Dr Hansford on HRQOL Outcomes With Tovorafenib in Pediatric BRAF-Altered Low-Grade Glioma

Our total scores are improved in multiple domains, including anxiety, cognition, and other features. Not only are we seeing great efficacy, which was presented previously, but diving into the QOL on this study is also showing the patients are feeling improved QOL with this new agent, as well."

Jordan R. Hansford, MD, lead, pediatric neuro-oncology, the Women’s and Children’s Hospital; lead, Pediatric Neuro-Oncology Group, the South Australia Health and Medical Research Institute; the South Australia ImmunoGenomics Cancer Institute, the University of Adelaide, discusses health-related quality-of-life (HRQOL) outcomes with the type II RAF inhibitor tovorafenib (Ojemda) in pediatric patients with BRAF-altered relapsed/refractory low-grade glioma from the phase 2 FIREFLY-1 trial (NCT04775485).

An exploratory analysis of the trial assessed HRQOL in patients with recurrent/refractory BRAF-altered pediatric low-grade glioma treated with tovorafenib. HRQOL was evaluated using the PedsQL 3.0 Cancer Module through parent and/or self reports for patients in arm 1 of the study.

Findings presented during the 2024 Society for Neuro-Oncology Annual Meeting showed that HRQOL generally remained stable or improved for most patients during the first year of treatment, Hansford reports. Improvements were noted across multiple domains, including anxiety and cognition, he adds. These findings complement previously reported efficacy data, emphasizing that tovorafenib not only provides clinical benefit but also enhances patients’ QOL, Hansford says.

Despite these encouraging results, long-term HRQOL analysis was limited by reduced completion of reports from parents and patients as the study progressed, Hansford notes. Although the phase 2 nature of the trial offers valuable insights into the short-term benefits of tovorafenib, the long-term impact of this therapy on HRQOL remains an important unanswered question. Given the potential for extended survival in pediatric patients, understanding how treatment affects their QOL over several years or after discontinuation is critical, Hansford emphasizes. Future studies will need to explore these aspects to assess the sustained impact of tovorafenib on overall wellbeing, he concludes.