Treatment of Recurrent Childhood CNS Atypical Teratoid/Rhabdoid Tumor

There is no standard treatment for children with recurrent central nervous system (CNS) atypical teratoid/rhabdoid tumor (AT/RT), and their outcomes are poor.[1]

Trials of molecularly targeted therapy are ongoing. In a study of the EZH2 inhibitor tazemetostat in adult patients with epithelioid sarcoma and non-CNS malignant rhabdoid tumors with SMARCB1 or SMARCA4 loss, prolonged stable disease and objective responses were observed.[2] In the National Cancer Institute (NCI)–Children's Oncology Group Pediatric MATCH APEC1621C (NCT03213665) trial, eight children with AT/RT received tazemetostat. One patient demonstrated disease stabilization.[3][Level of evidence B4]

Stereotactic radiation therapy/radiosurgery or focal radiation therapy can also be considered for the treatment of children with recurrent disease.[4]

Patients or families who desire additional disease-directed therapy should consider entering trials of novel therapeutic approaches because no standard agents have demonstrated clinically significant activity.

Regardless of whether a decision is made to pursue disease-directed therapy at the time of progression, palliative care remains a central focus of management. This ensures that quality of life is maximized while attempting to reduce symptoms and stress related to the terminal illness.