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Treatment for aplastic anemia is either primary or supportive.
Primary treatment for aplastic anemia
Allogeneic stem cell transplant: Allogeneic stem cell transplant is considered the best treatment for younger people with aplastic anemia. In this type of transplant, you receive stem cells (from the bone marrow or blood) from a donor. This type of transplant works best in children and young adults. As people get older, it's harder for them to tolerate this procedure. For people older than 30 or 40, many doctors prefer using immune therapy as the first treatment. In order for transplant to be an option, you need to have a donor who is matched to you. Someone who is closely related to you, like a brother or sister, is generally the best choice. In aplastic anemia, this stem cell transplant is successful up to 80% to 90% of the time if cells from a matched related donor are used.
Matching is determined by a type of test called HLA typing, which is done in the laboratory. If there is no match from a brother or sister, sometimes an unrelated donor will match. A transplant from a matched-unrelated donor is riskier than using a sibling match. Another option is to use stem cells from the umbilical cord of a newborn baby. A nationwide registry of potential stem cell donors and stored umbilical cord blood has been developed. Many doctors recommend using a non-related donor only if immune treatments described below don't work.
For a stem cell transplant, you will first receive chemotherapy (chemo). The chemo drugs used most often include cyclophosphamide (Cytoxan®) and fludarabine (Fludara®). A drug to suppress your immune system, like anti-thymocyte globulin (ATG) or alemtuzumab (Campath®), is also given. A newer drug, daclizumab (Zenapax®), may also be used. This immune treatment is important to keep your immune system from killing the new bone marrow (like it killed your original bone marrow). Immune treatment is also important to prevent the new bone marrow from attacking your body (this is called graft-versus-host disease and is discussed later in this section). You may also get a low dose of radiation therapy to your whole body. People with inherited forms of aplastic anemia are very sensitive to chemotherapy and radiation, and so often receive lower doses.
Soon after the radiation and/or chemotherapy, you will receive a transfusion with blood-forming stem cells from a donor. The stem cells can be obtained from the donor by removing bone marrow in the operating room while he or she is under general anesthesia. Sometimes a procedure called apheresis is used to remove stem cells from the bloodstream. Apheresis uses a machine that is hooked up to a large vein (through a catheter) and removes stem cells from the blood (returning the other cells). No matter which way they are collected, the stem cells are infused through your vein into the blood and then travel to the bone marrow, where they will grow
Stem cell transplantation is a major procedure with many risks and side effects. Some people may die during this procedure. The most serious side effects often occur during the first few weeks after the transplant. But with advances in this treatment, death from these early side effects is less common. The chance of severe side effects increases with the age of the patient.
In the first few weeks after the transplant there are usually side effects from the chemotherapy or radiation therapy. You may develop nausea, vomiting, diarrhea, and mouth sores from the treatment. Very low blood counts requiring red blood cell and platelet transfusions are common. Low white blood cell counts can lead to serious infections that require treatment with intravenous (IV) antibiotics. All of these problems generally go away in 3 to 4 weeks when the transplanted blood-forming stem cells start to produce normal blood cells.
If ATG is used, there is also a risk of serious allergic reactions during the infusion. These reactions can range from skin rashes to low blood pressure and problems breathing. Generally, these side effects can be controlled with medicines. This is discussed in more detail in the next section.
A very serious side effect of a stem cell transplant is graft-versus-host disease (GVHD). This occurs when the transplanted donor cells attack your own cells through an immune reaction. GVHD is more common with unrelated or mismatched donors. The risk of GVHD is also higher in older patients. GVHD can occur very early after the transplant. This is called acute GVHD. When GVHD develops later on or lasts a long time it is called chronic GVHD. Early signs of GVHD include skin rashes with severe itching and bowel disturbances such as diarrhea. There may be abnormal lab tests showing liver damage. GVHD is treated with medications (like prednisone, methotrexate, or tacrolimus) to try to suppress the immune system. In some cases, GVHD goes away and the medications can be stopped. In other cases, GVHD is only partly controlled, and medications are needed for a long time. GVHD can sometimes be very disabling and occasionally even cause death.
