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Myelodysplastic syndrome in children

Alexey Kryvenko, medical expert
Last reviewed: 07.07.2025

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Myelodysplastic syndromes (MDS) (preleukemia, small cell leukemia) are a heterogeneous group of clonal disorders characterized by abnormal growth of myeloid components of the bone marrow. Myelodysplastic syndromes are characterized by disruption of normal maturation of hematopoietic cells and signs of ineffective hematopoiesis. In myelodysplastic syndromes, clonal proliferation occurs in the bone marrow at the level of stem cells, in most cases, the production of granulocytes, erythrocytes and platelets is disrupted, which leads to the development of pancytopenia. Another feature of myelodysplastic syndromes is its frequent evolution into acute leukemia, the development of which is preceded by a long-term cytopenic syndrome. Myelodysplastic syndromes are classified as refractory anemias, more advanced stages of the disease reflect transformation into myeloid leukemia.

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Symptoms of myelodysplastic syndrome

The symptoms of myelodysplastic syndrome are a consequence of pancytopenia and include signs of anemia, bleeding due to thrombocytopenia, and infection due to neutropenia. Bone marrow cellularity is usually increased, but may be normal or decreased. The overall risk of transformation to myeloid leukemia is 10-20%, patients with RAS have the lowest risk of leukemic transformation (5%), and RABP-T has the highest (50%).

FAB classification of myelodysplastic syndromes

Refractory anemia (< 5% blasts in bone marrow).
Refractory anemia with ringed sideroblasts (< 5% blasts in bone marrow).
Chronic myelomonocytic leukemia (>20% blasts in bone marrow).
Refractory anemia with increased blast content (5-10% blasts in bone marrow).
Refractory anemia with an increased content of blasts in the transformation stage (10-30% blasts in the bone marrow).

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