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Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Myelodysplastic Syndromes


The French-American-British (FAB) and World Health Organization (WHO) classification systems of MDS and MPS have been difficult to apply to pediatric patients. Alternative classification systems for children have been proposed, but none have been uniformly adopted, with the exception of the modified 2008 WHO classification system.[14,15,16,17,18] The WHO system [19] has been modified for pediatrics.[17]

Diagnostic Categories for Myelodysplastic and Myeloproliferative Disease in Children

  • Down syndrome disease
    • Transient myeloproliferative disorder.
    • Myeloid leukemia of Down syndrome.
  • Myelodysplastic/myeloproliferative disease
    • Juvenile myelomonocytic leukemia.
  • Myelodysplastic syndrome
    • Refractory cytopenia (also called refractory anemia)-peripheral blood blasts less than 2% and bone marrow blasts less than 5%.
    • Refractory anemia with excess blasts-peripheral blood blasts 2% to 19% or bone marrow blasts 5% to 19%.
    • Refractory anemia with excess blasts in transformation-peripheral blood or bone marrow blasts 20% to 29%. In the FAB classification, refractory anemia with excess blasts in transformation required evidence of dysplasia, particularly in the red blood cell lineage, and 21% to 30% myeloblasts in the bone marrow; if there was greater than 30% myeloblasts this was considered to be AML. In part because of the artificial designation of the percentage of blasts, the WHO classification system now simply considers these patients to have AML and the refractory anemia with excess blasts in transformation subtype has been eliminated.

The refractory cytopenia subtype represents approximately 50% of all childhood cases of MDS. The presence of an isolated monosomy 7 is the most common cytogenetic abnormality, although it does not appear to portend a poor prognosis compared with its presence in overt AML. However, the presence of monosomy 7 in combination with other cytogenetic abnormalities is associated with a poor prognosis.[20,21] The relatively common abnormalities of -Y, 20q- and 5q- in adults with MDS are rare in childhood MDS. The presence of cytogenetic abnormalities found in AML defines disease that should be treated as AML and not MDS.[22]

The International Prognostic Scoring System can help to distinguish low-risk from high-risk MDS, although its utility in children with MDS is more limited than in adults as many characteristics differ between children and adults.[22,23] The median survival for children with high-risk MDS remains substantially better than adults and the presence of monosomy 7 in children has not had the same adverse prognostic impact as in adults with MDS.[24]

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