Characterization of somatic mutations in sporadic uveal melanoma and uveal melanoma in patients with germline BAP1 pathogenic variants

PLoS One. 2024 Oct 8;19(10):e0306386. doi: 10.1371/journal.pone.0306386. eCollection 2024.

Abstract

Genetic analyses were conducted on tumor samples from 88 patients with uveal melanoma (UM), 6 of whom carry pathogenic germline variants in BAP1. We assessed the frequency, pattern, and prognostic significance of somatic aberrations, and investigated differences between germline BAP1 variant carriers compared to sporadic cases. The frequency of the main oncogenic driver mutations was not significantly different between these groups. Patients with germline BAP1 variants did not have significantly different overall survival compared to the wildtype or somatic BAP1 mutation groups. Patients with a somatic BAP1 mutation (n = 24) had a significantly worse prognosis compared to wildtype (n = 58). All patients with stage III tumors and a somatic BAP1 mutation (n = 7) developed metastasis, however four of 28 stage I-II tumors without metastasis had somatic BAP1 mutations, with observation time >5 years. The tumor from one germline BAP1 carrier (stage IIIC) with a somatic EIF1AX splice variant, has not developed metastasis within a 22-year observation time.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Female
  • Germ-Line Mutation*
  • Humans
  • Male
  • Melanoma* / genetics
  • Melanoma* / pathology
  • Middle Aged
  • Prognosis
  • Tumor Suppressor Proteins* / genetics
  • Ubiquitin Thiolesterase* / genetics
  • Uveal Neoplasms* / genetics
  • Uveal Neoplasms* / pathology

Substances

  • Ubiquitin Thiolesterase
  • BAP1 protein, human
  • Tumor Suppressor Proteins

Supplementary concepts

  • Uveal melanoma

Grants and funding

KW: Funded by Fight for Sight NH: Funded by the National Health and Medical Research Council The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.