Real-life experience with a “modified-MEMMAT” regimen for relapsed medulloblastoma

Abstract

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 antiangiogenic approach. This retrospective observational study involved 14 pediatric patients with first or multiple MB recurrence, treated with a modified Medulloblastoma European Multitarget Metronomic Anti-Angiogenic Trial (MEMMAT) strategy. Median patients age 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 were 78.6% (IC 95% 47.2%-92.5%) and 14.3% (IC 95% 2.3%-36.6%), respectively. PFS at 12 and 24 months were 55.0% (95% IC: 25.8%-76.8%) and 15.7% (95% IC 2.5%-39.4%), respectively. Treatment was globally well tolerated. In conclusion, the “modified-MEMMAT” strategy is feasible and a well-tolerated outpatient regimen, leading to a prolonged OS compared to other salvage approaches.