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Chemotherapy and Drug Therapy

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

Low-Intensity Therapy

These drugs are less likely to produce severe side effects and are often given in an outpatient setting. There are two low-intensity chemotherapy drugs approved to treat MDS:

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

Both of these belong to a class of medications known as hypomethylating agents.

Immunosuppressive Therapy

This type of therapy uses drugs that suppress certain parts of the immune system. For instance, in some types of MDS, lymphocytes may attack the bone marrow, causing it to stop making enough healthy blood cells. The main IST drugs used to treat MDS are

  • Anti-thymocyte globulin (ATG) (Thymoglobulin®)
  • Cyclosporine (Neoral®)
  • Tacrolimus (Prograf®). 

This type of therapy does not work well for all types of MDS; it is most effective when MDS has features that are associated with an immune-system attack, such as

  • Presence of HLA-DR15 protein
  • A low number of cells in the bone marrow

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


Immunomodulators (IMIDs)

Immunomodulators modify different parts of the immune system. The following drug is approved to treat lower-risk MDS with 5q deletion [del(5q)]:

  • Lenalidomide (Revlimid®)

Treatment with this oral drug may lessen the need for red blood cell transfusions in certain patients. 

High-Intensity Therapy

This type of therapy involves the use of intensive chemotherapy drugs or stem cell transplantation. High-intensity chemotherapy includes drugs and regimens typically used to treat AML. Because these agents tend to cause more severe side effects, they are generally only used for MDS that is likely to progress to AML (high-risk disease). MDS patients in the intermediate –2 and high-risk IPSS categories may require treatment with high-intensity chemotherapy. The drugs used may include

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

Chemotherapy may be given alone or in combinations of two or three different agents (combination chemotherapy). When treating with combination therapy

  • Low-dose protocols may be used.
  • A patient’s blood cell count may worsen. If cell counts do get worse, a doctor will evaluate the patient’s condition to decide whether intensive chemotherapy may be continued.

Tyrosine Kinase Inhibitors (TKIs)

TKIs are drugs that target the abnormal proteins that cause uncontrolled cell growth. The following drug is approved for adult patients with myelodysplastic syndromes/ myeloproliferative neoplasms (MDS/MPN) associated with PDGFR (platelet-derived growth factor receptor) gene rearrangements:

  • Imatinib mesylate (Gleevec®)

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