Targeted Therapy with Sirolimus and Nivolumab in a Child with Refractory Multifocal Anaplastic Ependymoma

Pediatric ependymoma (EPN) is the third most common central nervous system (CNS) tumor, with 90% arising intracranially. Management typically involves maximal surgical resection and radiotherapy, but patients’ outcome poor. Moreover, there are only a few therapeutical options available for recurrent or refractory disease. In this report, we present case of 7-year-old girl relapsed multifocal grade III EPN who failed conventional treatments experienced stable durable response to immune checkpoint inhibitor (ICPI) nivolumab in association mammalian target rapamycin (m-TOR) sirolimus. This experimental therapy was targeted on phenotypical analyses patient’s last relapse tumor sample, procedure should be routinely done find new possible approaches solid tumors.