Study Design

AAML0531 supports the addition of MYLOTARG to standard chemotherapy in pediatric patients with newly diagnosed CD33-positive AML1
The AAML0531 trial was a multicenter, randomized study of 1063 patients with newly diagnosed AML (ages 0-29 years)
  • Patients were randomized to 5-cycle chemotherapy alone (n=531) or 5-cycle chemotherapy plus MYLOTARG 3 mg/m2/dose (n=532) administered once on Day 6 in Induction 1 and once on Day 7 in Intensification 2
  • Primary endpoint was EFS
  • All patients proceeded to Induction 2 regardless of post-Induction 1 remission status
  • In the absence of active disease, ANC >1 Gi/L and platelets >75 Gi/L were recommended before proceeding with subsequent therapy cycles
  • Patients not in remission after Induction 2 discontinued therapy permanently; all others proceeded to Intensification 1
  • Patients with high- and intermediate-risk disease with 5/6 or 6/6 matched family donors (MFD) proceeded to HSCT following Intensification 1
  • Patients with high-risk disease proceeded to HSCT with an alternative donor if no MFD was available
  • All patients with low-risk disease and any high- and intermediate-risk patients without appropriate donors proceeded with Intensification 2 with or without MYLOTARG according to their initial randomization, followed by Intensification 3
  • All patients in remission were to proceed to Intensification 2 or allogeneic HSCT
  • In Intensification 2, patients received MYLOTARG according to their initial randomization
  • Patients in remission after Intensification 2 proceeded to Intensification 3
Select baseline patient characteristics in AAML0531

AAML0531 included newly diagnosed pediatric patients in the following age groups in the MYLOTARG plus chemotherapy arm:

  • 2 patients <27 days old
  • 94 patients ≥28 days to <2 years old
  • 225 patients ≥2 years to <12 years old
  • 175 patients ≥12 years to <18 years old
  • 36 patients ≥18 years old

In the overall patient population (n=1063), the patients were:

  • 49% male, 51% female
  • 73% White, 18% Hispanic, 11% Black, 5% Asian, and 11% other or missing race

The proportion of patients in each disease risk group was:

  • Low risk (23% MYLOTARG plus chemotherapy arm vs 23% chemotherapy alone arm)
  • Intermediate risk (57% MYLOTARG plus chemotherapy arm vs 57% chemotherapy alone arm)
  • High risk (15% MYLOTARG plus chemotherapy arm vs 17% chemotherapy alone arm)
AML=acute myeloid leukemia; ANC=absolute neutrophil count; EFS=event-free survival; HSCT=hematopoietic stem cell transplantation.
1. MYLOTARG Prescribing Information. New York, NY: Pfizer Inc.