EPENDYMOMA II

The Ependymoma Program is a comprehensive program to improve the accuracy of the primary diagnosis of ependymoma and explore different therapeutic strategies in children, adolescents and young adults, accordingly.
After surgery and central review of imaging and pathology, patients will be offered the opportunity to undergo second look surgery, if possible. Patients will be enrolled in one of 3 different strata according to the outcome of the initial surgical resection, their age or eligibility to receive radiotheapy. These 3 different strata correspond to 3 therapeutic strategies according to the patient status.
Stratum 1 is designed as a randomised phase III study for patients who have had a complete resection, with no measurable disease. Those patients will be randomised to receive conformal radiotherapy followed by either 16 weeks of chemotherapy or observation.
Stratum 2 is designed as a randomised phase III study for patients who have inoperable measurable residual disease. Those patients will be randomised to two different treatment schedules of chemotherapy with or without high-dose methotrexate. After completion of the frontline chemotherapy, patients will be assessed for response and will receive second look surgery when feasible.
For those patients who remain unresectable with residual disease, there will be a study of safety of a radiotherapy boost of 8 Gy that will be administered to the residual tumor immediately after the completion of the conformal radiotherapy. All patients who have not shown progression under chemotherapy will receive a 16 weeks course of chemotherapy.
Stratum 3 is designed as a randomised phase III chemotherapy study in children < 12 months of age or those not eligible to receive radiotherapy. These patients will be randomised to receive a dose dense chemotherapy with or without the addition of the histone deacetylase inhibitor , valproate.
Observational study: Patients who will not fulfil the inclusion criteria of one of the interventional strata will be enrolled and followed up via an observational study.

The overall aim of this project is to improve the oputcome of patients diagnosed with ependymoma by improving and harmonising the staging and the standard of care of this patient population and to improve our understanding of the underlying biology thereby informing future treatment.

Ο αναμενόμενος αριθμός ασθενών στην Ελλάδα είναι: 9 ασθενείς. Μέχρι στιγμής, έχουν ενταχθεί στην μελέτη 3 ασθενείς.