Abstract Background Pediatric patients with relapsed ependymoma face a dismal prognosis, as current salvage therapies often fail to produce durable responses. Given the prominent vascularity and angiogenic potential of these tumors, metronomic anti-angiogenic strategies represent a promising therapeutic avenue. This study evaluates the clinical efficacy and safety profile of the MEMMAT (Metronomic Multi-drug Anti-angiogenic Therapy) regimen in children with recurrent or progressive ependymoma. Methods We conducted a retrospective review of sixteen pediatric patients treated with the MEMMAT protocol between July 2014 and December 2025. Data were extracted from electronic medical records to evaluate objective response rates (ORR), progression-free survival (PFS), overall survival (OS), and the regimen’s toxicity profile. Results The cohort (median age: 6.4 years) received biweekly intravenous bevacizumab in combination with oral thalidomide, celecoxib, fenofibrate and alternating etoposide/cyclophosphamide. The ORR was 44%, comprising three partial responses and four cases of stable disease. The median time to progression was 12 months, and the median OS was 3.95 years. PFS rates at 1 and 2 years were 45% and 25%, respectively. The 2-year and 5-year OS rates reached 87% and 24%, respectively. Although 50% of patients eventually progressed, the therapy was well-tolerated with a manageable safety profile; four deaths were attributed to causes unrelated to tumor progression or treatment-related toxicity. At last follow-up, three patients remained alive, including survivors at 14 and 100 months post-treatment initiation. Conclusion The MEMMAT regimen demonstrates encouraging clinical activity and acceptable tolerability in the challenging setting of relapsed pediatric ependymoma. These findings suggest that metronomic anti-angiogenic therapy is a viable therapeutic strategy when standard care options are exhausted. Additional cases from other international institutions are currently being collected and will be presented at the time of the meeting.
Ríos et al. (Tue,) studied this question.