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T2016-003: Epigenetic Reprogramming in Relapsed/Refractory AML


Patients must have relapsed/refractory Acute Myelogenous Leukemia (AML).


This is a pilot study using decitabine and vorinostat before and during chemotherapy with fludarabine, cytarabine and G-CSF (FLAG). Decitabine is a demethylating agent and vorinostat is a HDAC inhibitor. The use of demethylating agents and HDAC inhibitors in combination have been previously shown to have synergistic effects in altering neoplastic pathways of cancer cells and be well tolerated in human clinical studies. With the ability of decitabine and vorinostat to alter the abnormal cellular pathways of leukemic blasts and essentially turn off anti-apoptotic proteins, the leukemia cells have become primed for cytotoxic cell kill via chemotherapeutic agents. This study will ask the question as to whether or not the combination of decitabine and vorinostat followed by chemotherapy is feasible and whether it can positively impact outcome in patients with relapsed or refractory acute myelogenous leukemia.

Eligibility Criteria

  • Ages eligible for study: 1 year to 25 years (Child, Adult)
  • Patients with AML must have ≥ 5% blasts (by morphology) in the bone marrow
    • Patients may have CNS or other sites of extramedullary disease. No cranial irradiation is allowed during the protocol therapy.
    • Patients with secondary AML are eligible
    • Patients with Down syndrome and DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are excluded.
  • Performance level: Karnofsky >50% for patients >16 years of age and Lansky > 50% for patients ≤ 16 years of age
  • Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.

Detailed inclusion and exclusion criteria as listed on



Eric S. Schafer, MD, MHS
Local Principal Investigator
Texas Children’s Cancer and Hematology Center