Defining a Distinct Subtype of TP53-Mutated AML via BAP1 Loss: Implications for Targeted Therapy with BCL-xL Inhibitors

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This article reviews recent evidence identifying BAP1 loss as a defining feature of a unique TP53-mutated subtype of de novo acute myeloid leukemia (AML), notably associated with erythroid differentiation bias and worse survival outcomes. Mechanistic insights reveal cooperation between BAP1 and TP53 loss in driving transplantable erythroleukemia through genomic instability and epigenetic regulation. Importantly, this subtype shows dependency on BCL2L1 (BCL-xL) expression and exhibits in vivo sensitivity to BCL-xL inhibitors, presenting a novel targeted therapeutic opportunity.

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