Efficacy, Safety, And Pharmacokinetics (Pk) Of Azacitidine (Aza) In Children And Young Adults With Acute Myeloid Leukemia (Aml) In The Phase 2 Aza-Aml-004 Trial

Introduction: Pediatric AML accounts for approximately 20% of pediatric leukemias and represents a clinical challenge due to the poor prognosis for some patients (pts). AZA is a hypomethylating agent shown to slow leukemia progression; however, there are few data on the efficacy of AZA in children. Here we report results from the multicenter, open-label, phase 2 AZA-AML-004 study (NCT02450877) to determine the efficacy, safety, and PK of AZA in children and young adults with AML in molecular relapse after their first complete remission (CR1).