What is 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).

David Steensma, MD

“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 MDS treatment.

“Patients have problems from low blood counts before they get leukemia, so MDS is a more accurate term, even thought it is more complex.”

Dr. Steensma says that while drug therapy can benefit patients, the only potentially curative treatment for MDS 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.

We talked with Dr. Steensma, who cares for MDS patients in Dana-Farber’s Hematologic Oncology Program, about what MDS is and how it’s treated.

Q. What are the causes of myelodysplastic syndrome, and who is most likely to develop it?

A. In most cases we don’t know the cause, although exposures to radiation or industrial chemicals like benzene and xylene are risk factors. Most patients are 60 years old or above, but MDS can occur at younger ages, even in childhood, especially in patients who have been previously treated with chemotherapy or radiation. MDS affects only a small subset of patients treated with chemotherapy or radiation treatment. When it occurs, it happens typically five to seven years after treatment, but I’ve seen cases where it occurs 20 years later.

Q. What are typical symptoms that lead to a diagnosis?

A. Symptoms include fatigue, shortness of breath, easy bruising or bleeding, pale skin, and fever or frequent infections. But in many cases MDS causes no symptoms and is discovered during a routine blood examination. The results of a bone marrow biopsy are key to making the diagnosis.

Q. What do patients experience as the disease progresses?

A.  As bone marrow failure worsens, anemia becomes more severe and patients need frequent blood transfusions. About 30 percent of patients go on to develop acute myelogenous leukemia (AML), usually within a few months to a few years. A person’s genetic makeup may influence how likely this is to occur.

Q.  How is MDS treated?

A.  Patients often receive blood growth factors in addition to transfusions, and there are three drugs approved by the FDA that can slow the course of the disease and help people live longer. The length of time before patients develop AML can also be prolonged by drug treatment. Meanwhile, there are a number of experimental treatments in clinical trials.

The only treatment that can cure MDS at present is a stem cell transplant. Unfortunately, many patients who have MDS can’t get a transplant because they are either too old (the upper limit currently is in the early 70s) or have other medical problems that make a transplant too risky.

Q.  How successful are transplants, and can most people with MDS easily find a donor?

A.  We can cure up to half of eligible patients with a transplant from a matched donor.  But only about 50 percent of patients who are transplant candidates actually get them; the rest can’t find a match. It’s especially difficult for patients who are minorities to locate a matched donor because there are fewer minorities in the donor registries. We hope that greater awareness of MDS will lead to more people contacting the National Marrow Donor Program’s Be The Match Registry. You need only to provide a cheek swab sample to find out if you’re a potential map, and giving a donation of blood stem cells is a relatively painless process.

To join the Be The Match Registry of the National Marrow Donor Program (NMDP) at Dana-Farber or to get more information, call 866-875-DFCI (3324) or 617-632-2561 or email nmdpdonor@dfci.harvard.edu.

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