Germline Genetic Testing in Patients with Bone and Soft Tissue Sarcoma: A Prospective Multicenter Study to Evaluate Cancer Susceptibility

Int J Mol Sci. 2025 Mar 21;26(7):2839. doi: 10.3390/ijms26072839.

Abstract

Sarcomas are rare heterogenous mesenchymal tumors with over seventy-five different subtypes, with varying biology and outcomes, with no clear inciting factor in the vast majority. To determine the prevalence of pathogenic germline variants (PGV) in patients with sarcomas, we undertook a prospective multi-site study of germline sequencing using an 84-gene next-generation sequencing panel among patients receiving care at the four Mayo Clinic Cancer Centers. Of 115 patients with bone and soft tissue sarcoma, the median age was 60 years, 49.6% were female, 82.6% were White. The anatomical location of the primary tumor included extremities (34.8%), retroperitoneum (19.1%), trunk (13.0%), and head and neck (7.8%). Family history of cancer was present in 62.6% of the study population. Ten patients (8.7%) had a pathogenic/likely pathogenic variant (PGV). Of these, three had stage IV sarcoma, and seven had earlier-stage sarcoma (stages I-III). Among the 55 (48.7%) patients who had variant of uncertain significance (VUS), 41.1% (22/55) had stage IV sarcoma and 58.9% (33/55) had earlier-stage disease. Of the ten patients with PGV, high-to-moderate penetrance gene abnormalities were identified in eight patients (80%) involving TP53 (3), BRCA1 (1), SDHA (1), ATM (2), and NBN (1) genes. The vast majority of the PGVs (70%) would not have been detected using the current guidelines. Because of the paucity of sarcomas and lack of effective treatment options for advanced disease, germline testing in sarcomas represents a potentially impactful strategy to assess therapeutic options and for assessment of familial risk.

Keywords: genetic testing; germline testing; homologous recombination deficiency; sarcoma.

Publication types

  • Multicenter Study

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Bone Neoplasms* / genetics
  • Bone Neoplasms* / pathology
  • Female
  • Genetic Predisposition to Disease*
  • Genetic Testing* / methods
  • Germ-Line Mutation*
  • High-Throughput Nucleotide Sequencing
  • Humans
  • Male
  • Middle Aged
  • Prospective Studies
  • Sarcoma* / genetics
  • Sarcoma* / pathology
  • Soft Tissue Neoplasms* / genetics
  • Young Adult

Grants and funding

The study was funded by Mayo Transform the Practice Grant, Mayo Clinic Center for Individualized Medicine, Desert Mountain Members’ CARE Foundation, David and Twila Woods Foundation and a Faculty Career Development Award from the Gerstner Foundation (NJS). The funding sources did not play a role in the design, conduct, or reporting of the study or in the decision to submit the manuscript for publication.