Multimodal Treatment of Desmoplastic Small Round Cell Tumors

Desmoplastic small round cell tumor (DSRCT) is a rare soft tissue sarcoma characterized by a unique EWS-WT1 translocation. Unfortunately, DSRCT is often asymptomatic until advanced stages and associated with high mortality rates. Treatment regimens are multimodal, involving chemotherapy and cytoreductive surgery (CRS) often combined with whole abdomen radiation (WART) and/or heated intraperitoneal chemotherapy (HIPEC). The 5-year mortality rate remains 15-30% despite aggressive treatment. Each treatment modality carries significant potential morbidity. Given these risks, the survival benefits of a complete treatment regimen treatment must be weighed against the associated morbidity.  The first aim of this study will be to retrospectively evaluate the relative contributions of (1) complete CRS, (2) WART and (3) HIPEC on survival (5-yr abdominal EFS and OS) and to assess the relative impact of each modality on treatment-related morbidity. The second aim of this study will be to prospectively assess quality of life by (1) examining how the associated morbidity of these treatment components impact survivors’ quality of life through validated measures (2) exploring how parents and patients perceive treatment decisions.

PI: Tim Lautz and Roshni Dasgupta

Contact Maddie Peters (Madeline.Peters@cchmc.org) if you have any questions on how to activate the study at your PSORC institution.