A multicenter, randomized study of decitabine as epigenetic priming with induction chemotherapy in children with AML.

ABSTRACT:

Background

Decitabine is a deoxycytidine nucleoside derivative inhibitor of DNA-methyltransferases, which has been studied extensively and is approved for myelodysplastic syndrome in adults but with less focus in children. Accordingly, we conducted a phase 1 multicenter, randomized, open-label study to evaluate decitabine pre-treatment before standard induction therapy in children with newly diagnosed AML to assess safety and tolerability and explore a number of biologic endpoints.

Results

Twenty-four patients were fully assessable for all study objectives per protocol (10 in Arm A = epigenetic priming induction, 14 in Arm B = standard induction). All patients experienced neutropenia and thrombocytopenia. The most common grade 3 and 4 non-hematologic adverse events observed

SUBMITTER: Gore L 

PROVIDER: S-EPMC5629751 | biostudies-literature | 2017

REPOSITORIES: biostudies-literature