Immunosuppressive therapy: For people with acquired aplastic anemia who are not able to have a transplant (because of age or because they do not have a matched donor), doctors will often give immunosuppressive treatment. Most cases of acquired aplastic anemia are caused by the immune system attacking the bone marrow. This treatment helps stop the immune system from killing the bone marrow cells. This type of treatment is not usually helpful for cases of inherited aplastic anemia because they are not caused by the immune system.
The major drugs used are antithymocyte globulin (ATG) and cyclosporine. ATG contains antibodies against human T-lymphocytes. This medicine is given in the hospital through an intravenous (IV) line. ATG decreases (suppresses) your immune system function by lowering the number of T-cells in the body. The antibodies in ATG come from an animal (like a horse or a rabbit), so there is a risk of a serious allergic reaction when the ATG is given. Sometimes patients getting ATG also receive a corticosteroid medicine (like prednisone) to reduce the chance that a serious reaction will occur. Often the drug called cyclosporine is given as well. This drug suppresses the immune system in a different way than ATG.
Combining ATG and cyclosporine improves the blood counts in about 70% of patients with the most severe disease. The aplastic anemia may not actually be cured in most of these patients. Still, even when the blood counts do not become completely normal, they often improve enough for the patient to feel well and live a normal life. Often, after a period of remission, the aplastic anemia will come back. Usually, it will respond again to immune treatment, which can be safely repeated.
Some doctors now also give a drug called G-CSF along with ATG and cyclosporine. This drug is a growth factor for white blood cells; it tells the bone marrow to make more of these cells. It is also known as filgrastim or Neupogen®. A recent study showed that this drug can help lower the chance of a serious infection and lower the number of days that patients have to be in the hospital, but it doesn’t make it more likely that immunosuppressive therapy will be successful.
A newer drug, alemtuzumab, is sometimes used instead of ATG. It has a lower risk of serious allergic reactions, but doctors aren't sure that it works as well as ATG.
Another option for immunosuppressive therapy is to use the chemo drug cyclophosphamide in high doses. Cyclophosphamide is a chemo drug that can suppress the immune system and damage T-lymphocytes. Using it to treat aplastic anemia is controversial. Although this treatment can be effective, many experts believe that it is more dangerous than ATG. Most doctors in the United States prefer to delay using cyclophosphamide until ATG and cyclosporine are no longer working.
Immunosuppressive therapy can have serious side effects. Holding back the immune system impairs the body's ability to fight infection. People on immunosuppression can get life-threatening infections with bacteria, viruses, and fungi.
The drugs used in this therapy also have serious side effects. For example, ATG can cause serious allergic reactions with symptoms including skin rashes, low blood pressure, and problems breathing. Generally, these side effects can be controlled with medicines. Also, about 15% of patients develop leukemia or myelodysplasia several years after getting ATG. Myelodysplasia is a disorder of the bone marrow that can turn into leukemia.
Side effects of cyclosporine include high blood pressure as well as kidney and liver damage. To help prevent these problems, the doctor will check the level of cyclosporine in the blood regularly. Blood tests will also be done to check kidney and liver function.
Corticosteroids (like prednisone) can cause increases in blood sugar (like diabetes), high blood pressure, weight gain, changes in mood, and weak bones.
Supportive treatment for aplastic anemia
Transfusion : While your blood counts are low, you may need transfusions of red blood cells or platelets. Although blood transfusions are generally safe, their long-term use creates problems. The main problem is that the red cells contain iron. The transfused cells only live a few weeks and iron from these cells builds up in the body. Eventually this will result in a high level of body iron, which may be toxic. Although the build up of iron can be treated with drugs, these aren't always successful and some organs, mainly the liver and heart, can become damaged. Also, getting tranfusions before a stem cell transplant increases the chance that the transplant won't work. This is why doctors avoid giving transfusions when a transplant is planned.
Antibiotics: Because of your low white blood cell count, you may develop an infection and need antibiotics. Infection is the major cause of death from aplastic anemia. Treatment with antibiotics should be started as soon as infection is suspected. Transfusing white blood cells is not generally practical. They survive only a very short time, and it is not possible to get enough white blood cells from normal donors to raise a person's white blood cell count.
