Clonal dynamics of aggressive systemic mastocytosis on avapritinib therapy.

This document examines the clonal dynamics of advanced systemic mastocytosis (AdvSM) and associated acute myeloid leukemia (ASM-AHN) in patients receiving avapritinib therapy. The study utilized single-cell RNA sequencing and whole-genome sequencing to analyze genetic and cellular changes before and after treatment. The findings indicate that avapritinib targets different subclones and cellular compartments based on the position of the KITD816V driver mutation. The study suggests that a combination of avapritinib and hypomethylating agents may be effective in addressing the entire malignant network and promoting normal hematopoiesis in AdvSM. [Extracted from the article]