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Like leukemia, myelodysplastic syndrome is a disorder of the bone marrow, the spongy tissue inside bones where all blood cells are formed, and blood. In this disease, the production of normal blood cells, such as platelets (the clotting mechanism of the blood) or white cells (immune cells that fight infection) is affected. Myelodysplastic syndrome occurs mainly in adults, but while very rare, it can occur in children.The cause of myelodysplastic syndrome is currently unknown. There is a higher incidence of myelodysplastic syndrome in children with certain other clinical conditions, such as neurofibromatosis.
Bruising, due to a low platelet (the clotting mechanism of the blood) count, and recurrent infections are the most common signs in children. In some children, the abdomen becomes distended due to an enlarged spleen.
A diagnosis of myelodysplastic syndrome is made through the examination of the peripheral blood (circulating blood in veins and arteries) and the bone marrow. Certain genetic abnormalities in cells are typically associated with these disorders and may help to predict response to treatment and outcome.
Further tests will be done on the bone marrow to confirm the diagnosis and the specific cellular characteristics of the disorder. The diagnosis may not be clear-cut at the initial diagnosis; an observation period may be necessary prior to the administration of therapy.
The treatment of these disorders has changed over the last decade. Children with myelodysplastic syndrome are usually treated with intensive chemotherapy (treatment with anticancer drugs) similar to that of acute myeloid leukemia. Some patients do not respond to chemotherapy and may have to undergo a bone marrow or stem cell transplant to fix the diseased bone marrow. In this treatment, the diseased bone marrow is destroyed using high doses of chemotherapy. Healthy, donor (allogeneic) stem cells or bone marrow is given to the patient intravenously and travels to the bone to repopulate the bone marrow.