August 9, 2014 | by Kim Proescholdt
Myelodysplasia, sometimes referred to as myelodysplastic syndrome or MDS, is a rare group of blood disorders caused by disrupted development of blood cells within the bone marrow, resulting in a decreased number of healthy blood cells. People diagnosed with the condition, considered a precancer, may be at greater risk for leukemia.
According to the American Cancer Society, approximately 13,000 new cases of myelodysplastia are diagnosed each year. As the average age of the population in the U.S. goes up, the number of new cases seems to be increasing.
Here, Margaret O’Donnell, M.D., clinical professor and associate clinical director for City of Hope’s Hematopoietic Cell Transplantation Program, sorts out the facts about myelodysplasia. She also describes how our world-class experts excel in treating these complicated diseased and how the team works continuously to better understand and more effectively treat myelodysplasia.
What is myelodysplasia and who is most at risk?
Myelodysplasia is a group of diseases in which the bone marrow doesn't produce enough healthy blood cells, instead making too many immature cells. These immature cells die in the marrow or soon after entering the bloodstream, resulting in too few healthy blood cells and low blood counts.
Although many cases of myelodysplasia have no known cause, there are commonly accepted risk factors that can increase a person’s likelihood of developing the disease. Myelodysplasia usually occurs in older people, typically starting after age 60, although pediatric cases do occur, especially after previous chemotherapy. It is also more common in Caucasian males.
For some people, secondary myelodysplasia may develop after being exposed to chemotherapy or radiation therapy, and exposure to certain chemicals, including tobacco smoke, pesticides, heavy metals such as mercury or lead, and solvents such as benzene.
What are symptoms of myelodysplasia?
People with myelodysplasia have blood cells that are immature and defective, and instead of developing normally, they die in the bone marrow or just after entering the bloodstream. Over time, the number of immature, defective cells begins to surpass that of healthy blood cells, leading to problems such as anemia, infections and excess bleeding.
Myelodysplasia rarely cause signs or symptoms in the early stages of the disease. However, in time, myelodysplasia may cause fatigue, shortness of breath, unusual paleness due to anemia, easy or unusual bruising or bleeding, pinpoint-sized red spots just beneath the skin caused by bleeding (petechiae) and/or recurrent infections.
What are treatments for myelodysplasia?
The goal of treatment is to improve the number of healthy blood cells circulating in the bloodstream. So to achieve this, treatment can vary greatly from person to person. In other words, one size does not fit all.
For patients with lower-risk myelodysplasia, such as those with low blood counts and mild anemia, a red blood cell stimulating agent such as erythropoietin may be administered to see whether red cell production will improve enough that a transfusion won’t be needed. If more aggressive therapy is needed, the team of experts here at City of Hope work together to develop short- and long-term goals for the overall best treatment plan that often includes chemotherapy.
Higher-risk myelodysplasia patients usually need more aggressive therapy, but much depends on the age and condition of the patient. Younger patients with high-risk disease are considered for front-line chemotherapy approaches followed perhaps by allogeneic stem cell transplantation. For older patients, intensive chemotherapy is rarely considered.
Patients with myelodysplasia that do not respond to traditional chemotherapy also have access to innovative leukemia regimens being tested in clinical trials here at City of Hope.
What research is being conducted at City of Hope for myelodysplasia?
At City of Hope, patients with myelodysplasia benefit from one of the most active hematology research programs in the U.S. We are able to offer a range of clinical trials of new treatments for myelodysplasia.
We’ve conducted a lot of research to identify patients who may have an increased risk of developing myelodysplasia as a consequence of having undergone past chemotherapy treatment. All of our autologous transplant patients have long-term follow-up with repeated bone marrow examinations to look for changes in molecular markers. Dr. Ravi Bhatia’s lab is heavily involved in this research. We are also conducting clinical trials looking at newer agents, either alone or in combination with the hypomethylating agents, as new means of therapy.
Years ago, we didn’t have what we have today. Now, we have therapies that are curative. It’s a great feeling to be part of this process and know what we do is evolving the field and helping patients every day.
Why did you choose this specialty? What inspires you to do the work you do?
Hematology oncology is incredible and challenging, but always exciting. You can tell you’re having an impact relatively simply when you treat patients with blood disorders. The field of hematology is intellectually a fascinating biologic process and no case is ever the same. But when you can help someone become well again and back to a normal life, it is so gratifying. To know I can help cure someone is very rewarding. It’s been exciting to live in this time period to witness the change.
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