Chemotherapy and Drug Therapy

There are many different types of drugs used in the treatment of MDS.

Lower-Risk MDS

Immunosuppressive Therapy. Drugs that suppress certain parts of the immune system can help some patients with lower-risk MDS. In some types of MDS, lymphocytes, a type of white blood cell, may attack the bone marrow, causing it to stop making enough healthy blood cells. Immunosuppressive therapy lowers the body’s immune response to allow bone marrow stem cells to grow and make new blood cells. The main immunosuppressive therapy drugs used to treat MDS are:

  • Antithymocyte globulin (ATG, Atgam®, Thymoglobulin®) by IV infusion 
  • Cyclosporine (Neoral®, Sandimmune®) taken orally 
  • Tacrolimus (Prograf®) taken orally 

These drugs do not work well for all types of MDS. They are most effective when the disease has features associated with an immune-system attack.

Immunomodulators. These are drugs that modify different parts of the immune system and may help the body fight cancer. Lenalidomide (Revlimid®) is used to treat MDS with cells that are missing part of chromosome 5, referred to as del(5q). Treatment with this drug may lessen the need for red blood cell transfusions in certain patients. Lenalidomide is an oral medication. If this treatment is not effective, treatment with hypomethylating agents is another option.

Hypomethylating Agents. These drugs are a type of chemotherapy that work by blocking the DNA that helps cancer cells grow. They also help genes that are involved in cell growth work the way they should. Using one of these drugs may help improve blood cell counts, which may lead to fewer blood transfusions and improve quality of life. They may also slow the progression of MDS. These drugs are, in general, less likely to produce severe side effects.

  • Azacitidine (Vidaza®)
  • Decitabine (Dacogen®). 
  • Decitabine and cedazuridine (Inqovi®)

For information about the drugs listed on this page, visit Drug Listings.

Higher-Risk MDS

Treatment for higher-risk patients depends on whether they are candidates for an allogeneic stem cell transplantation. Regardless of therapy, supportive care should be given to all patients. Patients who are not candidates for a stem cell transplantation may receive one of the drug therapies below. 

Hypomethylating Agents. The major alternative to stem cell transplantation is hypomethylating agents. These drugs are a type of chemotherapy drug that work by blocking the DNA that helps cancer cells grow. They also help genes involved in cell growth work the way they should. Using one of these drugs may help improve blood cell counts which may lead to fewer blood transfusions and improved quality of life. They may also slow the progression of MDS.

  • Azacitidine (Vidaza®) by IV infusion or subcutaneously (under the skin)
  • Decitabine (Dacogen®) by IV infusion 
  • Decitabine and cedazuridine (Inqovi®) taken orally 

Targeted Therapy. This type of treatment uses drugs or other substances to identify and attack specific types of cancer cells with less harm to normal cells. Not all cancers have the same targets. Each type of targeted therapy works a little bit differently, but they all interfere with the growth and survival of cancer cells. To find the most effective treatment, your doctor may run tests to identify genes, proteins and other factors in your cancer cells. This helps the doctor choose the most effective treatment for you based on the specific factors of your disease. Some drugs that are not FDA-approved to treat MDS can be used as an “off label” treatment for patients. “Off-label” prescribing is when a doctor gives a drug that is FDA approved to treat one condition for another condition. Targeted therapy may be used alone or in combination with chemotherapy. Some types of targeted therapy for MDS include:

  • IDH Inhibitors: In some people with MDS, the cancer cells have a mutation of the IDH1 or IDH2 gene. These mutations cause cells to remain immature and divide and multiply too quickly. For these patients, the following targeted therapy, used as off-label treatment, may be used:
    • Ivosidenib (Tibsovo®) taken by mouth
    • Enasidenib (Idhifa®) taken by mouth
  • BCL2 Inhibitors: Overexpression of the BCL2 protein allows cancer cells to evade “programmed cell death,” meaning it helps cancer cells live longer than they should. BCL2 inhibitors target the BCL2 protein. This helps restore what is called apoptosis, a process of natural cell death that is disrupted when you have cancer, restoring the body's natural ability to tell cancer cells to die. Once this process is restored, your body can begin to kill cancer cells.
    • Venetoclax (Venclexta® taken by mouth
  • FLT3 Inhibitors: Some MDS patients have a mutation in the FLT3 gene that can increase the growth and division of cancer cells. FLT3 inhibitors are drugs that target these gene mutations. For MDS patients with CBL mutations, FLT3 inhibitors may also be helpful in treatment. These drugs are not FDA-approved to treatment MDS, but they are being studied in clinical trials and are also used as off-label treatments:
    • Midostaurin (Rydapt®)
    • Gilteritinib (Xospata®)
    • Sorafenib (Nexavar®)

Intensive Chemotherapy. For patients eligible for intensive therapy but who do not have a stem cell donor, the same intensive chemotherapy regimens used for the treatment of acute myeloid leukemia (AML) may be used. Because these agents tend to cause more severe side effects, they are generally used for higher-risk MDS that is likely to progress to AML. The drugs used may include:

  • Cytarabine (cytosine arabinoside, Ara-C; Cytosar-U®)
  • Idarubicin (Idamycin®)
  • Daunorubicin (Cerubidine®)
  • Mitoxantrone (Novantrone®)

Chemotherapy regimens may consist of a single drug or combinations of two or three different drugs (combination chemotherapy).

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