Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trial.

Background: Due to their anatomical locations, optic pathway gliomas (OPGs) can rarely be cured by resection. Given the importance of preserving visual function, we analyzed radiological and visual acuity (VA) outcomes for the type II RAF inhibitor tovorafenib in the OPG subgroup of the phase 2 FIREFLY-1 trial.

Methods: FIREFLY-1 investigated the efficacy (arm 1, n=77), safety, and tolerability (arms 1/2) of tovorafenib (420 mg/m2 once weekly; 600 mg maximum) in patients with BRAF-altered relapsed/refractory pediatric low-grade glioma (pLGG).

Comparative analysis of ischemic and hemorrhagic stroke hospitalization rates in end-stage kidney disease and kidney transplant patients with and without atrial fibrillation.

Introduction: Atrial fibrillation (AF) in end-stage kidney disease (ESKD) and kidney transplant (KTx) recipients presents challenges in stroke risk management. This study aimed to compare hospitalization rates for ischemic and hemorrhagic cerebrovascular events in ESKD and KTx patients with and without AF.

Methods: Using the National Inpatient Sample (2005-2019), retrospective analysis was conducted on hospitalizations for ESKD and KTx patients with and without AF.

Quality of life, healthcare usage and finances of UK cancer survivors five years post-diagnosis: a matched controlled study.

Purpose: Assessing the long-term impact of cancer on people's lives is challenging due to confounding issues such as aging and comorbidities. We aimed to investigate this impact by comparing the outcomes of cancer survivors with a matched control cohort.

Methods: This was a cross-sectional survey of breast, colorectal and ovarian cancer survivors approximately 5 years post-diagnosis and a cohort of age, sex and social deprivation-matched controls who had never had a cancer diagnosis.

Phase 2 Trial of Veliparib, Local Irradiation and Temozolomide in Patients with Newly Diagnosed High-Grade Glioma: A Children's Oncology Group Study.

Background: The outcome for pediatric patients with high-grade glioma (HGG) remains poor. Veliparib, a potent oral poly(adenosine diphosphate-ribose) polymerase (PARP) 1/2 inhibitor, enhances the activity of radiotherapy and DNA-damaging chemotherapy.

Methods: We conducted a single-arm, non-randomized phase 2 clinical trial to determine whether treatment with veliparib and radiotherapy, followed by veliparib and temozolomide, improves progression-free survival in pediatric patients with newly diagnosed HGG without H3 K27M or BRAF mutations compared to patient level data from historical cohorts with closely matching clinical and molecular features.

Synaptic gene expression changes in frontotemporal dementia due to the MAPT 10 + 16 mutation.

Aims: Mutations in the MAPT gene encoding tau protein can cause autosomal dominant neurodegenerative tauopathies including frontotemporal dementia (often with Parkinsonism). In Alzheimer's disease, the most common tauopathy, synapse loss is the strongest pathological correlate of cognitive decline. Recently, Positron Emission Tomography (PET) imaging with synaptic tracers revealed clinically relevant loss of synapses in primary tauopathies; however, the molecular mechanisms leading to synapse degeneration in primary tauopathies remain largely unknown.