Purpose
Atypical teratoid/rhabdoid tumor (AT/RT) of the central nervous system is a
rare cancer primarily affecting children younger than age five. Because patients are
young and receive intensive chemotherapy, there is concern regarding late radiation
toxicity, particularly as survival rates improve. Therefore, there is interest in using
proton therapy to treat these tumors. This study was undertaken to investigate outcomes
and acute toxicities associated with proton therapy for AT/RT.
Materials and Methods
The records of 31 patients with AT/RT treated with proton radiation from
October 2008 to August 2013 were reviewed. Demographics, treatment characteristics and
outcomes were recorded and analyzed.
Results
Median age at diagnosis was 19 months (range, 4 – 55 months), with
median age at radiation start of 24 months (range, 6 – 62 months). Seventeen
received local radiation with median dose of 50.4 GyRBE (range, 9 – 54). Fourteen
received craniospinal radiation; half received 24 GyRBE or less and half received 30.6
GyRBE or higher. For patients receiving craniospinal radiation, the median tumor dose
was 54 GyRBE (range, 43.2 – 55.8). Twenty-seven (87%) completed the planned
radiation. With median follow-up of 24 months for all patients (range, 3 – 53
months), median progression-free survival was 20.8 months and median overall survival
was 34.3 months. Five patients (16%) developed clinical findings and imaging changes in
the brainstem one to four months after radiation consistent with radiation reaction; all
resolved with steroids or bevacizumab.
Conclusions
This is the largest report of children with AT/RT treated with proton therapy.
Preliminary survival outcomes in this young pediatric population are encouraging
compared to historic results, but further study is warranted.