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


Journal

Journal of pediatric surgery
ISSN: 1531-5037
Titre abrégé: J Pediatr Surg
Pays: United States
ID NLM: 0052631

Informations de publication

Date de publication:
Mar 2020
Historique:
received: 23 02 2018
accepted: 27 02 2018
pubmed: 2 4 2018
medline: 12 9 2020
entrez: 2 4 2018
Statut: ppublish

Résumé

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. 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. 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. Patients with DSRCT can be risk-stratified at diagnosis based on specific imaging characteristics. Retrospective study with no comparison group. Level IV.

Sections du résumé

BACKGROUND 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 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 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 CONCLUSIONS
Patients with DSRCT can be risk-stratified at diagnosis based on specific imaging characteristics.
TYPE OF STUDY METHODS
Retrospective study with no comparison group.
LEVEL OF EVIDENCE METHODS
Level IV.

Identifiants

pubmed: 29605262
pii: S0022-3468(18)30153-2
doi: 10.1016/j.jpedsurg.2018.02.068
pmc: PMC6126997
mid: NIHMS949232
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

376-380

Subventions

Organisme : NCI NIH HHS
ID : P30 CA008748
Pays : United States

Informations de copyright

Copyright © 2018 Elsevier Inc. All rights reserved.

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Auteurs

James A Saltsman (JA)

Pediatric Surgical Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY.

Anita P Price (AP)

Department of Radiology, Memorial Sloan Kettering Cancer Center, New York, NY.

Debra A Goldman (DA)

Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY.

William J Hammond (WJ)

Pediatric Surgical Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY.

Enrico Danzer (E)

Pediatric Surgical Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY.

Heather Magnan (H)

Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY.

Emily Slotkin (E)

Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY.

William D Tap (WD)

Sarcoma Medical Oncology Service, Department of Medical Oncology, Memorial Sloan Kettering Cancer Center, New York, NY.

Todd E Heaton (TE)

Pediatric Surgical Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY.

Shakeel Modak (S)

Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY.

Michael P LaQuaglia (MP)

Pediatric Surgical Service, Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY. Electronic address: laquaglm@mskcc.org.

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