BackgroundEmbryonal 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.MethodsThis 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.ResultsThirty‐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.ConclusionsThe 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.

中文翻译：

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儿科和年轻成人患者的肝脏胚胎肉瘤：儿童肿瘤学小组研究 ARST0332 的报告

背景肝胚胎肉瘤（ESL）是一种罕见的间叶性肿瘤，最常见于儿童；最佳治疗方法尚不确定。对参加一项儿童肿瘤学组 (COG) 临床试验的 ESL 患者的临床特征和结果进行了评估，该临床试验评估了治疗年龄 <30 岁患者软组织肉瘤的基于风险的策略。 COG 研究 ARST0332。对记录、病理学和影像学的集中审查证实了诊断、表现特征、手术范围和并发症。所有患者均接受剂量密集型异环磷酰胺/阿霉素化疗，周期时间取决于手术和放疗。肿瘤切除发生在研究进入之前或四个周期的化疗之后；可选择对残余肿瘤进行放射治疗。结果对 39 名符合条件/可评估的 ESL 患者进行了分析。所有肿瘤直径>10厘米；四个是转移性的。 23 例提前进行肿瘤切除，16 例延迟进行。手术切缘阳性（n= 6) 和术中肿瘤破裂 (n= 6) 仅发生在前期切除中。八名患者接受了放射治疗。估计 5 年无事件生存率和总生存率分别为 79%（95% 置信区间 [CI]，65%–93%）和 95%（95% CI，87%–100%）。切缘阳性增加了局部复发风险。 13 名记录有出血性腹水和/或肿瘤破裂的患者中，有 1 名出现肝外腹腔内肿瘤复发。结论 尽管相当大比例的患者具有高危疾病特征，但 ARST0332 中使用的治疗策略为 ESL 患者取得了良好的结果。将肿瘤切除推迟到新辅助化疗后可能会降低术中肿瘤破裂的风险并提高获得足够手术切缘的可能性。