Myelodysplastic syndrome (MDS) is a group of rare bone marrow disorders in which the bone marrow fails to produce an adequate number of healthy blood cells. MDS is less common in children than in adults, but it can be a serious condition requiring intensive treatment. The disease leads to impaired production of red blood cells (erythrocytes), white blood cells (leukocytes), and platelets, which can result in anemia, infections, and bleeding.
Main symptoms of myelodysplastic syndrome in children
- Anemia. Pallor of the skin, weakness, fatigue, and dizziness.
- Frequent infections. Due to a deficiency of leukocytes, children may be more susceptible to infections, which can be severe and complicated.
- Bleeding and bruising. Low platelet counts may lead to bleeding, nosebleeds, gum bleeding, and unexplained bruises.
- Enlargement of the liver and spleen. Some children may develop hepatomegaly or splenomegaly.
Diagnosis of myelodysplastic syndrome
- Blood tests. A complete blood count (CBC) is performed to assess levels of red blood cells, white blood cells, and platelets. A peripheral blood smear is used to evaluate the shape and morphology of blood cells under a microscope.
- Bone marrow biopsy. A sample of bone marrow (usually obtained from the pelvic bone) is examined to confirm the diagnosis and assess the degree of impaired blood cell production.
- Genetic testing. Cytogenetic and molecular studies are performed to identify chromosomal abnormalities associated with MDS.
Treatment of myelodysplastic syndrome in children in Belarus
Treatment depends on the child’s age, severity of symptoms, bone marrow findings, and the presence of genetic abnormalities.
- Supportive therapy. Blood transfusions may be required to manage anemia and prevent bleeding and are administered as needed. Antibiotics are used to treat or prevent infections. Hematopoietic growth factors may be prescribed to stimulate red blood cell production.
- Immunosuppressive therapy. In selected cases where the immune system attacks the bone marrow, immunosuppressive medications may be used.
- Chemotherapy. Chemotherapy is indicated in more aggressive forms of MDS or when the disease progresses to leukemia. It helps eradicate abnormal bone marrow cells and prepare the patient for stem cell transplantation.
- Stem cell (bone marrow) transplantation. This is the only potentially curative treatment for MDS. It is considered in severe cases or when other therapies are ineffective. Transplantation involves replacing the diseased bone marrow with healthy stem cells from a compatible donor (related or unrelated). Prior to transplantation, intensive chemotherapy and/or radiation therapy is administered to eliminate abnormal bone marrow cells.
Factors influencing treatment outcomes
- Type and stage of MDS. Prognosis depends on the specific subtype of myelodysplastic syndrome and the presence of genetic abnormalities. Certain cytogenetic changes are associated with a higher risk of progression to acute myeloid leukemia (AML).
- Age and overall health. Younger age and better general health are associated with improved treatment outcomes.
- Response to therapy. A favorable response to treatment is associated with a more positive prognosis.
Treatment of myelodysplastic syndrome in children is a complex and highly individualized process that requires a comprehensive approach. Supportive care, chemotherapy, and stem cell transplantation are the main treatment options. Early diagnosis and appropriate therapy can significantly improve outcomes. Parents play a crucial role by closely monitoring their child’s condition and working in close collaboration with the medical team to achieve the best possible results.
