DOI: 10.3390/diagnostics16213463 ISSN: 2075-4418

Multispecimen Genomic Profiling Reveals Inter-Specimen Heterogeneity and Supports Ascites as a Complementary Biospecimen in Pseudomyxoma Peritonei

Shi Yan, Huanhuan Liu, Yiming Kang, Chong Wang, Pu Zhang, Haipeng Zhou, Zijie Huang, Guangjun Shi, Lubiao An, Huidan Zhang, Hongyu Zhao, Hongbin Xu, Ruiqing Ma

Background: Pseudomyxoma peritonei (PMP) is a rare peritoneal malignancy with limited genomic data across different biospecimen types. The extent to which ascites and plasma reflect tissue-detected genomic alterations remains incompletely characterized. This study compared genomic profiles across tumor tissue, ascites, and plasma and explored KRAS-defined molecular features in PMP. Methods: Sixty patients with pathologically confirmed PMP were retrospectively included. Next-generation sequencing (NGS) data were available from tumor tissue in 57 patients, ascites in 40, and plasma in 28, with overlapping specimen availability. Cross-specimen genomic concordance was evaluated in 37 patients with matched tissue–ascites samples, including 28 patients with matched tissue, ascites, and plasma. Exploratory analyses were performed to characterize genomic and pathway-level features across KRAS-defined subgroups. Results: In tumor tissue, KRAS (66.7%), GNAS (31.6%), and TP53 (21.1%) were the most frequently altered genes. Among 28 patients with matched tissue, ascites, and plasma, patient-level concordance was 35.7%. At the mutation level, ascites showed higher tissue-referenced positive percentage agreement (PPA) than plasma (93.1% vs. 36.2%). In the expanded cohort of 37 matched tissue–ascites pairs, ascites achieved a tissue-referenced PPA of 94.2% and revealed additional alterations not detected in the corresponding tissue samples. Exploratory KRAS mutation stratification identified G12D (35.0%), G12X (32.5%), and KRAS-wild-type (wt) (17.5%) tumors as the predominant subgroups. KRAS-mutant tumors harbored more detected genomic alterations and potentially clinically actionable alterations than KRAS-wt tumors. Wnt and PI3K pathway alterations were more commonly observed in the G12D and G12X subgroups, respectively, whereas canonical oncogenic pathway alterations were less frequent in KRAS-wt tumors. Conclusions: PMP exhibits substantial genomic heterogeneity across patients and biospecimen types. Ascites cfDNA showed high concordance with tissue and captured additional genomic alterations, suggesting that ascitic fluid may serve as a complementary source for molecular profiling in PMP. KRAS-defined subgroups exhibited distinct genomic characteristics, although their clinical relevance requires further validation in larger, well-characterized cohorts.

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