Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Children with Down Syndrome
The mean time for the development of AML in the 10% to 30% of children who have a spontaneous remission of TMD but then develop AML, has been reported to be approximately 16 months with a range of 1 to 30 months.[11,15,17] Thus, most infants with Down syndrome and TMD who later develop AML will do so within the first 3 years of life. Patients with Down syndrome who develop AML with an antecedent TMD have superior event-free survival (EFS) (91% ± 5%) compared with such children without TMD (70% ± 4%) at 5 years. While TMD is generally not characterized by cytogenetic abnormalities other than trisomy 21, the presence of additional cytogenetic findings may connote an increased risk for developing subsequent AML.
For children with Down syndrome who develop AML, outcome is generally favorable. The prognosis is particularly good (EFS exceeding 80%) in children aged 4 years or younger at diagnosis, the age group that accounts for the vast majority of Down syndrome patients with AML. Appropriate therapy for these children is less intensive than current AML therapy, and hematopoietic stem cell transplant is not indicated in first remission.[3,17,19,20,21,22,23,24]
Children with mosaicism for trisomy 21 are recommended to be treated similarly to those children with clinically evident Down syndrome. Children with Down syndrome who are older than 4 years have a significantly worse prognosis. Although an optimal treatment for these children has not been defined, they are usually treated on AML regimens designed for children without Down syndrome.
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