有关起初有远处转移的儿科非横纹肌软组织肉瘤（NRSTS）的临床表现的数据有限，目前尚未明确规定明确的治疗标准。此队列研究报道了两项同时进行的欧洲前瞻性研究，即随机的BERNIE研究和由欧洲儿科软组织肉瘤研究小组开发的单臂MTS 2008研究中登记的儿科成人型转移性NRSTS。治疗方案最初是为转移性横纹肌肉瘤患者设计的，即九个周期的多药化疗（在BERNIE研究中带或不带贝伐单抗），随后进行12个维持治疗周期，而放疗和/或手术（在原发肿瘤和/或转移部位）则延迟至注射七个化疗周期之后。该研究包括61名2008年7月至2016年12月期间接受治疗的<21岁患者。在75％的病例中，肺是转移部位。所有患者接受多种药物化疗，44％的患者对原发肿瘤进行了局部治疗，18％的患者对转移病灶进行了治疗。中位进展/复发时间为6个月。在随访时间中位数为41.5个月（范围为2-111个月）的情况下，3年无事件生存和总生存率分别为15.4％（95％置信区间[CI]，7.6-25.7）和34.9％（95％ CI，22.7-47.5）。根据所施与的治疗类型，结果没有统计学上显著的差异。研究证实了转移性NRSTS患者总体预后不佳，其治疗仍然具有挑战性。儿科非横纹肌软组织肉瘤形成了一组罕见的异质性肿瘤。尽管最近的国际研究已经定义了针对局部疾病患者的标准护理，但对于远处转移患者的临床行为仅有有限的数据可用。这项关于61个转移病例的研究，通过两个前瞻性欧洲方案的治疗证实，这类患者的生存几率通常不佳，仍缺乏标准治疗。©2023美国癌症协会。

Limited data exist on the clinical behavior of pediatric non-rhabdomyosarcoma soft tissue sarcomas (NRSTS) with distant metastases at onset, and a clear standard of care has not yet been defined.This cohort study reports on pediatric adult-type metastatic NRSTS enrolled in two concurrent prospective European studies, i.e., the randomized BERNIE study and the single-arm MTS 2008 study developed by the European paediatric Soft tissue sarcoma Study Group. Treatment programs were originally designed for patients with metastatic rhabdomyosarcoma, i.e., nine courses of multidrug chemotherapy (with or without bevacizumab in the BERNIE study), followed by 12 cycles of maintenance therapy, whereas radiotherapy and/or surgery (on primary tumor and/or metastases) were delayed until after seven courses of chemotherapy had been administered.The study included 61 patients <21 years old treated from July 2008 to December 2016. The lung was the site of metastases in 75% of the cases. All patients received multi-agent chemotherapy, 44% had local therapy to primary tumor, and 18% had treatment of metastases. Median time to progression/relapse was 6 months. A high rate of tumor progression was observed during the initial part of the chemotherapy program. With a median follow-up of 41.5 months (range, 2-111 months), 3-year event-free survival and overall survival were 15.4% (95% confidence interval [CI], 7.6-25.7) and 34.9% (95% CI, 22.7-47.5), respectively. There were no statistically significant differences in outcome depending on the type of treatment administered.The study confirmed the overall poor outcome for patients with metastatic NRSTS, whose treatment remains a challenge.Pediatric non-rhabdomyosarcoma soft tissue sarcomas form a heterogeneous group of rare tumors. Although recent international studies have defined the standard of care for patients with localized disease, limited data are available on the clinical behavior of patients with distant metastases. This study on 61 metastatic cases treated on two prospective European protocols confirms that the chances of survival of such patients are often dismal and a standard treatment is still lacking.© 2023 American Cancer Society.