Children with supratentorial midline pilocytic astrocytomas exhibit multiple progressions and acquisition of neurologic deficits over time

Abstract

Summary Tumor location has been proposed as a prognostic factor for pilocytic astrocytoma (PA), but since resection status varies by CNS location, these two variables are difficult to separate on multivariate analysis. To eliminate resection status as a confounding variable, we analyzed the outcomes of children with subtotally resected PA by brain location. We found that individuals with PA in the supratentorial midline region had an increased likelihood of multiple progression events. These children also exhibited more neurologic deficits over time compared to those with brainstem PA, frequently due to worsening vision and the acquisition of new endocrinopathies or weakness.

Everolimus for Children With Recurrent or Progressive Low-Grade Glioma: Results From the Phase II PNOC001 Trial

“…We found these breakpoints enriched in young patients (younger than 3 years) and patients with a supratentorial midline PA. These factors are known to be associated with poorer prognosis, [6][7][8][9][10][11]24 which suggests that rare/novel KIAA1549::BRAF fusion breakpoints provide a novel genetic biomarker for high-risk pLGG. We recommend routine assessment of KIAA1549::BRAF fusion breakpoints in future trials to confirm this association and move toward integrated molecular diagnosis for optimal clinical decision making.…”

“…[18][19][20] Previous trials have found that the mTOR inhibitor everolimus is safe and efficacious for recurrent adult LGGs and pLGGs. [21][22][23] However, biomarkers predicting everolimus response in pLGG remain uncertain, and several factors, such as young age at presentation, 6 supratentorial midline tumor location, 24 and rare KIAA1549::BRAF fusion breakpoints, 17 contribute to worse outcomes. A subset of cases are associated with genetic tumor syndromes, yet with unclear frequency or prognostic relevance.…”