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Using VAF and cytogenetics to predict PFS in TP53-mutated AML/MDS patients

Lontos K, Saliba RM, Kanagal-Shamanna R, et al. TP53 Mutant Variant Allele Frequency and Cytogenetics Determine Prognostic Groups in MDS/AML for Transplantation. Blood Advances. 2025; (doi: 10.1182/bloodadvances.2024014499).

Patients who have myeloid malignancies with TP53 mutations often fare poorly after hematopoietic stem cell transplantation (HSCT), but researchers say certain prognostic factors can help identify those with the best prospects for progression-free survival (PFS). Their study included 240 patients with TP53-mutated myelodysplastic syndrome or acute myeloid leukemia who underwent HSCT. Upon analysis, investigators noted several factors that correlated to favorable PFS outcomes — the most impactful of which were lower variant allele frequency (VAF) and the absence of complex cytogenetics/5q deletion/7q deletion. They calculated 2-year PFS for patients with TP53-mutated disease at 3% for those with VAF ≥ 50%; at 22% for those with VAF < 50% but with complex/5q/7q cytogenetic abnormalities; and at 60% for those with VAF < 50% and no complex/5q/7q cytogenetics. The study authors say these barometers of PFS can help select patients with TP53 mutations who are most likely to benefit from HSCT, instead of assuming that everyone in this sub-population is unsuitable for transplantation.

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