The Royal Marsden Hospital and The Institute of Cancer Research, Surrey, UK.
Hematology Oncology Division, Department of Women's and Children's Health, University of Padova, Padova, Italy.
Cancer. 2024 Dec 1;130(23):4071-4084. doi: 10.1002/cncr.35497. Epub 2024 Jul 26.
The authors report the prospective evaluation of reduced dose alkylator chemotherapy combined with radiotherapy for European Pediatric Soft Tissue Sarcoma Study Group (EpSSG) standard risk nonalveolar rhabdomyosarcoma (NA-RMS).
Localized node negative Intergroup Rhabdomyosarcoma Study (IRS) II/III NA-RMS at favorable sites (subgroup C), <25 years old, received five cycles of ifosfamide, vincristine, and dactinomycin (IVA) chemotherapy (30 g/m ifosfamide) and four cycles of vincristine and dactinomycin (if receiving radiotherapy), or nine cycles of IVA (54 g/m ifosfamide) ± radiotherapy. Delayed primary tumor excision was considered for IRS III tumors. The primary end points were event-free survival (EFS) and overall survival (OS).
From October 2005 to December 2016, 359 evaluable patients were recruited: orbit, 164 (45.7%); head and neck nonparameningeal, 77 (21.4%); and genitourinary non-bladder/prostate, 118 (32.9%). EFS and OS were 77.4% (95% confidence interval [CI], 72.5-81.6) and 93.5% (95% CI, 90.1-95.8), respectively. Lower dose alkylator chemotherapy and radiotherapy achieved 5-year OS of 93.7% but the difference with higher dose alkylator chemotherapy +/- radiotherapy was not significant (p = 0.8003). Adjuvant radiotherapy improved EFS with 5-year estimates of 84.7% versus 65.2% for nonirradiated (p < .0001), but not OS (p = .9298). Omitting radiotherapy for orbital tumors reduced OS (5-year was 87.1% vs. 97.3% for irradiated, p = .0257). Following R0 resection (n = 60), radiotherapy did not significantly improve EFS or OS.
Radiotherapy for local tumor control allows for reduction of cumulative dose of alkylators in EpSSG standard risk subgroup C RMS patients. The omission of radiotherapy did not affect OS in all patients except those with orbital RMS and was associated with inferior EFS.
作者报告了前瞻性评估低剂量烷化剂化疗联合放疗在欧洲儿科软组织肉瘤研究组（EpSSG）标准风险非肺泡横纹肌肉瘤（NA-RMS）中的应用。
局部淋巴结阴性的国际横纹肌肉瘤研究组（IRS）II/III 期优势部位（C 亚组）非肺泡 RMS，年龄<25 岁，接受五周期异环磷酰胺、长春新碱和多柔比星（IVA）化疗（30g/m 异环磷酰胺）和四周期长春新碱和多柔比星（如果接受放疗），或九周期 IVA（54g/m 异环磷酰胺）±放疗。对于 IRS III 期肿瘤，考虑延迟初次肿瘤切除。主要终点为无事件生存（EFS）和总生存（OS）。
2005 年 10 月至 2016 年 12 月，共招募了 359 例可评估患者：眼眶 164 例（45.7%）；头颈部非副神经节 77 例（21.4%）；泌尿生殖系统非膀胱/前列腺 118 例（32.9%）。EFS 和 OS 分别为 77.4%（95%CI，72.5-81.6）和 93.5%（95%CI，90.1-95.8）。较低剂量的烷化剂化疗和放疗可使 5 年 OS 达到 93.7%，但与高剂量烷化剂化疗+/-放疗相比，差异无统计学意义（p=0.8003）。辅助放疗可改善 EFS，5 年估计值为 84.7%比未放疗的 65.2%（p<0.0001），但 OS 无显著差异（p=0.9298）。眼眶肿瘤行放疗可降低 OS（5 年分别为 87.1%和 97.3%，p=0.0257）。行 R0 切除（n=60）后，放疗对 EFS 或 OS 无显著影响。
为了实现局部肿瘤控制，EpSSG 标准风险 C 亚组 RMS 患者可减少烷化剂的累积剂量。除了眼眶 RMS 患者外，放疗的缺失对所有患者的 OS 没有影响，但与 EFS 下降有关。
