TREATMENT OUTCOME AND CHALLENGES OF PEDIATRIC AML IN A LMIC: A SINGLE- INSTITUTION EXPERIENCE

Abstract

Background: Despite improvements in pediatric acute myeloid leukemia (AML) outcomes, results remain poor in low- and middle-income countries (LMICs).

Objectives: We aimed to evaluate the survival outcome of pediatric AML patients, with special emphasis on various prognostic factors.

Methods: This prospective study included all newly diagnosed pediatric AML patients (<18 years) treated at the National Cancer Institute, Egypt (2020–2022) using a protocol adapted from MyeChild 01.

Results: 100 patients were enrolled. By the end of follow-up, sustained remission was achieved in 34 cases while 28 cases relapsed or were refractory, and 38 died during supportive care. The 1-year and 2-year overall survival (OS) rates were 51% and 38% while the 1-year and 2-year event-free survival (EFS) rates were 44% and 33%, respectively.  Patients <2 years of age and those with KMT2A-rearrangements showed worse EFS (P=0.026 and P=0.049, respectively).  WBC count at diagnosis <100×10⁹/L and Core-binding factor leukemias had been associated with better OS (P=0.031 and P=0.032, respectively) and EFS (P=0.021 and P=0.020, respectively). Post-induction remission and being classified in the standard-risk group were both significantly associated with prolonged OS and EFS compared to patients who were refractory (p< 0.001,each), as well as those in the high-risk group (p< 0.001,each). High-risk patients undergoing allogeneic HSCT had better OS and EFS (P=0.001, each). Morphological response to induction was identified as independent prognostic factor (P< 0.001).

Conclusion: Treatment-related mortality and high relapse rate remain major barriers to improve outcomes in LMICs.