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Review
. 2025 Jun;114(6):924-937.
doi: 10.1111/ejh.14404. Epub 2025 Mar 4.

TP53-Mutated Acute Myeloid Leukemia: Review of Treatment and Challenges

Bipin Ghimire  1 , Markie Zimmer  1 , Vijayalakshmi Donthireddy  1
Affiliations
Review

TP53-Mutated Acute Myeloid Leukemia: Review of Treatment and Challenges

Bipin Ghimire et al. Eur J Haematol. 2025 Jun.

Abstract

Patients with acute myeloid leukemia (AML) harboring mutations in TP53 (TP53-MT) have poor responses to current therapies and unfavorable prognoses. Despite the recognition of variant TP53 as an adverse feature of AML, an optimal treatment regimen has not yet been established, underlining a critical need for new, more effective therapeutic combinations and novel treatments. We present the case of a patient with TP53-MT AML and marked myelodysplasia who developed primary refractory disease after induction therapy with the intensive chemotherapy regimen of liposomal daunorubicin and cytarabine. Our patient's optimal response to second induction chemotherapy with FLAG-Ida prompted an exploration of established and investigational treatment regimens for this specific high-risk AML subtype. Therefore, we performed a comprehensive literature review of findings from studies exploring AML therapies, focusing on outcomes for patients with TP53-MT AML. The summary provided here reveals the complexity of defining the therapeutic responses of patients with the heterogeneous TP53-MT genetic background and the challenges in treating this high-risk form of AML. Future work must continue to investigate novel therapies and combinations to improve patient outcomes in this vulnerable population.

Keywords: TP53; acute myeloid leukemia; myelodysplasia; p53.

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