Do targeted therapies treat myelodysplastic/myeloproliferative diseases?

There is no cure for myelodysplastic syndromes, but there are treatments to relieve symptoms and extend life. Patients initially receive drugs to boost their blood cell counts, such as epoietin alfa (Procrit) for red blood cells. If the M.D.S. does not respond well to this treatment, patients need blood transfusions once or twice a month for life.

The effectiveness of thalidomide has been established in patients with M.D.S. Today its chemical cousin, lenalidomide (Revlimid), is commonly prescribed for M.D.S. Azacitidine (Vindaza) and decitabine (Dacogen) are two other drugs also prescribed for M.D.S. – azacitidine is given by injection and decitabine is given intravenously (by vein). These new drugs, which were approved for M.D.S. in the last decade, have improved the outlook for patients diagnosed with the disease.


Some patients benefit from hematopoietic stem cell transplantation, in which they receive healthy bone marrow stem cells from a donor to repopulate their own diseased bone marrow after they have received high doses of chemotherapy to wipe it out. But patients over age 55 – the bulk of M.D.S. patients – cannot tolerate this treatment. M.D.S. researchers are therefore seeking to cure the disease at its roots.

Targeted therapies can be used to treat myelodysplastic/myeloproliferative diseases. Genetic changes lead to the overgrowth of cells in the bone marrow. In some cases of chronic myelomonocytic leukemia, the PDGFBR oncogene contributes to the overgrowth of cells. Imatinib mesylate can be used as a treatment in these cases. Mutations in ras or related genes may lead to cell overgrowth in some cases of juvenile myelomonocytic leukemia. In some of these cases, farnesyltransferase inhibitors may be effective.

Continue Learning about Hematologic/Blood Cancers

Hematologic/Blood Cancers

Caused when our bone marrow makes too many blood cells, cancers of the blood lead to anemia, frequent infections and excessive bleeding.Common types of blood cancer, also called hematologic cancer, are leukemia, lymphoma and multi...

ple myeloma. These three cancers form when abnormal white blood cells crowd out the healthy ones that are responsible for fighting infections. Other myeloma disorders cause too many red blood cells and excessive platelets to be produced. When this happens, we become more susceptible to abnormal blood clotting or excessive bleeding. Although the exact cause of blood cancers are unknown, your risk increases as you get older and if you are exposed to excessive radiation or certain chemicals. You are also more vulnerable to these diseases if you are a male. See your doctor if you have fever and night sweats, pain in your stomach, or swollen lymph nodes.

Important: This content reflects information from various individuals and organizations and may offer alternative or opposing points of view. It should not be used for medical advice, diagnosis or treatment. As always, you should consult with your healthcare provider about your specific health needs.