Real-life experience with a "modified-MEMMAT" regimen for relapsed medulloblastoma.

Background: Medulloblastoma (MB) relapse is typically resistant to available treatments. An emerging alternative strategy focuses on disrupting tumor angiogenesis at various stages, using a combined metronomic anti-angiogenic approach.

Objectives: The study aims to assess the efficacy and safety of this modified treatment approach in managing recurrent MB in the pediatric population.

Designs: This study is a retrospective observational analysis involving 14 pediatric patients diagnosed with first or multiple recurrences of MB.

Methods: We analyzed clinical, molecular, radiological, and outcome data of our cohort treated using a modified Medulloblastoma European Multitarget Metronomic Anti-Angiogenic Trial (MEMMAT) strategy.

Results: Median age of patients was 11.6 years (range: 6.4-26 years). All 14 patients presented with a metastatic relapse after conventional treatments. The median time from primary diagnosis/prior relapse to the start of "modified MEMMAT" was 22 months (range: 2-60 months). Fifty-seven percent received the "modified MEMMAT" schema as second-line treatment, while 43% received it as third-line or beyond after recurrence. At a median follow-up of 17.9 months, the median overall survival (OS) from the MEMMAT start date was 18.2 months, and the median progression-free survival (PFS) was 12.8 months. OS at 12 and 24 months was 78.6% and 28.6%, respectively. PFS at 6 and 12 months was 100% and 55.0%, respectively. Treatment was globally well tolerated.

Conclusion: The modified MEMMAT strategy shows promise in treating recurrent MB, achieving a 12-month OS rate from date of starting treatment of 78.6%, with manageable toxicity. These findings suggest its potential as a viable option for heavily pre-treated pediatric patients, warranting further validation in larger prospective studies.