An exploratory analysis of the phase 2 FIREFLY-1 trial (NCT04775485) revealed that tovorafenib (Ojemda) improved the health-related quality of life (HRQOL) in pediatric patients with BRAF-altered relapsed/refractory low-grade glioma. The findings, presented at the 2024 Society for Neuro-Oncology Annual Meeting, highlight improvements in multiple domains, including anxiety and cognition.
Improved Quality of Life
Jordan R. Hansford, MD, lead of pediatric neuro-oncology at the Women’s and Children’s Hospital, reported that HRQOL generally remained stable or improved for most patients during the first year of treatment. The exploratory analysis utilized the PedsQL 3.0 Cancer Module through parent and/or self-reports to evaluate HRQOL in patients treated with tovorafenib.
"Our total scores are improved in multiple domains, including anxiety, cognition, and other features," Dr. Hansford stated. "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."
Limitations and Future Directions
Despite the encouraging results, Dr. Hansford noted that long-term HRQOL analysis was limited by reduced completion of reports from parents and patients as the study progressed. The phase 2 nature of the trial provides valuable insights into the short-term benefits of tovorafenib, but the long-term impact 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. Future studies will need to explore these aspects to assess the sustained impact of tovorafenib on overall wellbeing.
