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.

Management of desmoplastic small round-cell tumors in children and young adults.

Desmoplastic small round cell tumor (DSRCT) is a rare type of sarcoma which is primarily abdominal in origin. Less than 200 cases have been reported. DSRCT presents with multifocal tumors believed to begin in the peritoneal surfaces of the abdominal cavity. Up to hundreds of tumors can be found in the abdominal cavity. Adolescent males are primarily affected. Because of the rarity of the tumor and the unusually aggressive presentation, treatment is challenging and has not been standardized. Here we summarize the clinical presentation, diagnosis and management options of this rare tumor.