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Myelodysplastic syndromes (MDS) are rare in childhood and represent only about 5% to 7% of all pediatric hematological malignancies. Research suggests that about 30% of children with MDS have an inherited genetic disorder such as Fanconi's anemia, Down's syndrome, or Shwachman's sydnrome. In general, myelosdylplastic syndromes in children tend to follow an aggressive clinical course and are characterized by the absence of subgroups such as RARS as well as by the presence of a variant type of MDS called juvenile myelomonocytic leukemia (JMML). The incidence of JMML is higher in males than females and children with neurofibromatosis-type 1 are at higher risk for developing this disorder. JMML is a particularly aggressive form of MDS with a median survival rate of less than 10 months after diagnosis.

Treatment for children with myelodysplastic syndromes tends to be aggressive with the goal to cure the disease by eradicating the malignant clone and restoring normal hematopoiesis. It has been reported that supportive treatments, such as the use of hematopoietic growth factors and differentiating agents, are of limited use in treating children with MDS. Like adults, the remission followed by intensive chemotherapy tends to be short in duration. Studies to determine if chemotherapy prior to allogeneic stem cell transplantation was more effective demonstrated similar results to children who were directly transplanted without prior chemotherapy. Allogeneic stem cell transplantation is currently the only treatment that has effectively cured a significant population of children with myelodysplastic syndromes.