Venetoclax-based low-intensity therapy in pediatric AML: A viable option for chemotherapy-intolerant patients

Childhood acute myeloid leukemia (AML) comprises 15-20% of pediatric leukemias and carries promising survival rates (60-70%) in high-income countries with use of intensive chemotherapy. This is associated with high treatment-related mortality in low- and middle-income countries, especially during induction, due to malnutrition, infections, and insufficient resources. Venetoclax, a BCL-2 inhibitor effective in older adults unfit for standard therapy, has shown potential in pediatric relapse AML. Here, we describe two children with newly diagnosed AML who developed life-threatening toxicities with intensive chemotherapy and were subsequently treated with venetoclax-based low-intensity regimens. Both patients achieved complete remission, attained measurable residual disease negativity, and maintained durable responses with minimal toxicity, allowing largely outpatient management. These cases suggest a promising, feasible alternative for chemotherapy-intolerant patients.