XAF1 as a modifier of p53 function and cancer susceptibility.

Pinto, Emilia M; Figueiredo, Bonald C; Chen, Wenan; Galvao, Henrique C R; Formiga, Maria Nirvana; Fragoso, Maria Candida B V; Ashton-Prolla, Patricia; Ribeiro, Enilze M S F et al. · Sci Adv · 2020

case_control · Level III

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Abstract

Cancer risk is highly variable in carriers of the common <i>TP53-</i>R337H founder allele, possibly due to the influence of modifier genes. Whole-genome sequencing identified a variant in the tumor suppressor <i>XAF1</i> (E134*/Glu134Ter/rs146752602) in a subset of R337H carriers. Haplotype-defining variants were verified in 203 patients with cancer, 582 relatives, and 42,438 newborns. The compound mutant haplotype was enriched in patients with cancer, conferring risk for sarcoma (<i>P</i> = 0.003) and subsequent malignancies (<i>P</i> = 0.006). Functional analyses demonstrated that wild-type XAF1 enhances transactivation of wild-type and hypomorphic <i>TP53</i> variants, whereas <i>XAF1</i>-E134* is markedly attenuated in this activity. We propose that cosegregation of <i>XAF1-</i>E134* and <i>TP53-</i>R337H mutations leads to a more aggressive cancer phenotype than <i>TP53-</i>R337H alone, with implications for genetic counseling and clinical management of hypomorphic <i>TP53</i> mutant carriers.