Treatment-related morbidity in atypical teratoid/rhabdoid tumor: multifocal necrotizing leukoencephalopathy

Atypical teratoid/rhabdoid tumor (AT/RT) is an aggressive malignant brain tumor that, since it was first identified, has been treated with aggressive treatment regimens, e.g. high-dose chemotherapy with stem cell rescue and early radiotherapy. We reviewed our experience because of concerns with respect to treatment-related toxicity in our patients.

Seven patients with a median age at presentation of 18 months were diagnosed with AT/RT between 1996 and 2006. Tumor location was supratentorial in 2 patients, in the posterior fossa in 4 and spinal in 1. Gross total resection was performed in 1 patient, subtotal resection in 5 and biopsy only in 1. Adjuvant treatment consisted of chemotherapy and radiotherapy in 5 patients.

Median progression-free survival was 4 months, and median overall survival was 7 months. Two children are alive at 44 and 102 months. Significant surgical and chemotherapy-related morbidity was seen. Biopsy-proven multifocal necrotizing leukoencephalopathy (MNL) was seen in one patient who is alive 44 months after diagnosis. Another patient who was thought to have recurrent tumor in the brainstem 9 months after diagnosis had imaging findings compatible with MNL.

Although improving results are reported for AT/RT using intensive treatment regimens, treatment-related morbidity is considerable in this young patient population.