What question did this study set out to answer?

This review aims to characterize toxicities, outcomes, and molecular features in adolescent and young adult patients with medulloblastoma.

June 26, 2026Open Access

ID #1028 A Retrospective Review Characterizing the Toxicities, Outcomes, and Molecular Characteristics of Adolescent and Young Adult Patients with Medulloblastoma

Key Points

This review aims to characterize toxicities, outcomes, and molecular features in adolescent and young adult patients with medulloblastoma.
Reviewed demographics, clinical features, and tumor tissue analysis of 14 AYA patients with medulloblastoma treated at a single institution since January 2006.
Patients were treated with upfront resection, followed by different chemotherapy regimens and standard radiation therapy.
Median follow-up of four years with data on toxicities and outcomes collected.
Five-year overall survival in the cohort was 86%.
The most common adverse events were peripheral neuropathy and weakness (64%), with ataxia (50%) and depression (50%) also noted.
Molecular classification revealed one WNT-activated tumor, six SHH-activated tumors, and two other groups represented.

Abstract

Abstract Introduction Medulloblastoma, the most common high-grade tumor of the central nervous system (CNS) in children, accounts for less than 1% of CNS tumors in adults. Due to their low prevalence, adolescent and young adult (AYA) patients (ages 15-39 years) with medulloblastoma remain understudied. There have been no large-scale studies correlating molecular characteristics in the AYA population with outcomes, toxicities, or long-term side effects. Methods We reviewed demographics, clinical features, and tumor tissue analysis from 14 AYA patients with medulloblastoma treated at a single institution since January 2006 to better understand their clinical courses, toxicities, and long-term side effects. Results Among 11 molecularly-classified tumors, one was WNT-activated, six were SHH-activated, one belonged to Group 3, and three belonged to Group 4. All patients were treated with upfront resection followed by enrollment to either ACNS0331 (36%), ACNS0332 (29%), SJMB-96 (14%), or a custom regimen containing a platinum agent and vincristine (14%); one patient did not receive chemotherapy at our institution. All patients obtained standard radiation therapy, nine receiving proton radiation and five receiving photon radiation, with boosts ranging from 54 to 61.2 Gy. The most common adverse events during treatment included peripheral neuropathy (64%), weakness (64%), ataxia (50%), depression (50%), hearing loss (43%), and anxiety (36%). Two patients were deceased at the time of evaluation, (one WNT-activated, one Group 3). Median follow-up of our cohort was 4.3 years (range: 0.9 to 20.9 years) and five-year overall survival was 86%. Conclusion AYA patients with medulloblastoma are understudied due to their low prevalence and are often treated according to pediatric protocols. Our review describes AYA patients with medulloblastoma with the goal to delineate age-specific adverse events and outcomes. We hope to increase the size of our review through collaboration with other institutions looking to improve outcomes in the AYA medulloblastoma population.

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