Growth factors : Drugs can be given to increase the white blood cell count. These are called filgrastim (also called G-CSF or Neupogen), pegfilgrastim (Neulasta®), and sargramostim (also called GM-CSF or Leukine®). These work only slightly for most patients with aplastic anemia.
Androgens: Some patients with early or mild aplastic anemia can be treated with androgens (instead of stem cell transplantation or ATG). Androgens are also often the first treatment given to patients with inherited forms of aplastic anemia (like Fanconi anemia and dyskeratosis congenita). Androgens are male sex hormones that also stimulate blood production. They are the reason that men have higher red blood cell counts than do women. Although they can be effective in improving blood counts, they do not cure aplastic anemia. Also, using androgens for a long time has been linked to liver tumors and liver cancer. Androgens are male hormones, so women taking this medicine can develop masculine characteristics such as facial hair, balding, deepening voice, etc.
Treatment to lower iron levels: Blood transfusions can cause excess iron to build up in the body. This extra iron can deposit in the liver and heart, causing the organs to function poorly. This build up of iron is only seen in people who receive many transfusions over a period of years. Drugs called chelating agents (substances that bind with metal so that the body can get rid of it) can be used in patients who may develop iron overload from transfusions. The most commonly used drug is desferoxamine. This drug helps treat and prevent iron overload. This is given intravenously or as an injection under the skin. It is inconvenient because the injection must be given slowly (over several hours) 5 to 7 times per week. In some patients, treatment continues for years. Deferasirox (Exjade®) is a newer drug that is taken by mouth once a day to treat iron overload. Although it has been used more for patients with certain congenital anemias (like thalassemia), it can also help patients with aplastic anemia and iron overload.
For some people with aplastic anemia, treatment may control the disease. Completing treatment can be both stressful and exciting. You may be relieved to finish treatment, yet it is hard not to worry about aplastic anemia coming back (when a disease returns, it is called recurrence or relapse). This is a very common concern.
It may take a while before your confidence in your own recovery begins to feel real and your fears are somewhat relieved. Even with no recurrences, people who have had aplastic anemia learn to live with uncertainty.
For some people with aplastic anemia, treatment improves blood counts, but the disease doesn’t go away completely. These people may have frequent follow-up visits with their doctor and may need transfusions and other treatments. Learning to live with a disease that does not go away can be difficult and very stressful. It has its own type of uncertainty.
Follow-up care: When treatment ends, your doctors will still want to watch you closely. It is very important to go to all of your follow-up appointments. During these visits, your doctors will ask questions about any problems you may have and do exams and lab tests. The doctors will continue to watch your blood counts for signs of the disease coming back, as well as for short-term and long-term side effects of treatment. Almost any treatment can have side effects. Some may last for a few weeks to months, but others can last the rest of your life. This is the time for you to talk to your care team about any changes or problems you notice and any questions or concerns you have.
Your blood doctor will see you for many years after your treatment. Ask what kind of follow-up schedule you can expect.
If aplastic anemia returns, it is often in the first year after treatment. Another concern is that some people with aplastic anemia go on to develop other blood problems, such as paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome, or acute leukemia. These conditions can be found early with the blood tests done as a part of your follow-up.
It is important to keep medical insurance. Tests and doctor visits cost a lot, and even though no one wants to think of their disease coming back, this could happen.
If treatment for aplastic anemia stops working
If aplastic anemia keeps coming back after one kind of treatment, it is possible that another treatment plan might still help. But when a person has tried many different treatments, the chances of another treatment helping becomes slight. If this happens, it’s important to weigh the possible limited benefits of a new treatment against the possible downsides. Everyone has their own way of looking at this.
This is likely to be the hardest part of your battle with this disease -- when you have been through every medical treatment the doctors offer you and nothing’s working anymore. Your doctor may offer you new options, but at some point you need to consider that treatment is not likely to improve your health or change your outcome or survival.
If you want to continue to get treatment as long as you can, you still need to think about the odds of treatment having any benefit. In many cases, your doctor can estimate how likely the aplastic anemia will respond to treatment you are considering. For instance, the doctors may say that a certain treatment may have about a 1% chance of working. Some people are still tempted to try this. But it is important to think about and understand your reasons for choosing this plan.
