Dissecting the prognostic signature of patients with astrocytoma isocitrate dehydrogenase-mutant grade 4: a large multicenter, retrospective study

ESMO Open. 2024 Jun;9(6):103485. doi: 10.1016/j.esmoop.2024.103485. Epub 2024 Jun 3.

Abstract

Background: The World Health Organization (WHO) 2021 classification of central nervous system (CNS) tumors classified astrocytoma isocitrate dehydrogenase-mutant (A IDHm) with either microvascular proliferation and/or necrosis or homozygous deletion of CDKN2A/B as CNS grade 4 (CNS WHO G4), introducing a distinct entity and posing new challenges to physicians for appropriate management and prognostication.

Patients and methods: We retrospectively collected information about patients diagnosed with A IDHm CNS WHO G4 at three reference neuro-oncological Italian centers and correlated them with survival.

Results: A total of 133 patients were included. Patients were young (median age 41 years) and most received post-operative treatment including chemo-radiation (n = 101) and/or temozolomide maintenance (n = 112). With a median follow-up of 51 months, the median overall survival (mOS) was 31.2 months, with a 5-year survival probability of 26%. In the univariate analysis, complete resection (mOS: 40.2 versus 26.3 months, P = 0.03), methyl-guaninemethyltransferase (MGMT) promoter methylation (mOS: 40.7 versus 18 months, P = 0.0136), and absence of telomerase reverse transcriptase (TERT) promoter mutation (mOS: 40.7 versus 18 months, P = 0.0003) correlated with better prognosis. In the multivariate models, lack of TERT promoter mutation [hazard ratio (HR) 0.23, 95% confidence interval (CI) 0.07-0.82, P = 0.024] and MGMT methylation (HR 0.40, 95% CI 0.20-0.81, P = 0.01) remained associated with improved survival.

Conclusions: This is the largest experience in Western countries exploring the prognostic signature of patients with A IDHm CNS G4. Our results show that MGMT promoter methylation and TERT promoter mutation may impact clinical outcomes. This may support physicians in prognostication, clinical management, and design of future studies of this distinct diagnostic entity.

Keywords: IDH mutation; adult-type diffuse gliomas; astrocytoma IDH-mutant WHO G4.

Publication types

  • Multicenter Study

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Astrocytoma* / genetics
  • Astrocytoma* / mortality
  • Astrocytoma* / therapy
  • Brain Neoplasms / genetics
  • DNA Methylation
  • DNA Modification Methylases / genetics
  • DNA Repair Enzymes / genetics
  • Female
  • Humans
  • Isocitrate Dehydrogenase* / genetics
  • Male
  • Middle Aged
  • Mutation*
  • Neoplasm Grading
  • Prognosis
  • Retrospective Studies
  • Telomerase / genetics
  • Tumor Suppressor Proteins / genetics
  • Young Adult

Substances

  • Isocitrate Dehydrogenase
  • DNA Repair Enzymes
  • DNA Modification Methylases
  • Telomerase
  • MGMT protein, human
  • TERT protein, human
  • Tumor Suppressor Proteins