Improved Outcome in Pediatric Acute Myeloid Leukemia: Progress With Hematopoietic Cell Transplantation

Pediatric acute myeloid leukemia (AML) is a heterogenous disease; however, development of diagnostic methods has led to a better understanding of its nature and improvement in therapy outcomes. In this study, we evaluated treatment results in children with AML treated in a single oncology center in comparison with international results.

Data from 77 children with AML treated in a single oncology center between 1994 and 2020 were analyzed. Patients had been treated according to five consecutive therapy protocols: AML NOPHO 88; ANLL 98; BFM AML 2004; BFM AML 2012, and BFM AML 2019. Five-year overall survival (OS), 5-year event-free survival (EFS) and 5-year relapse-free survival (RFS) were evaluated for each therapy protocol to determine prognostic factors and clarify differences between international and individual center results.

During the observational period, 5-year OS increased from 55.6% to 85.7%, 5-year EFS from 45.7% to 87.5% and 5-year RFS from 51.4% to 85.7%. Hematopoietic cells transplantation (HCT) introduction in early 2000' improved treatment outcomes in the high-risk group, what has been mostly seen in the results of 5-year EFS. Treatment-related mortality was the most frequent cause of death in the analyzed group.

Despite the significant improvement in therapy of pediatric AML, treatment outcomes remain unsatisfying. Introduction of HCT relevantly improved therapy results, especially in the high-risk group. International cooperation is crucial because of the small patient numbers in individual oncology centers.