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

Background/aim: 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.

Patients And Methods: 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.

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.

Conclusion: 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.