What is myelodysplastic syndrome (MDS)?

20150325_Drew-1When a 6-year-old boy who loves Facebook missed Halloween because of a rare blood disorder, inpatient nurses at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center stepped in. They organized a belated Halloween celebration, and Boston Children’s Hospital posted a photo on its Facebook page, along with a request “to show him support and cheer him on.” Now it was Facebook users’ turn to step in, and step in they did. By the next morning, the post had more than 10,000 likes. Local media and Dana-Farber Cancer Institute posted the story on their Facebook pages, leading to thousands more likes. By the next week, the Boston Children’s post had attracted more than 17,000.

The disease that caused young Drew to miss Halloween is pediatric myelodysplastic syndrome (MDS), for which he was treated with a stem cell transplant. While MDS is more common in adults – “Good Morning America” co-host Robin Roberts was treated for it in 2012 – it occurs in only four of every million children.

What is pediatric myelosdysplastic syndrome?

MDS develops in the bone marrow, the soft, spongy center of long bones that produces white blood cells that fight infection, red blood cells that carry oxygen and platelets that help blood clot to stop bleeding. The cells in the bone marrow that produce these cells are called blood stem cells. MDS disturbs the process by which stem cells mature to become red cells, white cells and platelets. About one-third of children with MDS develop leukemia, usually within months or a few years.

What are the symptoms of MDS?

The most common symptom is bleeding or abnormal bruising, due to a low platelet count. Other symptoms include anemia, which occurs when problems in the production of red blood cells decrease oxygen in the blood, making the child appear tired or pale or breathe faster than normal. A child with MDS may be prone to infections because of an inadequate amount of infection-fighting white blood cells. Sometimes MDS is picked up accidentally in a routine blood test for something else.

How is MDS in children treated?

Treatment of the consequences of MDS includes blood transfusions of red cells and platelets to improve symptoms of anemia and to prevent bleeding. Antibiotics are often used to treat infections. The only cure for MDS is a stem cell transplant, often called a bone marrow transplant. The child is given high doses of chemotherapy and/or radiation therapy  to destroy all the cells in the bone marrow, healthy and diseased ones. Healthy cells from the bone marrow of another person—either a relative (usually a sibling) or an unrelated individual—are given to the patient through an infusion to replace the diseased bone marrow with healthy marrow. Without a stem cell transplant, the long-term outlook for children with MDS is poor.

Learn about Dana-Farber/Boston Children’s Myelodysplastic Syndrome Specialty Program.