Department of Pediatrics, Division of Hematology/Oncology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, Arkansas, USA.
Department of Pathology, Microbiology, and Immunology, Vanderbilt University School of Medicine, Nashville, Tennessee, USA.
Pediatr Blood Cancer. 2024 Aug;71(8):e31062. doi: 10.1002/pbc.31062. Epub 2024 May 16.
In retrospective analyses, the Pediatric Oncology Group [POG) and the Federation National des Centres de Lutte Contre le Cancer (FNCLCC) histologic grade predict outcome in pediatric non-rhabdomyosarcoma soft tissue sarcoma (NRSTS), but prospective data on grading, clinical features, and outcomes of low-grade NRSTS are limited.
We analyzed patients less than 30 years of age enrolled on Children's Oncology Group (COG) study ARST0332 (NCT00346164) with POG grade 1 or 2 NRSTS. Low-risk patients were treated with surgery alone. Intermediate-/high-risk patients received ifosfamide/doxorubicin and radiotherapy, with definitive resection either before or after 12 weeks of chemoradiotherapy.
Estimated 5-year event-free and overall survival were 90% and 100% low risk (n = 80), 55% and 78% intermediate risk (n = 15), and 25% and 25% high risk (n = 4). In low-risk patients, only local recurrence was seen in 10%; none with margins greater than 1 mm recurred locally. Sixteen of 17 intermediate-/high-risk patients who completed neoadjuvant chemoradiotherapy underwent gross total tumor resection, 80% with negative margins. Intermediate-/high-risk group events included one local and seven metastatic recurrences. Had the FNCLCC grading system been used to direct treatment, 29% of low-risk (surgery alone) patients would have received radiotherapy ± chemotherapy.
Most low-risk patients with completely resected POG low-grade NRSTS are successfully treated with surgery alone, and surgical margins greater than 1 mm may be sufficient to prevent local recurrence. Patients with intermediate- and high-risk low-grade NRSTS have outcomes similar to patients with high-grade histology, and require more effective therapies. Use of the current FNCLCC grading system may result in overtreatment of low-risk NRSTS curable with surgery alone.
在回顾性分析中，儿科肿瘤学组（POG）和法国全国癌症中心联合会（FNCLCC）的组织学分级可预测儿科非横纹肌肉瘤软组织肉瘤（NRSTS）的预后，但关于低级别 NRSTS 的分级、临床特征和结果的前瞻性数据有限。
我们分析了入组儿童肿瘤学组（COG）研究 ARST0332 的年龄小于 30 岁的患者，这些患者的 POG 分级为 1 或 2 级 NRSTS。低危患者仅接受手术治疗。中危/高危患者接受异环磷酰胺/多柔比星和放疗，在 12 周化疗和放疗前后进行确定性切除术。
低危患者的 5 年无事件生存率和总生存率估计分别为 90%和 100%（n=80），中危患者分别为 55%和 78%（n=15），高危患者分别为 25%和 25%（n=4）。在低危患者中，仅 10%发生局部复发；无 1 例边缘大于 1mm 的患者局部复发。17 例中危/高危患者中，有 16 例完成新辅助化疗和放疗后进行了大体全肿瘤切除术，其中 80%的患者切缘阴性。中危/高危组的事件包括 1 例局部复发和 7 例远处转移复发。如果使用 FNCLCC 分级系统来指导治疗，29%的低危（单独手术）患者将接受放疗±化疗。
大多数完全切除 POG 低级别 NRSTS 的低危患者单独手术治疗即可获得成功，且大于 1mm 的手术边缘可能足以预防局部复发。中危和高危低级别 NRSTS 患者的预后与高级别组织学患者相似，需要更有效的治疗方法。使用当前的 FNCLCC 分级系统可能会导致对单独手术即可治愈的低危 NRSTS 过度治疗。
