AAML1331 — A Phase III Study for Patients With Newly Diagnosed Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide and All-Trans Retinoic Acid (Children's Oncology Group AAML1331)

Phase III · 2021 · Acute Promyelocytic Leukemia

Design

Nonrandomized, noninferiority phase III trial evaluating a largely chemotherapy-free all-trans retinoic acid plus arsenic trioxide regimen against a historical control (AAML0631), with idarubicin added only during induction for high-risk patients and no maintenance therapy.

Population

Patients aged 1 to 21 years with newly diagnosed acute promyelocytic leukemia, stratified into standard-risk (WBC < 10,000/μL) and high-risk (WBC ≥ 10,000/μL) cohorts.

Primary Endpoint

2-year event-free survival

Key Result

Standard-risk patients achieved a 2-year event-free survival of 98.0% and overall survival of 99.0% with a chemotherapy-free ATRA plus arsenic trioxide regimen, with outcomes noninferior to the historical comparator.

Guideline Impact

Established chemotherapy-free ATRA/arsenic trioxide as standard for pediatric standard-risk APL