Treatments for Myelodysplastic Syndrome (MDS)

There is only one generally recognized curative treatment for MDSallogenic stem cell transplant (SCT), or bone marrow transplant (BMT). Most doctors recommend SCT for otherwise healthy patients under the age of 50. The American Cancer Society states about one-third of MDS patients receiving allogenic SCT may be cured, but between one-fourth to one-third may die from complications of this treatment.

Certain medications have also helped treat the disease. One called 5-azacytidine has produced positive results in several trials, apparently by stimulating bone marrow cells to resume their differentiation into mature, functioning cells. Others, like thalidomide, have also recently shown some benefit for patients.

All other interventions are supportive and depend on which family of blood cells is involved. The mainstays of treatment have been blood component transfusions to replace the deficient cell types and antibiotics to treat the infections. Radiation therapy is not used for the treatment of MDS.

Promising new treatments on the horizon have recently generated increased interest. The US Food and Drug Administration (FDA) approved the drug Revlimid (lenalidomide) in 2005 for MDS patients with a certain subset of the disease called 5q Syndrome. Clinical Trials Treatment protocols have been established and continue to be modified through clinical trials. The research studies are essential to determine whether or not new treatments are both safe and effective. Since highly effective treatments for many cancers remain unknown, numerous clinical trials are always underway around the world. You may wish to ask your doctor if you should consider participating in a clinical trial. You can find out about clinical trials at the government website ClinicalTrials.gov. References: American Cancer Society website. Available at: http://www.cancer.org/. Castro-Malaspina H, OReilly RJ. Aplastic anemia and the myelodysplastic syndromes. In Harrison's Principles of Internal Medicine. 14th ed. McGraw-Hill; 1998. Childrens Hospital Boston website. Available at: http://www.childrenshospital.org. Lewis R. Silverman MD. Myelodysplastic syndrome. In: Cancer Medicine. American Cancer Society website. Available at: http://www.nci.nih.gov/cancer_information/. Accessed November 2002.
Last reviewed July 2007 by Igor Puzanov, MD Please be aware that this information is provided to supplement the care provided by your physician. It is neither intended nor implied to be a substitute for professional medical advice. CALL YOUR HEALTHCARE PROVIDER IMMEDIATELY IF YOU THINK YOU MAY HAVE A MEDICAL EMERGENCY. Always seek the advice of your physician or other qualified health provider prior to starting any new treatment or with any questions you may have regarding a medical condition.
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