Blood Transfusions
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. 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:
- Red blood cell transfusion
- Platelet transfusion
A transfusion for red blood cells takes 2 to 4 hours. A 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… 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 bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the 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 Folic acid: See folate. , also called 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. 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 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.
Red cell growth factor is called erythropoietin erythropoietin: (i-rith-row-POY-uh-tun) A protein made by the kidneys. Erythropoietin, also called EPO, is created in response to low oxygen levels in the body (anemia). EPO causes the bone marrow to make more red blood cells. A shortage of EPO can also cause anemia. 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 growth factor: A substance made by the body that stimulates the bone marrow to produce blood cells. Some growth factors are man-made in the laboratory and used for treating low blood counts. These include red blood cell growth factors called erythropoietin (EPO) and darbepoetin, and white blood cell growth… 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 blood clot: A mass of blood that forms when platelets stick together. Harmful blood clots are more likely to happen in PNH. The term thrombus describes a blood clot that develops and attaches to a blood vessel. The term embolus describes a blood clot or other foreign matter that gets into the bloodstream and… . 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 Filgrastim: It is known as a hematopoietic growth factor or colony stimulating factorand is given by injection under the skin (subcutaneous) or in the vein (intravenous). Filgrastim is approved by the U.S. Food and Drug Administration (FDA) for use in cancer patients who are at risk of developing infection due… ® 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 Eltrombopag: What are the possible side effects of eltrombopag (Promacta)? Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat. Stop using eltrombopag and call your doctor at once if you have: … (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 aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… patients. This drug is beginning to be given as first line treatment for PNH, along with standard immunosuppressive therapy immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… with ATG and cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. .
Prednisone
Prednisone Prednisone: Prednisone a man-made version of a hormone produced naturally by the adrenal gland that can help reduce inflammation and stop allergic responses. It is prescribed when the body is not producing enough of this chemical on its own. How does it work? For PNH, it may be used to decrease… 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 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. 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 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… 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 Clinical research: A type of research that involves individual persons or a group of people. There are three types of clinical research. Patient-oriented research includes clinical trials which test how a drug, medical device, or treatment approach works in people. Epidemiology or behavioral studies look at the… studies, also called clinical trials clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… , 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 warfarin: It is used to prevent blood clots from forming. Warfarin is a tablet that is taken by mouth. in your blood. Your doctor will keep an eye on the dose to prevent bleeding problems and to make sure the Coumadin Coumadin: Coumadin (warfarin) is an anticoagulant (blood thinner). Reduces the formation of blood clots, which is important in the prevention of heart attacks, strokes and blockage of major veins and arteries. 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 eculizumab: Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes. You will probably get an IV once a week for the first 4 weeks. Starting in the 5th week, you will get a slightly higher dose of Soliris every 2 weeks. … (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.