Leary SES, Packer RJ, Li Y, et al. - Therapy intensification with carboplatin enhanced event-free survival by 19% at 5 years in this randomized clinical trial of children with high-risk group 3 medulloblastoma. Such data indicate the importance of combining clinical and molecular risk stratification for medulloblastoma.

• After central radiologic and pathologic review, 261 patients with medulloblastoma were evaluable; median age, 8.6 years (range, 3.3-21.2); 183 (70%) male; 189 (72%) with metastatic disease; 58 (22%) with diffuse anaplasia; and 14 (5%) with greater than 1.5-cm ² residual disease.

• All participants had a 5-year event-free survival rate of 62.9% and an overall survival rate of 73.4%.

• Isotretinoin randomization was terminated early due to futility.

• Five-year event-free survival was 66.4% with carboplatin vs 59.2% without, with the impact shown only in group 3 subgroup patients: 73.2% with carboplatin vs 53.7% without.

• Overall survival differed by molecular subgroup: WNT pathway activated, 100%; SHH pathway activated, 53.6%; group 3, 73.7%; and group 4, 76.9%.