How is acute myelocytic leukemia (AML) treated in children with Down syndrome?

Updated: Sep 12, 2017
  • Author: Mark E Weinblatt, MD; Chief Editor: Jennifer Reikes Willert, MD  more...
  • Print

Unlike most children with acute myeloid leukemia who should receive intense therapy, young children (< 4 y) with Down syndrome fare best with reduced-intensity therapy, which results in an improved likelihood of long-term, disease-free remission. Many children with trisomy 21 have had transient myeloproliferative disease as infants. This picture resembles acute myeloid leukemia in many ways, but it usually disappears with only supportive care. About 20-30% of the children who had this syndrome as neonates develop true acute myeloid leukemia requiring chemotherapy.

Children with Down syndrome also seem to have marked complications of intense therapy. As a result, treatment for children with trisomy 21 involves lowered doses of induction chemotherapy (daunomycin, cytosine arabinoside, and 6-thioguanine) with prolonged periods between treatments. These children receive intensified chemotherapy high-dose cytosine arabinoside rather than bone marrow transplantation. Consolidation and intensification courses of therapy with high-dose cytosine arabinoside do not cause increased toxicity or mortality in patients with Down syndrome.

Age has been shown to be an important prognostic factor for children with Down syndrome; children younger than 2 years have the best outlook. A COG study (A2971) has shown that the 2-year-old to 4 year-old age group does as well as those younger than 2 years. Older children with Down syndrome continue to have a worse outlook than children younger than 4 years.

Did this answer your question?
Additional feedback? (Optional)
Thank you for your feedback!