Aplastic Anemia

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This is an individual decision and should be discussed in detail with your doctor and the medical team that would be involved in either treatment approach. They can help you understand your specific risks and potential benefits of these options.

The main goal of 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… treatment is to increase the number of healthy cells in your blood so you will feel better, no longer need transfusions and have improved quality of life.

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… using 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. works by preventing your immune system from attacking your bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. , letting your 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… grow back and raising your blood count. This is a standard treatment for older patients or those without a stem cell donor.

Stem cell transplantation involves putting blood-forming stem cells from a healthy donor into your bloodstream. These cells travel to your bone marrow and start making healthy cells. This is a standard treatment for younger patients with severe aplastic anemia (SAA) and 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… diseases if a donor is not available.

Cyclosporine Cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. is an immunosuppressive drug that works by preventing T-lymphocytes from becoming active. Once the T-lymphocytes are turned off, they stop attacking 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… in the bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. . That means stem cells may be able to grow back. It is used along with antithymocyte globulin antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. (ATG) as a standard treatment for 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… , and may also be used to treat MDS and PNH.

Some aplastic anemia patients may require prolonged ongoing treatment with cyclosporine to prevent or minimize the risk of disease relapse aplastic anemia coming back. Generally speaking, patients can remain on cyclosporine indefinitely, as long as there is evidence that the drug is providing some benefit and there are no unwanted or adverse side effects.

For patients who respond well to cyclosporine, alone or in combination with antithymocyte globulin (ATG), hematologists may reduce the cyclosporine dosage. Tapering, or reducing, the dosage is done slowly over a long period of time. If low blood counts and disease symptoms return, your doctor may increase or restart cyclosporine. For those who experience bad side effects such as kidney toxicity, the drug must be discontinued.

Cyclosporine Cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. is a drug that lowers your body's immune response. Cyclosporine is used along with antithymocyte globulin antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. (ATG), another immunosuppressant, for treating 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… and some 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… diseases. If you are taking cyclosporine as part of your treatment regimen, your doctor should monitor you for side effects. Cyclosporine may cause high blood pressure, kidney damage, loss of magnesium or potassium, liver inflammation, increased hair growth and gum swelling.

These problems can usually be fixed. Your doctor might prescribe medicine to lower your blood pressure and to replace magnesium or potassium.

It is important to weigh both the risks and the benefits of a bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. /stem cell transplant. A transplant doctor can answer your questions and help you decide if a transplant is an option for you.

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… has serious risks. Some patients suffer from life-threatening problems as a result of their transplant. These problems can include serious infections and graft-versus-host disease (GVHD) graft-versus-host disease (GVHD): Also called GVHD, it is a common complication of bone marrow/stem cell transplantation. It is caused when the donor's immune cells, now in the patient, begin to see the the patient's body as foreign and mount an immune response. GVHD most commonly effects the recipient's skin, intestines, or liver… , in which the transplanted cells attack the patient's body.

For some patients, a transplant may be the best option for a longer life, as the procedure remains the only known cure for 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… diseases like 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… , MDS and PNH. You, your doctor and your family need to consider many factors when making a decision on a transplant, including your disease stage, your age, your overall health, whether a matching donor is available and other treatment options.

Doctors sometimes refer to particular drugs as an “active treatment” or “active drug therapy.” Active treatments treat the underlying cause of the 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… . These drugs are typically approved by the U.S. Food and Drug Administration (FDA) to treat a specific disease. It is important to note that active treatments do not cure the disease. The only potential cure for a bone marrow failure disease is a bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. /stem cell transplant. Unfortunately, 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… is not an option for many people. In addition, it carries many risks and potential long-term side effects.

For 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… , antithymocyte globulin antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. (ATG), typically given with cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. , is an active drug therapy often used.

Doctors will sometimes refer to certain treatments as supportive therapy or  supportive care supportive care: Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive… . They help you manage the symptoms of your disease, although they do not treat the underlying cause of the disease.

