Embryonal sarcoma of the liver in pediatric and young adult patients: A report from Children's Oncology Group study ARST0332

Authors

Sheri L. Spunt, Department of Pediatrics, Stanford University School of Medicine, Palo Alto, California, USA.
Wei Xue, Department of Biostatistics, University of Florida College of Public Health and Health Professions and College of Medicine, Gainesville, Florida, USA.
Zhengya Gao, Department of Biostatistics, University of Florida College of Public Health and Health Professions and College of Medicine, Gainesville, Florida, USA.
Andrea Hayes Dixon, Department of Surgery, Howard University College of Medicine, Washington, District of Columbia, USA.
Lynn Million, Department of Radiation Oncology, Stanford University School of Medicine, Palo Alto, California, USA.

Document Type

Article

Publication Date

4-3-2024

Journal Title

Cancer

Abstract

BACKGROUND: Embryonal sarcoma of the liver (ESL) is a rare mesenchymal tumor most common in childhood; the optimal treatment approach is uncertain. The clinical features and outcomes of patients with ESL enrolled in a Children's Oncology Group (COG) clinical trial that evaluated a risk-based strategy for treating soft tissue sarcomas in patients aged <30 years were evaluated. METHODS: This subset analysis included patients with ESL enrolled in COG study ARST0332. Central review of records, pathology, and imaging confirmed the diagnosis, presenting features, and surgery extent and complications. All patients received dose-intensive ifosfamide/doxorubicin chemotherapy, with cycle timing dependent on surgery and radiotherapy. Tumor resection occurred before study entry or after four cycles of chemotherapy; radiotherapy for residual tumor was optional. RESULTS: Thirty-nine eligible/evaluable patients with ESL were analyzed. All tumors were >10 cm in diameter; four were metastatic. Tumor resection was performed upfront in 23 and delayed in 16. Positive surgical margins (n = 6) and intraoperative tumor rupture (n = 6) occurred only in upfront resections. Eight patients received radiotherapy. Estimated 5-year event-free and overall survival were 79% (95% confidence interval [CI], 65%-93%) and 95% (95% CI, 87%-100%), respectively. Positive margins increased the local recurrence risk. One of 13 patients with documented hemorrhagic ascites and/or tumor rupture developed extrahepatic intra-abdominal tumor recurrence. CONCLUSIONS: The treatment strategy used in ARST0332 achieved favorable outcomes for patients with ESL despite a substantial proportion having high-risk disease features. Deferring tumor resection until after neoadjuvant chemotherapy may decrease the risk of intraoperative tumor rupture and improve the likelihood of adequate surgical margins.

Recommended Citation

Spunt, Sheri L.; Xue, Wei; Gao, Zhengya; Hayes Dixon, Andrea; and Million, Lynn, "Embryonal sarcoma of the liver in pediatric and young adult patients: A report from Children's Oncology Group study ARST0332" (2024). MaineHealth Maine Medical Center. 3658.
https://knowledgeconnection.mainehealth.org/mmc/3658