Myelodysplastic Syndrome Treatment Options – Healthline


Your bone marrow produces immature blood cells, also called stem cells. These then develop into one of three types of mature blood cells:

If you have MDS, not all of these stem cells mature. As a result, immature cells can remain in the bone marrow or die. You will have lower levels of mature blood cells in your bloodstream.

A reduced mature blood cell count is a condition known as cytopenia, and its a main characteristic of MDS. A blood test known as a complete blood count (CBC) is one of the first diagnostic tests your doctor will order if they suspect you have MDS.

Removing bone marrow samples through aspiration and biopsy can also help your doctor better understand the nature of your blood marrow disorder. Once your doctor has diagnosed and treated your MDS, these tests can also show how well your bone marrow is responding to treatment.

You will often have a healthcare team if you have this type of disorder, which may include:

The team will develop a treatment plan thats partially based on the type of MDS you have. The World Health Organization (WHO) identifies six main types of MDS.

Your treatment plan may also be based on the prognostic score (outlook for survival) of the disease. MDS is different from most forms of cancer, which are grouped in stages and grades, because its scored on several factors, including:

Before you start MDS treatment, you might also consider your:

Next, well discuss each of the main treatment options for this group of diseases.

Supportive therapy is meant to treat MDS symptoms and prevent complications, rather than treat the underlying disorder itself. Supportive therapy is often used alongside other treatments.

Examples of supportive therapy include:

Lenalidomide is a type of medication called an immunomodulatory agent. Your doctor may suggest this oral medication if you have MDS and a certain genetic change known as an isolated del(5q) chromosome abnormality.

Lenalidomide helps boost red blood cell production in your bone marrow. Its designed to reduce your dependence on blood transfusions.

Experts in a 2017 research review called it an excellent option for MDS patients with low or intermediate disease risk. Potential side effects include:

Antithymocyte globulin is in a large group of drugs known as immunosuppressants that weaken the bodys immune response. Organ transplant recipients usually take them to help prevent rejection of the new organ. You may take antithymocyte globulin to keep your immune system from attacking stem cells in your bone marrow.

Immunosuppressant therapy may be appropriate for you if you have lower-risk MDS and havent had effective results with ESAs and transfusions. You might also try it if you have one or more autoimmune diseases.

One 2018 study found that antithymocyte globulin was effective in about 50 percent of the MDS cases studied.

Certain chemotherapy drugs, known as hypomethylating agents, activate specific genes in your stem cells to help them mature. Two examples of these agents are azacitidine and decitabine. These drugs are used when your doctor determines there is a serious risk for leukemia, which is a serious potential complication of MDS.

Chemotherapy can help improve blood cell counts, sometimes to the point where you no longer need transfusions, and reduce your risk of leukemia. Potential side effects include:

Other chemotherapy drugs may be given with the goal of killing atypical stem cells and allowing more of your healthy cells to mature.

While high-dose chemotherapy drugs can be very effective at sending MDS into remission, the side effects can be severe. You could experience a serious drop in white blood cells, and later, a greater risk of infections developing and progressing quickly.

A stem cell transplant involves removing some of your bone marrow, usually from the pelvic bone, and replacing it with bone marrow that produces healthy blood cells.

The procedure is usually reserved for more serious cases of MDS. Stem cell transplant is the closest treatment option to a cure, but its an invasive and challenging therapy. Because of this, stem cell transplant is limited to people who are considered healthy enough for both the procedure and the recovery process.

Doctors often use chemotherapy drugs with stem cell transplant to treat MDS. Together, they help support the growth of healthy blood cells to replace unhealthy or atypical cells.

While transplants are often very effective at achieving disease remission, particularly in certain patients, the Aplastic Anemia and MDS International Foundation says that a majority of MDS patients will see their condition return over time.

MDS can affect anyone at any age but generally affects adults ages 70 and older. According to the American Cancer Society (ACS), about 10,000 people are diagnosed with MDS in the United States per year.

Disease outcomes vary considerably. Its difficult to predict someones outcome because people respond differently to treatment.

ACS survival statistics range from a median survival of 8.8 years for people with a very low risk score to less than 1 year for MDS patients with a very high risk score.

These survival rates are based on data that includes years before treatments like chemotherapy were available. Its important to remember that researchers are continuing to develop new treatments that may improve disease outcomes.

You may have no symptoms early on or if you have mild MDS. However, for most people with the disease, anemia (low red blood cell counts) and symptoms such as chronic fatigue and shortness of breath are common.

If you have low white blood cell counts, the risk of serious infection may always be present. Low platelet counts can lead to easy bruising and bleeding complications.

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Myelodysplastic Syndrome Treatment Options - Healthline

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