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Understanding Bone Marrow Transplantation as a Treatment Option

When you are diagnosed with a serious illness, it is helpful to understand the disease, treatment options, and factors affecting a treatment choice. By becoming informed and then talking with your doctor, you can take part in developing a treatment plan with your doctor.

On this page:

• Diseases Treatable with a Bone Marrow Transplant or Cord Blood Transplant
• Cells Used for a Transplant
• Transplant as a Treatment
• When Your Doctor May Consider a Transplant
• Talking with Your Doctor

Diseases Treatable with a Bone Marrow Transplant or Cord Blood Transplant

A bone marrow transplant (also called a BMT) or umbilical cord blood transplant replaces diseased blood-forming cells with healthy cells. Diseases that may be treated with a bone marrow or cord blood transplant include:

• Leukemias and lymphomas
• Multiple myeloma and other plasma cell disorders
• Severe aplastic anemia and other marrow failure states
• Sickle cell disease and thalassemia
• Inherited immune system disorders, such as severe combined immunodeficiency (SCID) and Wiskott-Aldrich syndrome
• Inherited metabolic disorders, such as Hurler's syndrome and leukodystrophies
• Myelodysplastic syndromes

To learn about the symptoms, diagnosis, and treatment options for these and other diseases, see Learning More about Your Disease.

Cells Used for a Transplant

The healthy cells for a transplant can come from three sources:

• Bone marrow
• Peripheral (circulating) blood that has an increased number of healthy blood-forming cells (also called peripheral blood stem cells or PBSC)
• Umbilical cord blood that is collected after a baby is born

If you need a transplant, your doctor will choose the source of the cells. Your doctor will also decide whether to use cells collected from you or another person.

• An autologous transplant uses cells collected from your body.
• An allogeneic transplant uses cells donated from a family member or an unrelated marrow donor or umbilical cord blood unit.

Your doctor decides on the cells used for transplant based on your disease, other treatments you have had, and your overall health.

Transplant as a Treatment

When a bone marrow or cord blood transplant is being considered as a treatment option, it is helpful to understand the transplant process.

Your Doctor Searches for a Marrow Donor or Cord Blood Unit

If you need an allogeneic transplant, your doctor will look for a marrow donor or cord blood unit that matches your HLA tissue type. HLA stands for human leukocyte antigen, a marker your immune system uses to recognize which cells belong in your body and which do not.

If a donor is not found in your family, your doctor can search for an unrelated donor or cord blood unit through the registry of the C.W. Bill Young Cell Transplantation Program, also called the Be The Match Registry^®. The registry is a listing of potential marrow donors and donated cord blood units.

The registry is operated under Federal contracts by the National Marrow Donor Program^® (NMDP). The NMDP has agreements with its global partners that provide access to more than 18.5 million potential marrow donors and more than 590,000 cord blood units.

Your Body is Prepared for a Transplant

Before your body can receive the healthy cells, the diseased cells must be destroyed. This is done using chemotherapy and sometimes radiation. The destruction of diseased cells is called a preparative regimen or a conditioning regimen.

You Receive the Cells for the Transplant

On the day of transplant, the cells from the marrow donor or cord blood unit are infused intravenously (go into your body through a large vein). These healthy cells move into the spaces inside your bones where they create new marrow. They grow and make healthy new red blood cells, white blood cells, and platelets.

For more information about chemotherapy and radiation, see The Transplant Process.

For more about the search process, HLA matching, and steps of a transplant, such as evaluating your health before transplant, the preparative regimen, receiving your new cells, and recovery, see The Donor Selection & Transplant Process.

When Your Doctor May Consider a Transplant

Your doctor chooses your treatment based on several factors, including the status of your disease and your health. A disease can change over time. For example, when a disease is first diagnosed, it may be in a chronic (slow-moving), stable phase. Within weeks or months the disease can change to a crisis status. Or, after receiving one treatment, the disease can be in remission. Different treatments are better at different times for each disease.

Your health and risk factors must also be considered, and that, too, can change over time. For example, some treatments given before a transplant could damage your liver or kidneys, causing a risk factor you did not have before.

Your doctor will consider both your disease status and your current health when deciding on a treatment method. In general, results are better when the transplant is done:

• In the early process of the disease or when the disease is in remission
• When the disease responds to chemotherapy
• When you are as healthy as can be expected and your organs work well

Talking with Your Doctor

When you have a disease that may be treated with a bone marrow or cord blood transplant, talk to your doctor. Ask questions about your disease, your risk factors, and treatment options. Every question you have is important.
Some questions you may want to ask your doctor about treatment options are:

• What treatment do you recommend and why?
• Is the goal to cure my disease, stop the disease progression, or control symptoms?
• What are other possible treatments?
• Will a bone marrow or cord blood transplant be an option at some point in my treatment?

Questions specific to a bone marrow or cord blood transplant might include:

• When do you recommend a transplant?
• When do you begin searching for a marrow donor or cord blood unit?
• What are the risks of waiting or trying other treatments first?
• What do you think the chances are that my transplant will be successful?

For more questions and topics to discuss with your doctor, see:

• Discussing Options with Your Doctor
• Understanding Your Treatment Options