Cancer predisposition syndromes are recognized in about 10% of pediatric malignancies with several genes specifically involved in a subset of pediatric tumors such as DICER1, in pleuropulmonary blastoma, cystic nephroma, and brain sarcomas. By contrast, the role of BRCA1/2 in pediatric cancer predisposition is still under investigation. We present two cases of young first-degree cousins, both carrying a germline BRCA2 variant and developing tumors characterized by somatic DICER1 mutations. Patient 1 presented with a cystic nephroma harboring a somatic DICER1 variant (p.Asp1810Tyr), while patient 2 had a primary intracranial DICER1-mutated sarcoma showing a distinct somatic DICER1 variant (p.Asp1709Glu) as well as biallelic inactivation of TP53 (p.Val173Leu, VAF 91%) and APC (p.Ile1307Lys, VAF 95%) and a pathogenic variant in KRAS (p.Gln61His). Both patients carried the same germline BRCA2 variant (p.Arg2842Cys) of unknown significance. The same variant was found in the mother of patient 2 and in the father of patient 1, who are siblings. A homologous recombination deficiency signature was not identified in any of the two tumors, possibly suggesting a reduction of BRCA2 activity. The association of BRCA2 and DICER1 variants in our cases hints at a potential cooperative role in cancer pathogenesis. Further studies are warranted to elucidate the interplay between BRCA1/2 and DICER1 variants and their implications for cancer predisposition and treatment in pediatric patients.

The coexistence of a BRCA2 germline and a DICER1 somatic variant in two first-degree cousins suggests their potential synergic effect / DEL BALDO, Giada; Mastronuzzi, Angela; Cipri, Selene; Agolini, Emanuele; Matraxia, Marta; Novelli, Antonio; Cacchione, Antonella; Serra, Annalisa; Carai, Andrea; Boccuto, Luigi; Stefania Colafati, Giovanna; DI PAOLO, PIER LUIGI; Miele, Evelina; Barresi, Sabina; Alaggio, Rita; Rossi, Sabrina; Giovannoni, Isabella. - In: SCIENTIFIC REPORTS. - ISSN 2045-2322. - 14:(2024).

The coexistence of a BRCA2 germline and a DICER1 somatic variant in two first-degree cousins suggests their potential synergic effect

Giada Del Baldo;Angela Mastronuzzi
;
Emanuele Agolini;Antonio Novelli;Antonella Cacchione;Pier Luigi Di Paolo;Evelina Miele;Rita Alaggio
;
Isabella Giovannoni
2024

Abstract

Cancer predisposition syndromes are recognized in about 10% of pediatric malignancies with several genes specifically involved in a subset of pediatric tumors such as DICER1, in pleuropulmonary blastoma, cystic nephroma, and brain sarcomas. By contrast, the role of BRCA1/2 in pediatric cancer predisposition is still under investigation. We present two cases of young first-degree cousins, both carrying a germline BRCA2 variant and developing tumors characterized by somatic DICER1 mutations. Patient 1 presented with a cystic nephroma harboring a somatic DICER1 variant (p.Asp1810Tyr), while patient 2 had a primary intracranial DICER1-mutated sarcoma showing a distinct somatic DICER1 variant (p.Asp1709Glu) as well as biallelic inactivation of TP53 (p.Val173Leu, VAF 91%) and APC (p.Ile1307Lys, VAF 95%) and a pathogenic variant in KRAS (p.Gln61His). Both patients carried the same germline BRCA2 variant (p.Arg2842Cys) of unknown significance. The same variant was found in the mother of patient 2 and in the father of patient 1, who are siblings. A homologous recombination deficiency signature was not identified in any of the two tumors, possibly suggesting a reduction of BRCA2 activity. The association of BRCA2 and DICER1 variants in our cases hints at a potential cooperative role in cancer pathogenesis. Further studies are warranted to elucidate the interplay between BRCA1/2 and DICER1 variants and their implications for cancer predisposition and treatment in pediatric patients.
2024
BRCA genes; DICER1; cancer predisposition syndrome; cystic nephroma; pediatric oncology; primary intracranial DICER1-mutated sarcoma
01 Pubblicazione su rivista::01a Articolo in rivista
The coexistence of a BRCA2 germline and a DICER1 somatic variant in two first-degree cousins suggests their potential synergic effect / DEL BALDO, Giada; Mastronuzzi, Angela; Cipri, Selene; Agolini, Emanuele; Matraxia, Marta; Novelli, Antonio; Cacchione, Antonella; Serra, Annalisa; Carai, Andrea; Boccuto, Luigi; Stefania Colafati, Giovanna; DI PAOLO, PIER LUIGI; Miele, Evelina; Barresi, Sabina; Alaggio, Rita; Rossi, Sabrina; Giovannoni, Isabella. - In: SCIENTIFIC REPORTS. - ISSN 2045-2322. - 14:(2024).
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1726662
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