Improvement in Outcomes of Childhood Acute Myeloid Leukemia Treatment: A Survival Analysis at a Reference University...

Acute myeloid leukemia (AML) represents 15% to 20% of all pediatric acute leukemias and is responsible for nearly 30% of deaths in this population. While high-income countries report overall survival (OS) rates near 75%, survival in Brazil remains significantly lower, ranging between 30% and 40%.

The objective of this study is to analyze the probability of OS and event-free survival (EFS) in pediatric patients with AML treated at a Brazilian university hospital, evaluate prognostic factors, and compare outcomes with international data.

This retrospective study included 51 patients (under 14 years old) diagnosed between 2004 and 2023 and treated with BFM-based protocols. The median age was 4.6 years. Patients with acute promyelocytic leukemia (APL) achieved an OS of 100% and EFS of 90%. For other AML subtypes, the 5-year OS and EFS were 53.7% and 43.7%, respectively. Implementation of improved supportive care (ISC) after 2008-including the opening of a pediatric ICU and better infection management-was a pivotal prognostic factor (p < 0.001). Following ISC implementation, OS increased dramatically from 8.3% to 74.5%, and the early death rate dropped to 5.7%, reaching levels comparable to high-income countries.

Pediatric AML outcomes in developing regions can achieve international standards when intensive chemotherapy is combined with optimized supportive care. The establishment of specialized pediatric intensive care and advanced management of infectious complications are essential pillars for bridging the survival gap in these populations.