Myelodysplastic syndrome (MDS) refers to a group of bone marrow disorders that interfere with the healthy production of blood cells.
Treatments for MDS can range from long-term medication to stem cell transplants. Your options depend on the type of MDS you have and how serious it is. You might also consider your age and overall health when choosing a treatment plan.
Various treatments can slow disease progression, relieve symptoms, and prevent complications. You can also try certain lifestyle changes and natural remedies that may help relieve your symptoms and improve your quality of life.
Your bone marrow is required to produce 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 properly, or at all. 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 it’s 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:
- oncologists
- hematologists
- bone marrow transplant specialists
- primary care physicians
- nurses
The team will develop a treatment plan that’s partially based on the type of MDS you have. The World Health Organization (WHO) identifies
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 it’s scored on several factors, including:
- percentage of blasts (immature blood cells in the bone marrow)
- red blood cell count
- white blood cell count
- platelets in circulation
Before you start MDS treatment, you might also consider your:
- age
- overall health
- treatment preference
Next, we’ll discuss each of the main treatment options for this group of diseases.
Supportive therapy
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:
- Blood transfusion. Also known as transfusion therapy, this treatment boosts levels of red blood cells or platelets. Blood transfusions can help with MDS symptoms but typically only give you temporary relief. Frequent transfusions may lead to organ or tissue injury. They can also cause a buildup of iron in your body, so you may need a treatment known as iron chelation.
- Erythropoiesis-stimulating agents (ESAs). These medications help bone marrow produce more red blood cells. According to a
2019 research review , they are generally safe and effective for people with low-risk MDS and anemia. Potential side effects include high blood pressure, fever, and dizziness. - Antibiotic therapy. This may be necessary if you develop a bacterial infection during treatment.
Chemotherapy
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:
- fever
- nausea
- weakness
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.
Lenalidomide
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. It’s designed to reduce your dependence on blood transfusions.
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- nausea
- diarrhea
- constipation
Antithymocyte globulin
Antithymocyte globulin is in a large group of drugs known as immunosuppressants that weaken the body’s 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 haven’t had effective results with ESAs and transfusions. You might also try it if you have one or more autoimmune diseases.
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Stem cell transplant
A stem cell transplant involves receiving chemotherapy and/or radiation therapy to kill the cells in the bone marrow, and replacing it with healthy blood-forming stem cells donated by someone else.
The procedure is usually reserved for more serious cases of MDS. Stem cell transplant is the closest treatment option to a cure, but it’s 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, many MDS patients will see their condition return over time.
In addition to traditional medications and procedures to treat MDS, complementary or alternative medicine (CAM) may also help provide you with symptom relief. While these therapies may not affect blood cell production, they may help you cope with symptoms such as anxiety and stress that can come with a chronic disease such as MDS.
Consider some of these treatments and remedies after first discussing them with a healthcare professional:
MDS can affect anyone at any age but generally affects adults ages 70 and older. According to the American Cancer Society (ACS), about
Disease outcomes vary considerably. It’s difficult to predict someone’s outcome because people respond differently to treatment.
ACS
These survival rates are based on data that includes years before treatments like chemotherapy were available. It’s 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.
MDS is disorder in which your bone marrow doesn’t produce high enough levels of mature red blood cells, white blood cells, or platelets.
A variety of treatments are available to manage MDS, including blood transfusions, immunosuppressant drugs, chemotherapy, and stem cell transplants. Each treatment comes with its own risks, but your doctor or cancer treatment team will help guide you toward an approach that may work best for you.