PURPOSE We analysed the cohort of paediatric patients with metastatic non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) treated in the BERNIE protocol, i.e. open-label, multicentre, randomised phase II study evaluating the role of bevacizumab (BO20924/ITCC-006; ClinicalTrials.gov: NCT00643565). METHODS Eligible patients were randomised 1:1 to add or not add bevacizumab to nine courses of intensive multi-drug chemotherapy, followed by 12-month maintenance chemotherapy (plus surgery and radiotherapy). The primary end-point was event-free survival (EFS); secondary objectives were objective response rate (ORR) and overall survival (OS). RESULTS From 2008 and 2013, 49 NRSTS patients (out of 154 cases) were treated, 26 in the standard arm and 23 in the bevacizumab arm. ORR was seen in 10 out of 36 evaluable cases (27.7%), i.e. 4/18 standard arm cases and 6/18 bevacizumab arm cases. Two-year EFS was 27.3% (95% confidence interval [CI] 13.9-42.5) for all NRSTS patients, i.e. 34.9% (95% CI 14.6-56.2) for bevacizumab arm and 22.9% (95% CI 7.1-43.9) for standard arm (p-value = 0.19). Three-year OS (median follow-up 48.6 months) was 35.2%, with no differences in the two arms. Time to event and time to death were 16.3 and 17.2 months for bevacizumab arm and 2.1 and 7.6 months for standard arm, respectively. Patients not receiving any local treatment on primary disease had a worse outcome as compared to others. Treatment results were better for patients receiving surgical resection and worse for those who did not receive any specific treatment. CONCLUSION The addition of the anti-angiogenic agent to the standard chemotherapy did not show statistically significant improvement in survival in metastatic NRSTS.

中文翻译：

---

在BERNIE研究中治疗的转移性非横纹肌肉瘤软组织肉瘤（NRSTS）的结果：一项随机的II期研究，评估在化学疗法中添加贝伐单抗。

目的我们分析了在BERNIE方案中治疗的转移性非横纹肌肉瘤软组织肉瘤（NRSTS）的儿科患者队列，即开放性，多中心，随机II期研究，评估贝伐单抗的作用（BO20924 / ITCC-006; ClinicalTrials。 gov：NCT00643565）。方法将符合条件的患者按1：1比例随机分配至9个疗程的强化多药化疗方案中，加或不加贝伐单抗，然后进行12个月的维持化疗（加手术和放疗）。主要终点是无事件生存（EFS）。次要目标是客观反应率（ORR）和总体生存率（OS）。结果从2008年至2013年，共治疗了49例NRSTS患者（154例患者），其中标准组26例，贝伐单抗组23例。在36例可评估病例中，有10例（27.7％）观察到ORR，即 4/18标准手臂病例和6/18贝伐单抗手臂病例。所有NRSTS患者的两年EFS为27.3％（95％置信区间[CI] 13.9-42.5），即贝伐单抗组为34.9％（95％CI 14.6-56.2）和22.9％（95％CI 7.1-43.9）。标准手臂（p值= 0.19）。三年OS（中位随访48.6个月）为35.2％，两组之间无差异。贝伐单抗组的事件发生时间和死亡时间分别为16.3和17.2个月，标准组为2.1和7.6个月。与其他人相比，未接受任何原发疾病局部治疗的患者的预后较差。对于接受外科手术切除的患者，治疗结果更好，而对那些未接受任何特定治疗的患者，治疗结果则更差。