Acute myeloid leukemia (AML) with internal tandem duplication in FMS-like tyrosine kinase 3 () is associated with poor outcomes. This study aimed to analyze the outcomes of pediatric AML patients with mutations in the pre-FLT3 inhibitor era.
We retrospectively reviewed and identified 18 patients diagnosed with non-M3 AML with mutations at Seoul National University Children’s Hospital between May 2008 and August 2019.
The median age was 13 years (range, 6-19 yr). The median follow-up time was 43 months (range, 6-157 mo). Fourteen patients received BH-AC-based (N4-Behenoy1-1-β-Darabinofuranosy1cytosine) and 4 received cytarabine-based induction chemotherapy. Complete remission (CR) was achieved in 72.2% of the patients after the first induction chemotherapy and 80% of the patients achieved CR after salvage therapy. The overall CR rate was 94% (17/18 patients). These 17 patients underwent hematopoietic stem cell transplantation (9 matched unrelated donors, 5 matched related donors, and 3 haploidentical donors). Relapse occurred in 22% of the patients. Event free survival and overall survival rates were 53.8±12.1% and 53.6±12.1%, respectively, and they were not significantly different according to the type of induction chemotherapy (=0.690) or the type of donor (=0.102).
This study outlines the outcomes of pediatric AML patients with -mutations in one institution over a decade. Outcomes were significantly improved in this study compared to our previous report in 2004, where RFS and EFS were 0%. This study can provide baseline data for pediatric patients in the pre-FLT3 inhibitor era.