ID #462 Health-related quality-of-life in patients with pediatric low-grade glioma : Findings from the initial phase of the TRAM-01 Study
Emma Clini, Sarah Lippé, Léandra Desjardins, Serge Sultan, Edith Cantin, Marie-Eve Routhier, Valérie Larouche, Nada Jabado, Sébastien Perreault, Maxime CaruAbstract
Background
Patients with pediatric low-grade gliomas (PLGG) are living longer at the expense of a compromised health-related quality of life (HRQoL). These patients frequently report poorer physical and psychosocial health compared with their healthy peers, and parent proxy reports similarly indicate reduced HRQoL in this population. As such, HRQoL is recognized as a critical survivorship outcome, but it remains insufficiently explored in phase II clinical trials. To address this gap, the TRAM-01 study prospectively evaluates the impact of trametinib on HRQoL in patients with PLGG.
Methods
Patients (N = 114) completed HRQOL assessments in the pre-treatment phase of the TRAM-01 study using the PedsQL generic core scales and brain tumor module. Child self-reports were obtained for patients aged ≥5 years, and parent proxy-reports for children aged ≥2 years. HRQOL was examined across four clinical groups: (1) NF1-associated progressive or refractory glioma; (2) NF1-associated plexiform neurofibroma; (3) progressive or refractory glioma with KIAA1549–BRAF fusion; and (4) progressive or refractory glioma with MAPK/ERK pathway activation.
Results
HRQoL was impaired across all subgroups, with overall PedsQL scores ranging from 70.0 to 82.7, reflecting moderate but clinically meaningful reductions prior to treatment. Generic Core scores (74.4±5.9) were similar to Brain Tumor Module HRQOL scores (76.1±2.7), highlighting the specific burden of their neurological disease. Patients with NF1, particularly those with plexiform neurofibromas, demonstrated lower baseline HRQoL compared with non-NF1 PLGG patients, notably in physical functioning and pain-related domains. Across all groups, parent proxy-reports systematically yielded lower HRQoL scores compared with the child self-reports, with mean differences ranging from 3 to 8 points.
Conclusion
Patients with PLGG have impaired HRQoL at baseline, before starting their treatments. Discrepancies between child self-reported and parent proxy-reported HRQoL were identified, consistent with existing literature. These findings highlight the importance of HRQoL assessment in pediatric neuro-oncology clinical trials to provide supportive care.