Diagnostic Uptake of Targeted Sequencing in Adults With Steatotic Liver Disease and a Suspected Genetic Contribution.

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Tác giả: Cristiana Bianco, Hadi Eidgah Torghabehei, Maria Rosaria De Filippo, Oveis Jamialahmadi, Francesco Malvestiti, Vittoria Moretti, Serena Pelusi, Giulia Periti, Daniele Prati, Stefano Romeo, Jessica Rondena, Luisa Ronzoni, Luca Valenti

Ngôn ngữ: eng

Ký hiệu phân loại: 220.5203 Modern versions and translations

Thông tin xuất bản: United States : Liver international : official journal of the International Association for the Study of the Liver , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 80876

BACKGROUND AND AIMS: In patients with steatotic liver diseases (SLD), genetic factors may account for severe liver involvement despite mild or absence of triggering factors or a strong family history. Aim of this study was to examine the diagnostic uptake of targeted sequencing (TS), covering both coding and non-coding regions, of a broad panel of 82 liver and lipid metabolism genes in patients with unexplained SLD. METHODS: We enrolled 49 adult patients with SLD and a suspected genetic contribution. Genetic variants were detected through a customised TS panel, whereas the contribution of common genetic variation to the individual susceptibility to SLD was captured by a polygenic risk score (SLD-PRS). RESULTS: A diagnosis of rare Mendelian disorder was established in 11 patients (22%), independently of age or family history. Rare variants possibly contributing to clinical phenotype were detected in additional 29 patients (59%). Increased SLD-PRS values were detected in 17 patients (35%), enabling an increase in diagnostic uptake of 24%, especially in those without a strong family history (p = 0.03). Genetic diagnosis allowed refinement of clinical management in 23 (47%) patients. CONCLUSIONS: The diagnostic uptake of TS was 22% for Mendelian disorder and 59% for possible contribution to clinical phenotype in selected adult patients with SLD. Evaluation of common variants, as captured by SLD-PRS, yields complementary information increasing the overall utility of the genetic examination.
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