Therapeutic advances in medical oncology · 2025
Real-life experience with a "modified-MEMMAT" regimen for relapsed medulloblastoma.
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Cacchione Antonella, Del Baldo Giada, D'Antonio Federica, Di Ruscio Valentina, Megaro Giacomina, Pilotto Chiara, Tornesello Assunta, Cocciolo Alessandro, Vennarini Sabina, Chiesa Silvia, Carai Andrea, De Salvo Andrea, Albino Giulia, Colafati Giovanna Stefania, Slavc Irene, Mastronuzzi Angela
Аннотация
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.
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.