肺泡横纹肌肉瘤伴区域性淋巴结累及：两个合作组联合分析的结果。
Alveolar rhabdomyosarcoma with regional nodal involvement: Results of a combined analysis from two cooperative groups.
机构信息
University Hospital Vall d'Hebron, Barcelona, Spain.
Children's Hospital Los Angeles, University of Southern California, Los Angeles, California.
出版信息
Pediatr Blood Cancer. 2021 Mar;68(3):e28832. doi: 10.1002/pbc.28832. Epub 2020 Nov 27.
BACKGROUND
Treatment of children and adolescents with alveolar rhabdomyosarcoma (ARMS) and regional nodal involvement (N1) have been approached differently by North American and European cooperative groups. In order to define a better therapeutic strategy, we analyzed two studies conducted between 2005 and 2016 by the European paediatric Soft tissue sarcoma Study Group (EpSSG) and Children's Oncology Group (COG).
METHODS
We retrospectively identified patients with ARMS N1 enrolled in either EpSSG RMS2005 or in COG ARST0531. Chemotherapy in RMS2005 comprised ifosfamide + vincristine + dactinomycin + doxorubicin (IVADo), IVA and maintenance (vinorelbine, cyclophosphamide); in ARST0531, it consisted of either vincristine + dactinomycin + cyclophosphamide (VAC) or VAC alternating with vincristine + irinotecan (VI). Local treatment was similar in both protocols.
RESULTS
The analysis of the clinical characteristics of 239 patients showed some differences between study groups: in RMS2005, advanced Intergroup Rhabdomyosarcoma Study Group (IRS) and large tumors predominated. There were no differences in outcomes between the two groups: 5-year event-free survival (EFS), 49% (95% confidence interval [CI]: 39-59) and 44% (95% CI: 30-58), and overall survival (OS), 51% (95% CI: 41-61) and 53.6% (95% CI: 40-68) in RMS2005 and ARST0531, respectively. In RMS2005, EFS of patients with FOXO1-positive tumors was significantly inferior to those with FOXO1-negative (49.3% vs 73%, P = .034). In contrast, in ARST0531, EFS of patients with FOXO1-positive tumors was 45% compared with 43.8% for those with FOXO1-negative.
CONCLUSIONS
The outcome of patients with ARMS N1 was similar in both protocols. However, patients with FOXO1 fusion-negative tumors enrolled in RMS2005 showed a significantly better outcome, suggesting that different strategies of chemotherapy may have an impact in the outcome of this subgroup of patients.
背景
北美和欧洲的合作组对患有肺泡横纹肌肉瘤（ARMS）和区域淋巴结受累（N1）的儿童和青少年的治疗方法有所不同。为了制定更好的治疗策略，我们分析了欧洲儿科软组织肉瘤研究组（EpSSG）和儿童肿瘤组（COG）在 2005 年至 2016 年期间进行的两项研究。
方法
我们回顾性地确定了在 EpSSG RMS2005 或 COG ARST0531 中登记的 ARMS N1 患者。RMS2005 中的化疗包括异环磷酰胺+长春新碱+放线菌素 D+多柔比星（IVADo）、IVA 和维持（长春瑞滨、环磷酰胺）；在 ARST0531 中，它由长春新碱+放线菌素 D+环磷酰胺（VAC）或 VAC 与长春新碱+伊立替康（VI）交替组成。两种方案的局部治疗相似。
结果
对 239 例患者的临床特征分析显示，两组间存在一些差异：在 RMS2005 中，晚期 Intergroup Rhabdomyosarcoma Study Group（IRS）和大肿瘤更为常见。两组之间的结局无差异：5 年无事件生存率（EFS）分别为 49%（95%置信区间[CI]：39-59）和 44%（95% CI：30-58），总生存率（OS）分别为 51%（95% CI：41-61）和 53.6%（95% CI：40-68）。在 RMS2005 中，FOXO1 阳性肿瘤患者的 EFS 明显低于 FOXO1 阴性肿瘤患者（49.3%比 73%，P=0.034）。相比之下，在 ARST0531 中，FOXO1 阳性肿瘤患者的 EFS 为 45%，而 FOXO1 阴性肿瘤患者的 EFS 为 43.8%。
结论
两种方案中 ARMS N1 患者的结局相似。然而，在 RMS2005 中登记的 FOXO1 融合阴性肿瘤患者的结局明显更好，这表明不同的化疗策略可能对这亚组患者的结局有影响。
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