Back to square one: Treatment for ependymoma in the post-ACNS0831 era

In this issue, Smith et al report the highly anticipated results of the phase 3 randomized clinical trial of post-irradiation chemotherapy in patients with newly diagnosed ependymoma enrolled on the Children’s Oncology Group study ACNS0831.1 In this article, the team highlights several key findings important to the management of ependymoma moving forward and provides key questions for planning the next generation of studies. Based on previous studies, standard therapy for ependymoma has included the best safe resection possible, followed by conformal radiation therapy.2,3 While the evolution of our understanding of the molecular underpinnings of ependymoma has led to further refinements of risk stratification and consideration for treatment modifications with the identification of different disease drivers dependent on which CNS compartment the disease is found in (reviewed in 4), the role of chemotherapy in each subgroup has remained unclear. Previous studies have suggested the possible benefit of 4 cycles of chemotherapy with resection and radiation (CCG9942)5 leading to the currently reported study.