given as an injection (shot) in the arm, abdomen or thigh.
    • Darbepoetin alpha (Aranesp®):  Darbepoetin alpha may be given as an injection (shot) in the arm, abdomen or thigh or by infusion into a vein (intravenous IV).

    Your doctor may ask you to take a white blood cell growth factor along with EPO. This combination may improve red blood cell counts in certain patients.

    These medicines have some risks, including the chance of getting a blood clot. You and your doctor will decide whether the benefits of these drugs outweigh the risks.

    White cell growth factors may be prescribed if you don't have enough healthy white cells in your blood, your doctor may ask you to take a white cell growth factor to increase your count and improve your immune system. The most common ones are:

    • G-CSF (granulocyte colony-stimulating factors) is sold under the names Filgrastim® and Neupogen®.
    • GM-CSF (granulocyte macrophage colony-stimulating factors) is sold under the names Leukine® and Sargramostim®.

    A platelet growth factor may be prescribed if you don’t have enough platelets in your blood. Only one platelet growth factor is currently available. It was recently approved to treat PNH. This drug is Eltrombopag (Promacta ®), a synthetic drug that mimics thrombopoetin, the body’s natural platelet growth factor.

    Eltrombopag has been shown to both increase platelet counts and, in some cases, to improve red blood cell counts in aplastic anemia patients. This drug is beginning to be given as first line treatment for PNH, along with standard immunosuppressive therapy with ATG and cyclosporine.

    Prednisone

    Prednisone is a steroid that can decrease hemolysis caused by PNH. It may make the complement system less active. It may also increase counts of white blood cells and platelets in some people.

    Prednisone does not stop all hemolysis, however, and it does have a lot side effects, especially if taken for a long time. Currently, doctors disagree about whether it should be used by PNH patients.

    Most people who take prednisone for a long time use it only every other day. This schedule eases the most severe side effects of prednisone.

    If your hemolysis is worse than normal, your doctor may recommend that you take 60 mg for 3 to 4 days, then gradually cut back to the regular schedule.

    Androgens

    Androgens 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 problems besides PNH. They include:

    • Danazol (Danacrine®)
    • Fluoxymestrone (Halotestin®)Oxymetholone
    • Stanazolol (Winstrol®)
    • Testosterone

    Bone Marrow Transplantation

    Some people with PNH also have bone marrow failure. The most likely cause is aplastic anemia. If treatments like ATG and cyclosporine do not stop hemolysis, clotting, or bone marrow failure, then a bone marrow transplant may be the next step.

    Bone marrow transplants are usually good options for younger patients or those with a matched donor. If your doctor thinks you may be a candidate for a bone marrow transplant, you should start looking for a matched donor right away. Each of your siblings has a 1 in 4 chance of being a match. If you don’t have a sibling match, you will need to find a matched unrelated donor (MUD) through a bone marrow donor registry like the one managed by the National Marrow Donor Program.

    In this procedure, healthy bone marrow stem cells and other bone marrow cells are taken from a donor. These cells are given to the patient by IV. The donor's cells make their way through the blood and into 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.

    Vaccinations

    Patients with PNH should receive vaccinations against certain types of bacteria to prevent infection. Ask your doctor which ones are right for you.

    Clinical Trials

    Clinical research studies, also called clinical trials, are at the heart of all medical advances. They help identify new ways to prevent, detect and treat disease. If you have PNH, you may want to consider taking part in a research study. They can be a good treatment option for some PNH patients.

    Blood Clot Treatment

    Blood clots are very dangerous. If you have a blood clot, you need to get help right away. Here are some ways blood clots are treated.

    Blood thinners are the most common way to treat blood clots. They decrease the ability of blood to clot. Blood thinners are also called anticoagulation drugs, or anticoagulants. Here are some of the ones prescribed for PNH patients:

    • Enoxaparin (Lovenox®) is taken as a shot 1 or 2 times per day.
    • Heparin (Calciparine® or Liquaemin®) is taken as a shot or by IV. Your doctor will keep an eye on the dose to prevent bleeding problems.
    • Warfarin (Coumadin®) comes in pill form. The foods you eat can affect the level of warfarin in your blood. Your doctor will keep an eye on the dose to prevent bleeding problems and to make sure the Coumadin in your blood is at a high enough level.

    Some doctors think PNH patients who are more at risk of blood clots should take blood thinners to prevent them; others disagree.

    tPAs (tissue plasminogen activators) may be used in an emergency to break up an existing blood clot, especially if the clot is large. These drugs usually work only in the first 48 hours after a clot has formed. They can cause bleeding.

    Some drugs, such as aspirin and ibuprofen can also help stop blood clots. However, doctors do not know how well they prevent or treat clots in people with PNH. Take these drugs only if your doctor recommends them.

    Learn more about blood clot treatments.

    Eculizumab (Soliris ®)

    Studies have shown that eculizumab (Soliris®) can also reduce the risk of blood clots.

    Eculizumab is the only drug approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Evaluation Agency (EMEA) to treat PNH. It works by making your complement system less active.

    Eculizumab has many benefits. It has been found to:

    • Improve anemia
    • Reduce or eliminate the need for transfusions
    • Reduce the destruction of red blood cells (hemolysis) over both the short and
    • long term
    • Reduce the risk of blood clots

    Soliris does not help increase white blood cell count or platelet cell count.

    Learn more about eculizumab.