In general, supportive care includes the following:

• Blood transfusions Blood transfusions: A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some… to raise blood cell counts
• Antibiotics Antibiotics: The most common white blood cells are called neutrophils. They fight infection. Patients who don't have enough healthy neutrophils are said to have neutropenia. Because of their low white count, they may get infections easily and have trouble getting rid of infections. If you have neutropenia,… to treat infection
• Iron chelation therapy Iron chelation therapy: (kee-LAY-shun) A drug therapy to remove extra iron from the body. Patients with high blood iron (ferritin) levels may receive iron chelation therapy. The U.S. Food and Drug Administratin (FDA) has approved two iron chelators to treat iron overload in the U.S. - deferasirox, an oral iron chelator,… to treat iron overload iron overload: A condition that occurs when too much iron accumulates in the body. Bone marrow failure disease patients who need regular red blood cell transfusions are at risk for iron overload. Organ damage can occur if iron overload is not treated. , a side effect of having many 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
• 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… that are man-made versions of natural hormones that tell your body to make more blood cells.

These diseases can not be passed down through the genes from parent to child. Most cases of 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… , MDS and PNH are considered “acquired” and the cause is not typically known. In very rare cases, inherited 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… syndromes can increase the chances of getting aplastic anemia or MDS.

How long do I have to live?

This is most people’s first question, along with “what can I expect to happen.” The doctor’s answer is called a prognosis (an educated guess about the likely course of your disease and how long you might live). Because each person is unique, and each person’s disease is different, a prognosis can be difficult to make. Also, how a disease progresses over time is unpredictable, and less is known about rare diseases than common diseases.

Whether you are an 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… , MDS or PNH patient, you need to talk to your doctor about your prognosis. This may be hard to hear, but getting a prognosis will give you key information about the treatment decisions you need to make so you can plan for the future.

Prognosis guidelines based on current data

Aplastic Anemia

With standard treatments, about 8 out of 10 aplastic anemia patients get better. Standard treatments include 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 antithymocyte globulin antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. (ATG) with cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. and promacta, or 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… . The chance for recovery depends on many factors, including how severe your case is and how you respond to treatment.

MDS

There are many different subtypes of MDS, which are identified by testing the blood and bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. . Your MDS subtype is a key factor in a doctor’s decision about your treatment and your prognosis (an educated guess about the likely course of your disease and how long you might live).

To figure out a prognosis for a given patient, doctors also use a prognostic scoring system. The most common one used today is the International Prognostic Scoring System International Prognostic Scoring System: A system that turns patient data into a score. The score tells how quickly a myelodysplastic syndrome (MDS) case is progressing and helps predict what may happen with the patient's MDS in the future. Also called IPSS. -R, or IPSS-R for short. This system looks at three things:

• Number of low blood counts you have
• Percentage of young white blood cells (blasts) in bone marrow cells
• Number of cytogenetic changes (abnormal gene changes) in bone marrow cells

This prognostic scoring system tells your doctor how severe your disease is and how likely it is that your MDS might become acute myeloid leukemia acute myeloid leukemia: (uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become… (AML). It also gives your doctor a general idea about how long you might live.

With current treatments, patients with lower-risk types of some MDS can live for 5 years or even longer. Patients with higher-risk MDS that becomes acute myeloid leukemia (AML) are likely to have a shorter life span. About 30 out of 100 MDS patients will develop AML. Most patients with AML need treatment soon after diagnosis because the disease often progresses fast. The initial goal is to put the patient into remission. The long-term goal is to cure the disease, although this is not always possible.

PNH

Many people with PNH live for decades. People with PNH who develop blood clots in key parts of the body or develop MDS (myelodysplastic syndromes) or AML (acute myeloid leukemia) may have a shorter life span.

Treatments available for PNH are helping people with PNH to live longer. Older research you may have come across state that PNH patients live an average of 15 to 20 years after diagnosis. More recent research shows that the PNH lifespan has climbed over the past 20 years. It’s possible that PNH patients – who rarely develop MDS or AML – will soon have a lifespan that is normal compared with people their own age.

It is always best to talk with your treating doctor about your prognosis. Remember, each patient is different.