Strategies for the Treatment of Desmoplastic Small Round Cell Tumor: A Case Series

Desmoplastic small round cell tumor (DSRCT) is a rare, aggressive pediatric sarcoma that has a poor prognosis despite a multimodal approach of surgical resection, chemotherapy, and radiation. Incidence is only approximately 0.2 cases per million, limiting clinical trials from which to derive a standard of treatment. Advancement instead relies on case reports and series. The Children's Hospital at Montefiore in the Bronx, New York, a tertiary care hospital associated with the Montefiore Einstein Comprehensive Cancer Center, has treated eight such patients in the last decade, resulting in one of the largest single-institution case series of DSRCT patients to date. Though these patients are demographically unusual for DSRCT, including two women and two with rare extra-abdominal tumors of the brain and bone, through treatment of these patients we have accrued experience regarding various treatment strategies in both primary and refractory DSRCT. We treat primary DSRCT with interval-compressed vincristine/doxorubicin/cyclophosphamide alternating with cycles of ifosfamide/etoposide and irinotecan/temozolomide/temsirolimus (ITT). This is the first descriptive series of this size describing the use of ITT in DSRCT. Treatment for refractory cases focuses on targeted therapies when available, emphasizing the personalization that DSRCT treatment requires.