Stem Cell Transplant the only 'definitive treatment' for myelodysplastic syndrome

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When Good Morning America host Robin Roberts revealed that she has myelodysplastic syndrome (MDS), she turned a spotlight on a group of blood disorders that affect an estimated 35,000 to 55,000 people in the United States.

In patients with MDS, the bone marrow fails to produce normal quantities of blood cells and the cells themselves are often abnormal, resulting in anemia and an array of symptoms including paleness, fatigue, susceptibility to infections and easy bruising or bleeding. The syndrome, of which there are at least 15,000 new diagnoses each year in the United States, formerly was known as "pre-leukemia" because about one-third of patients go on to develop acute myelogenous leukemia (AML).

"But the term was misleading, because most people with MDS never get leukemia," says David Steensma, MD, a medical oncologist at Dana-Farber, which is designated a Center of Excellence by the MDS Foundation in myelodysplastic syndrome treatment. "Patients have problems from low blood counts before they get leukemia, so MDS is a more accurate term, even though it is more complex."

He says that while drug therapy can benefit patients, the only potentially curative treatment for myelodysplastic syndrome is a stem cell or bone marrow transplant, which can cure up to 50 percent of patients. Wider awareness of MDS and the need for donors could increase the pool of potential donors and make more life-saving transplants possible, says Steensma.

Dana-Farber's Insight talked with Dr. Steensma, who cares for patients with myelodysplastic syndrome in Dana-Farber Cancer Institute's Hematologic Oncology Program, about what MDS is and how it's treated.


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