Paediatric Research Institute, Division of Paediatric and Adolescent Medicine, Oslo University Hospital, Oslo, Norway.
Hematology Oncology Division, Department of Women's and Children's Health, University of Padova, Padova, Italy.
Eur J Cancer. 2021 Jul;151:84-93. doi: 10.1016/j.ejca.2021.04.007. Epub 2021 May 7.
BACKGROUND/OBJECTIVES: The primary aim of this study was to analyse and evaluate the impact of different local treatments on the pattern of relapse in children with primary head and neck non-parameningeal (HNnPM) rhabdomyosarcoma (RMS), treated in the European paediatric Soft tissue sarcoma Study Group (EpSSG) RMS2005 study. The secondary aim was to assess whether current risk stratification is valid for this specific site.
DESIGN/METHODS: This study includes all patients with localised HNnPM RMS enrolled in the RMS2005 study between 2005 and 2016. Treatment comprised chemotherapy adapted to risk group, with local surgery and/or radiation therapy. The main outcome measures were event-free survival (EFS) and overall survival (OS).
A total of 165 patients were identified; the median age was 6.4 years (range, 0.1-25). The most common tumour sites were cheek/chin (22%) and nasal ala/nasolabial fold (20%). Histology was unfavourable for 40%, and regional nodal involvement present in 26%. Local therapy included surgery (58%) and/or radiotherapy (72%) to primary tumour and/or regional lymph nodes. After a median follow-up of 66 months (range, 6-158), 42 patients experienced an event, and 17 are still alive. Tumour events were frequent in oral primary (36%), parotid site (26%), cheek/chin (24%), and nasal ala/nasolabial fold (24%) and included locoregional failure in 84% of cases. The 5-year EFS and OS were 75% (95% confidence interval [CI]: 67.3-81.2) and 84.9% (95% CI: 77.5-89.7), respectively. Favourable histology was associated with a better EFS (82.3% versus 64.6%; p = 0.02) and nodal spread with a worse OS (88.6% versus 76.1%; p = 0.04). Different sublocations within the HNnPM primary did not have significant impact on outcome.
Locoregional relapse/progression is the main tumour failure event in this site. Despite frequent unfavourable risk factors, HNnPM RMS remains a favourable location in the context of a risk-adapted strategy.
背景/目的：本研究的主要目的是分析和评估不同局部治疗对欧洲儿科软组织肉瘤研究组（EpSSG） RMS2005 研究中儿童原发性头颈部非副神经节（HNnPM）横纹肌肉瘤（RMS）复发模式的影响。次要目的是评估当前的风险分层是否适用于该特定部位。
方法/设计：本研究包括 2005 年至 2016 年间在 RMS2005 研究中入组的局部 HNnPM RMS 患者。治疗包括根据风险组调整的化疗，辅以局部手术和/或放疗。主要观察指标为无事件生存（EFS）和总生存（OS）。
共确定了 165 例患者；中位年龄为 6.4 岁（范围，0.1-25）。最常见的肿瘤部位为脸颊/下巴（22%）和鼻翼/鼻唇褶皱（20%）。组织学为不良型占 40%，区域淋巴结受累占 26%。局部治疗包括手术（58%）和/或放疗（72%）至原发肿瘤和/或区域淋巴结。中位随访 66 个月（范围，6-158）后，42 例患者出现肿瘤事件，17 例患者仍存活。口腔原发（36%）、腮腺部位（26%）、脸颊/下巴（24%）和鼻翼/鼻唇褶皱（24%）的肿瘤事件较为常见，其中 84%的病例为局部区域失败。5 年 EFS 和 OS 分别为 75%（95%可信区间[CI]：67.3-81.2）和 84.9%（95% CI：77.5-89.7）。良好的组织学与更好的 EFS 相关（82.3%比 64.6%；p=0.02），而淋巴结扩散与更差的 OS 相关（88.6%比 76.1%；p=0.04）。HNnPM 原发性肿瘤的不同亚部位对结局无显著影响。
局部区域复发/进展是该部位肿瘤失败的主要事件。尽管存在频繁的不良危险因素，但在风险适应策略的背景下，HNnPM RMS 仍然是一个有利的部位。
