ID #722 Individualized therapeutic approaches for relapsed and refractory pediatric ependymoma: A Case Report

Background Pediatric PFA ependymoma that responds poorly to standard treatment lacks effective systemic therapeutic options and carries a dismal prognosis. We report a case of pediatric refractory ependymoma treated with bozitinib, a highly selective c-MET tyrosine kinase inhibitor (TKI). Case Presentation A 7-year-old boy diagnosed with posterior fossa ependymoma (WHO Grade III, PFA group) presented with progressive disease and severe neurological deficits despite undergoing standard treatments, including surgery and radiotherapy. Molecular profiling via next-generation sequencing (NGS) identified MET amplification as the oncogenic driver. Based on this target, the patient was treated with bozitinib (a highly selective c-MET inhibitor with high blood-brain barrier penetration) combined with anlotinib (multi-target tyrosine kinase inhibitor). Follow-up MRI after three months demonstrated a marked reduction in tumor volume in the brainstem and cerebellum, achieving a partial response (PR). Furthermore, the patient experienced significant neurological recovery, regaining the ability to walk independently and perform daily self-care activities. The combination regimen was well tolerated, with no severe adverse events reported. Conclusion Bozitinib exhibits promising antitumor activity and a favorable safety profile in treating refractory pediatric ependymoma with MET amplification. This case suggests that a mechanism-based synergistic targeted combination strategy offers a viable approach to induce tumor regression in refractory pediatric ependymoma. These findings underscore the critical role of molecular profiling in guiding precision medicine.