No matter what you decide to do, you need to feel as good as you can. Make sure you are asking for and getting treatment for any symptoms you might have, such as nausea or pain. This type of treatment is called palliative treatment.
Palliative care helps relieve symptoms, but is not expected to cure the disease. It can be given along with treatment, or can even be a type of treatment. The difference is its purpose - the main purpose of palliative care is to improve the quality of your life, or help you feel as good as you can for as long as you can. Sometimes this means using drugs to help with symptoms like pain or nausea. Sometimes, though, the treatments used to control your symptoms are the same as those used to treat aplastic anemia. For instance, transfusions are a type of palliative treatment for aplastic anemia. They can help relieve the fatigue and shortness of breath that goes along with anemia, but they don’t treat the underlying cause. This is not the same as treatment to try to cure your disease.
At some point, you may benefit from hospice care. This is special care that treats the person rather than the disease; it focuses on quality rather than length of life. Most of the time, it is given at home. Your disease may be causing problems that need to be managed, and hospice focuses on your comfort. You should know that while getting hospice care often means the end of treatments such as immunosuppressants and sometimes even transfusions, it doesn’t mean you can’t have treatment for the problems caused by your disease or other health conditions. In hospice the focus of your care is on living life as fully as possible and feeling as well as you can at this difficult time. You can learn more in our document Hospice Care.
Staying hopeful is important, too. Your hope for a cure may not be as bright, but there is still hope for good times with family and friends -- times that are filled with happiness and meaning. Pausing at this time in your cancer treatment gives you a chance to refocus on the most important things in your life. Now is the time to do some things you’ve always wanted to do and to stop doing the things you no longer want to do. Though the cancer may be beyond your control, there are still choices you can make.
New understanding about the causes of some forms of inherited aplastic anemia, especially Fanconi Anemia, Diamond-Blackfan Syndrome, and Schwachman-Diamond Syndrome, may provide entirely new approaches to treatment of aplastic anemia in the future.
Other major advances in treating aplastic anemia may come from further research on the immune system. As doctors learn more about how the immune system works, they can develop new methods of suppressing it. Researchers are working to improve the drugs used for immunosuppression and stem cell transplantation. The goal is to find treatments that have fewer side effects than the present treatments with better results.
One drug under study is called daclizumab (Zenapax®). This is a monoclonal antibody (a laboratory-made antibody that attaches to specific substances) that blocks the action of interleukin-2 (IL-2). IL-2 is a normal part of the immune system that stimulates the immune response. Blocking IL-2 suppresses the immune system in a way different from anti-thymocyte globulin (ATG). Allergic reactions to this drug are rare. It is currently being used as a part of stem cell transplants, and is under study as a treatment for aplastic anemia.
Another drug being looked at is called mycophenolate mofetil. It is already used as an immunosuppressant in people who have received organ transplants and is being studied as a treatment for acquired aplastic anemia.
A new drug called eltrombopag (Promacta®) has become available to treat some people with low platelet counts caused by the immune system. In one study of patients with acquired aplastic anemia that was no longer responding to immunosuppression, it helped improve blood counts in some patients. This drug didn’t just affect platelet counts. In some patients, it improved white counts and/or red blood cell counts. More studies of this drug in aplastic anemia are going on now.
Alefacept is a new drug that can turn off T cells. It has been helpful in treating psoriasis and graft-versus-host disease and is now being studied in the treatment of severe acquired aplastic anemia.
Researchers are also studying the possibility of using gene therapy to treat some inherited forms of aplastic anemia. Another approach is to give a drug called amifostine (Ethyol®) to help protect the bone marrow in children with Fanconi anemia.
Doctors are also trying to make stem cell transplants safer and more available. One approach is to use stem cells that come from the umbilical cord blood of newborns. This is a very rich source of stem cells. Efforts are being made nationwide to develop storage facilities for cord blood. The advantage of cord blood stem cells is that they may lead to less graft-versus-host disease. The disadvantage is that there may be too few cells to successfully “take.” In one case, doctors have overcome this by transplanting cord blood stem cells from 2 separate donors. This has proven successful and may have an even lower risk of graft versus host disease. This method is currently under study.