Successful outcome with tandem myeloablative chemotherapy and autologous peripheral blood stem cell transplants in a patient with atypical teratoid/rhabdoid tumor of the central nervous system

Atypical teratoid rhabdoid tumors (ATRT) are highly malignant tumors of the central nervous system with a peak incidence in children less than 3 years of age. Despite multimodal therapy including surgery, radiation and chemotherapy, the prognosis remains dismal. No specific treatment guidelines are defined for ATRTs but a gross total resection and radiation therapy (RT) appear to improve overall outcome. In children less than 3 years of age, the prognosis is dismal due in part to the reluctance to utilize RT given its severe neurological sequelae. To avoid RT in this age group, intensification of chemotherapy has been tried and has shown to improve outcome. Myeloablative chemotherapy followed by autologous stem cell re-infusion has been used as a modality to intensify therapy but there are no reports of use of tandem myeloablative regimens and autologous stem cell re-infusions for treatment of ATRT. We herein report the case of a 4-month-old boy with ATRT with partial resection of his tumor who achieved complete remission using tandem high-dose therapy followed by autologous peripheral blood stem cell re-infusions despite having biopsy proven disease at the time of starting the tandem regimens. This was achieved without the use of RT as a treatment modality.