Myelodysplastic Syndrome (MDS) Treatment

Reviewed by: HU Medical Review Board | Last reviewed: May 2023

Myelodysplastic syndromes (MDS) are blood cancers that arise from abnormal blood cells in the bone marrow. There are various forms of MDS. Treatment decisions are based in part on the type of MDS and the age and general health of the person. Each treatment option has its own benefits and potential side effects.1

Types of treatment for myelodysplastic syndromes

Different types of treatment may be used for MDS, including:1,2

  • Supportive care
  • Chemotherapy
  • Stem cell transplant
  • Watchful waiting
  • Immunosuppressants

Supportive care

In people with MDS, cancerous cells in the bone marrow cause abnormal blood cells and can cause a change in how normal healthy red blood cells, white blood cells, and platelets function. When the number of healthy blood cells is too low, it can cause symptoms like:2,3

  • Fatigue and lightheadedness
  • Shortness of breath
  • Easy bruising and bleeding
  • Frequent infections

Lab findings from blood tests may show:

  • Low red blood cell counts (anemia)
  • Low white blood cell counts (neutropenia)
  • Low platelet counts (thrombocytopenia)

Supportive care for people with MDS can treat the symptoms of low blood counts. It is often used along with other treatments for MDS. Supportive care for MDS may include receiving:2,3

  • Growth factors to boost red blood cells
  • Growth factors to boost platelets
  • Growth factors to boost white blood cells
  • Blood transfusions
  • Antibiotics to prevent infections

People who receive multiple blood transfusions over several years can be at risk of organ damage due to a buildup of extra iron. They may be treated with iron chelation therapy to help remove the excess iron.2,3

Drug therapy

Different drugs may be used to treat people with MDS. Some drugs can reduce the need for transfusions of red blood cells. Immunosuppressive therapy with anti-thymocyte globulin (ATG) and cyclosporine helps suppress the immune system.2,4

In some people with MDS, the lymphocytes (a type of white blood cell that helps control immune reactions) can interfere with healthy blood cell production. Suppressing the immune system can help increase the number of healthy blood cells.2,4

Chemotherapy

Chemotherapy is the use of drugs to stop cancer cells. Chemotherapy works by targeting fast-growing cells, such as cancer cells. However, there are other fast-growing cells in the body that can also be affected, such as those in the gastrointestinal tract and hair.4

Chemotherapy drugs may be used along with other drugs or alone. They may be taken by mouth or injected into a vein or muscle.4

A "low-intensity" chemotherapy option like a hypomethylating agent (azacitidine or decitabine) may be used to improve blood counts, though this is not a cure for blood cancer.4

Stem cell transplants

Stem cell transplants are used along with high doses of chemotherapy and may be a treatment option for some people with MDS. The high doses destroy cancer cells. However, they also damage healthy blood cells. The transplant of stem cells (immature cells that can become new blood cells) is given to restore the bone marrow.2

In MDS, the stem cells are typically gathered from a donor. This is called an allogeneic transplant. Less often, stem cells may be gathered from the person with MDS before chemotherapy. This is called an autologous transplant. Not everyone is a candidate for stem cell transplants. The high doses of chemotherapy can be very taxing on a person’s body and may not be tolerated by older adults or those with other health problems.2

Clinical trials

Clinical trials are a type of research where new treatments are studied. Clinical trials are an important part of the scientific process to find and prove the safety and effectiveness of new treatments. They offer people a chance to receive the latest treatments and be closely monitored by doctors. You can learn more about clinical trials by talking to your doctor or visiting the ClinicalTrials.gov website. Your doctor can help you decide if a clinical trial may be right for you.2,5

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