Prevalence and Prognostic Impact of NPM1 Mutation in Childhood Acute Myeloid Leukemia: Experience from a Single Tertiary Cancer Centre in India.
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BACKGROUND AND AIMS: Nucleophosmin (NPM1) mutations are associated with favorable outcomes in adults; however, their prognostic relevance in pediatric acute myeloid leukemia (AML) remains unclear due to their rarity. This single center study from India, investigates the prevalence and prognostic impact of NPM1-mutated childhood AML. METHODS: Patients less than 15 years of age with newly diagnosed AML treated between January 2016 and December 2023 were included. NPM1 and FLT3 mutations were detected via multiplex PCR or next generation sequencing (NGS). Treatment consisted of standard 3 + 7 induction followed by 3 cycles of high-dose cytarabine and 1 year of oral maintenance. FLT3-ITD positive patients also received sorafenib or midostaurin. RESULTS: Of the 552 patients enrolled, 35 (6.3%) had NPM1 mutation. Patients with NPM1- mutations had higher median age (9 vs. 8 years; P = .05) and a greater frequency of FLT3/ITD co-mutations (40% vs. 11%; P < .001). Among the 32 NPM1-mutated patients who underwent NGS, type A (59%) was the most common. While complete remission rates and measurable residual disease negativity were comparable, NPM1-mutated patients demonstrated significantly better 3-year event-free survival (52.1% vs. 26%; P = .001) and overall survival (56.3% vs. 27.8%; P = .004). Although FLT3-ITD status was independently prognostic, it did not influence survival within the NPM1-mutated subgroup. Patients with a VAF < 39% had a better 3-year EFS (73.5% vs. 40.4%; P = .35). CONCLUSIONS: Our study confirms NPM1 mutations independently predict improved survival in pediatric AML, though outcomes remain inferior to those reported from high income countries. Larger studies are needed in pediatric AML due to its rare occurrence.