ID #747 A Protocol for Children and Young Adults Diagnosed with a Central Nervous System Tumor to Assess Cognitive, Quality of Life, and Comprehensive Effects of Therapies (“PNOC COMP”)
Jessica Schulte, Shannon Lundy, Gina Pfeifle, Maya Lodish, Alyssa Reddy, Marie Jaeger-Krause, Jane Minturn, Erin Crotty, Iris Paltin, Michael Prados, Ashley Margol, Annette Molinaro, Cassie Kline, Sabine MuellerAbstract
Introduction
As we introduce novel therapeutic strategies to treat young patients with central nervous system (CNS) tumors, it is critical to understand longitudinal impacts on brain development and overall health. PNOC COMP is a prospective study to collect data on long-term health outcomes, cognition, and quality of life (QOL).
Methods
Patients diagnosed 39 years old with CNS tumors were enrolled onto PNOC COMP concurrently with, or independent to, a PNOC therapeutic study. Study procedures included prospective collection of social determinants of health, self/proxy-report of QOL (Peds-QL, PROMIS, EORTC QLQ), self/proxy-report of executive function (BRIEF), functional independence (ABAS-3, MoFI), computerized cognitive assessments (Cogstate), long-term health effects, imaging (MRIs), and biospecimen banking of whole blood. Assessments were collected prior to treatment (baseline), during treatment, at end-of-treatment (EOT), and will continue to be collected for 20 years after EOT. This interim analysis includes data from study year 1 (Jan 2025-Jan 2026).
Results
77 patients enrolled across 13 sites. Pathologic diagnosis included diffuse midline glioma (42%), pilocytic astrocytoma (14%), ependymoma (8%), other glioma (8%), medulloblastoma (7%), and atypical teratoid rhabdoid tumor (8%). Participants had median age of 10 years; 26% of patients were >/=18 years. # of patients who completed study procedures at each timepoint were 77 (baseline), 24 (3 months), 15 (6 months), 5 (9 months), and 4 (end-of-treatment). Baseline QOL measures were completed in 60% of pediatric (<18yo) and 75% of adult patients (>/=18yo), and in 62.7% of high-grade tumor patients and 62.5% of low-grade tumor patients. Of 42 eligible patients, 32 completed >/=1 Cogstate assessment, and 7 completed repeated (>/=2) Cogstate assessments.
Conclusions
PNOC COMP provides a framework to collect critical longitudinal information to inform upfront treatment decisions, provide anticipatory guidance regarding late effects, and minimize long-term deficits in children and young adults diagnosed with CNS tumors.