Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA, USA.
Department of Pediatrics, CHC-Clinique de l'Esperance, rue Saint Nicolas, Montegnee, Belgium.
Eur J Cancer. 2019 May;112:98-106. doi: 10.1016/j.ejca.2019.02.001. Epub 2019 Apr 5.
Data on the clinical features, optimal treatment and outcomes of paediatric patients with epithelioid sarcoma (ES) are limited and mostly retrospective.
A subset analysis of ES patients < 30 years of age enrolled on two international prospective clinical trials conducted between 7/2005 and 11/2015 was performed. Risk-adapted therapy was based on tumour diameter, histologic grade, extent of surgery and presence/absence of metastases and included surgery ± radiotherapy for all patients with the addition of ifosfamide/doxorubicin chemotherapy for intermediate-/high-risk patients. Response to therapy, event-free and overall survival and pattern and predictors of treatment failure were evaluated.
Sixty-three ES patients (median age 13.1 years, 52% male) were eligible. Clinical features included the following: 68% extremity, median tumour diameter 3.5 cm, 56% high histologic grade, 14% nodal metastases, 14% distant metastases. Thirty-four low-risk patients underwent surgery (n = 30) or surgery/radiotherapy (n = 4); 16 intermediate-risk and 13 high-risk patients received chemotherapy ± surgery ± radiotherapy. Partial response was observed in 11/22 (50%) patients receiving neoadjuvant therapy. Events were local recurrence (n = 10) and distant recurrence (n = 15); estimated 5-year survival was 86.4%, 63.5% and 0%, respectively, for low-, intermediate- and high-risk patients. Locoregional nodal involvement, invasive tumour, high grade and lesser extent of resection predicted event-free survival in patients without metastases.
Most low-risk ES patients who have undergone an adequate resection fare well without adjuvant therapy. Large tumour size, high histologic grade, tumour invasiveness, inadequate tumour resection and metastatic disease predict poorer outcomes in higher risk ES patients, for whom more effective therapies are needed.
COG ARST0332: ClinicalTrials.gov Identifier NCT00346164, EpSSG NRSTS 2005: European Union Drug Regulating Authorities Clinical Trials No. 2005-001139-31.
关于儿童上皮样肉瘤（ES）患者的临床特征、最佳治疗方法和结局的数据有限，且主要为回顾性研究。
对 2005 年 7 月至 2015 年 11 月期间参与两项国际前瞻性临床试验的年龄<30 岁的 ES 患者进行了亚组分析。风险适应性治疗基于肿瘤直径、组织学分级、手术范围以及有无转移，并对所有患者进行手术+放疗，对于中高危患者，在此基础上增加异环磷酰胺/多柔比星化疗。评估治疗反应、无事件生存和总生存以及治疗失败的模式和预测因素。
共有 63 例 ES 患者（中位年龄 13.1 岁，52%为男性）符合条件。临床特征包括：68%为四肢，肿瘤直径中位数为 3.5cm，56%为高级别组织学分级，14%有淋巴结转移，14%有远处转移。34 例低危患者行手术（n=30）或手术+放疗（n=4）；16 例中危患者和 13 例高危患者接受化疗+手术+放疗。22 例接受新辅助治疗的患者中，11 例（50%）观察到部分缓解。事件为局部复发（n=10）和远处复发（n=15）；低危、中危和高危患者的 5 年生存率分别为 86.4%、63.5%和 0%。无转移患者中，局部淋巴结受累、侵袭性肿瘤、高级别和切除范围较小预测无事件生存。
大多数接受充分切除的低危 ES 患者无需辅助治疗即可获得良好的疗效。大肿瘤大小、高级别组织学分级、肿瘤侵袭性、肿瘤切除不充分和转移性疾病预测高危 ES 患者预后较差，这些患者需要更有效的治疗。
COG ARST0332：ClinicalTrials.gov 标识符 NCT00346164，EpSSG NRSTS 2005：欧盟药品监管机构临床试验编号 2005-001139-31。
