Sometimes, More is Less in Atypical Teratoid Rhabdoid Tumor (ATRT): The Risks of Maintenance Intrathecal Chemotherapy

Atypical Teratoid Rhabdoid Tumors (ATRT) are rare and aggressive pediatric brain tumors, and lack of consensus exists regarding optimal therapy. Some physicians recommend maintenance intrathecal chemotherapy, extrapolating from the salvage setting where it has been reported to promote remission. However, the risks of this therapy may be underappreciated. Here we present a case of a young patient with ATRT who received consolidation intrathecal chemotherapy following the ACNS 0333 regimen (resection, induction chemotherapy, high-dose chemotherapy with stem cell rescue, and radiotherapy) and developed life-altering neurologic sequelae. In the absence of clear benefit, we recommend against routine maintenance intrathecal chemotherapy.