About this study
Acute myelogenous leukemia (AML) is a cancer of the bone marrow, the spongy tissue inside the large bones of the body where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood cells called blasts. These blast cells crowd out the normal cells of the bone marrow. They may also invade body organs including the brain, testes, ovaries, or skin. These cancerous AML cells can sometimes form a solid tumor called a chloroma.
This study will compare the good and bad effects of giving either azacitidine OR decitabine before the usual chemotherapy regimen for childhood AML. Azacitidine and decitabine both belong to a class of drugs called "DNA methyl-transferase inhibitors" or DMTi. DMTi’s are known to alter the DNA within the genes of leukemia cells, possibly making them more sensitive to chemotherapy. Giving a DMTi before the usual chemotherapy is called "epigenetic priming" because the intent is to change the genetics of the leukemia cell by “priming” it to be more sensitive to the chemotherapy that will follow.
The U.S. Food and Drug Administration (FDA) has approved azacitidine and decitabine to treat adults with myelodysplastic syndrome. The FDA has not approved these drugs for treating children with leukemia.
Eligibility overview
- Diagnosis of 1 of the following:
- Acute myeloid leukemia (AML)
- 5% to 20% marrow myeloblasts and evidence of a clonal de novo AML genetic abnormality
- Myeloid sarcoma
- High grade myelodysplastic syndrome (MDS) with greater than 5% blasts
- Treatment-related myeloid neoplasms, including AML and MDS
- 28 days to 21 years old
- No prior therapy, except for one dose of intrathecal therapy and the use of hydroxyurea or low-dose cytarabine
- Not pregnant