A novel image-based system for risk stratification in patients with desmoplastic small round cell tumor

Background Desmoplastic small round cell tumor (DSRCT) is an aggressive soft tissue sarcoma affecting children and young adults with 5-year overall survival (OS) of approximately 20%. Despite generally poor prognosis, long-term survival does occur. However, no evidence-based system exists to risk-stratify patients at diagnosis. Methods We retrospectively reviewed all DSRCT cases diagnosed at our institution between January 2000 and September 2016. Demographics, diagnostic imaging, and clinical data were reviewed. Univariate and multivariate Cox proportional hazard modeling was used to evaluate associations between imaging characteristics and OS. Results There were 130 patients (85% male; median age at presentation: 21.2 years) with confirmed DSRCT and sufficient imaging and clinical information for analysis. Median 5-year OS was 28% (95% CI: 19%–37%). In univariate analysis, shorter OS was associated with presence of liver lesions (hazard ratio [HR] 2.1, 95% CI: 1.28–3.45), chest lesions (HR 1.86, 95% CI: 1.11–3.1), and ascites (HR 1.69, 95% CI: 1.06–2.7). In multivariate analysis, liver involvement and ascites were predictive and were used to stratify risk (intermediate = no liver involvement or ascites; high = either liver involvement or ascites; very high = both liver involvement and ascites). Intermediate-risk patients had a 5-year survival of 61% (95% CI: 40%–76%) versus 16% (95% CI: 6%–29%) among high-risk patients and 8% (95% CI: 1%–29%) among very high risk patients. Conclusion Patients with DSRCT can be risk-stratified at diagnosis based on specific imaging characteristics. Type of study Retrospective study with no comparison group. Level of evidence Level IV.