Oberoi Sapna, Xue Wei, Qumseya Amira, Scharschmidt Thomas, Binitie Odion, Sorger Joel I, Kumar Kiran A, Wong Kenneth, Donaldson Sarah S, Teot Lisa, Rudzinski Erin R, Venkatramani Rajkumar
Department of Pediatrics and Child Health, University of Manitoba, Winnipeg, Manitoba, Canada.
Section of Pediatric Hematology-Oncology, CancerCare Manitoba, Winnipeg, Manitoba, Canada.
Cancer. 2025 Jun 15;131(12):e35929. doi: 10.1002/cncr.35929.
Long-term survival and prognostic factors for patients with extremity rhabdomyosarcoma (RMS) treated on contemporary Children's Oncology Group (COG) trials are unknown.
Data of extremity RMS patients enrolled on COG trials from 1998 to 2014 were analyzed to estimate event-free survival (EFS) and overall survival (OS), and factors associated with survival.
The authors identified 264 extremity RMS patients, 159 (60%) localized and 105 (40%) metastatic. The 5-year EFS and OS of patients with localized disease was 62.6% (54%, 71.2%), and 78.7% (71.4%, 85.9%) and of those with metastatic disease was 7.7% (2.2%, 13.2%) and 22.7% (13.9%, 31.4%). Age at diagnosis was associated with EFS, whereas both sex and age at diagnosis were associated with OS. In metastatic patients, a lower Oberlin score was associated with improved EFS and OS, and females had improved OS. The 5-year EFS of patients with localized disease treated on the D9803 trial was not statistically different from those treated on ARST0531, but 5-year OS was statistically superior (adjusted hazard ratio [adjHR] 0.43 [0.21, 0.86]) after adjusting for potential confounders. The 5-year EFS of group 3 patients undergoing delayed primary excision (DPE) with R0 margins was better than those with DPE with positive margins (adjHR 0.31 [0.11, 0.87]) and comparable to group 2 RMS patients.
Outcomes for metastatic extremity RMS remain poor. Among patients with localized disease, younger age, female sex, and treatment on D9803 were associated with improved OS, whereas DPE with R0 margins led to better EFS compared to DPE with positive margins in group 3 patients.
当代儿童肿瘤协作组（COG）试验中接受治疗的肢体横纹肌肉瘤（RMS）患者的长期生存情况及预后因素尚不清楚。
分析1998年至2014年参加COG试验的肢体RMS患者的数据，以估计无事件生存期（EFS）和总生存期（OS），以及与生存相关的因素。
作者确定了264例肢体RMS患者，其中159例（60%）为局限性病变，105例（40%）为转移性病变。局限性疾病患者的5年EFS和OS分别为62.6%（54%，71.2%）和78.7%（71.4%，85.9%），转移性疾病患者的5年EFS和OS分别为7.7%（2.2%，13.2%）和22.7%（13.9%，31.4%）。诊断时的年龄与EFS相关，而诊断时的性别和年龄均与OS相关。在转移性患者中，较低的奥伯林评分与改善的EFS和OS相关，女性的OS有所改善。在D9803试验中接受治疗的局限性疾病患者的5年EFS与在ARST0531试验中接受治疗的患者无统计学差异，但在调整潜在混杂因素后，5年OS在统计学上更优（调整后风险比[adjHR]为0.43[0.21，0.86]）。3组患者接受切缘为R0的延迟一期切除（DPE）后的5年EFS优于切缘阳性的DPE患者（adjHR为0.31[0.11，0.87]），且与2组RMS患者相当。
转移性肢体RMS的预后仍然很差。在局限性疾病患者中，年龄较小、女性以及接受D9803治疗与改善的OS相关，而在3组患者中，切缘为R0的DPE与切缘阳性的DPE相比，EFS更好。
