Department of Hematology/Oncology, Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University, Shanghai, 200127, China.
World J Pediatr. 2010 Feb;6(1):43-9. doi: 10.1007/s12519-010-0005-6. Epub 2010 Feb 9.
Little was known about the therapeutic result of rhabdomyosarcomas (RMSs) and other malignant tumors until the end of the last century in China. Very few prospective clinical research results have been reported. We designed a RS-99 protocol under close cooperation of a multidisciplinary team including surgeons, radiologists, pathologists, and pediatric oncologists at Shanghai Children's Medical Center. This study aimed to improve the prognosis of childhood solid tumors and analyze the results of different tumors with the same protocol, including RMSs, the Ewing sarcoma family of tumors (ESFTs), and ex-cranial germ cell tumors (GCTs).
Sixty-six patients with malignant solid tumors [RMS (n=30), GCT (n=22), and ESFT (n=14)] were enrolled on the RS-99 protocol from October 1998 to October 2006. They were 34 girls and 32 boys aged 9 to 194 months. The protocol involved surgery, radiotherapy and chemotherapy which included VCP (vincristine, cisdiaminedichloroplatinum, and cyclophosphamide) and IEV (etoposide, vincristine and ifosfamide) for the low-risk group, AVCP (adriamycin, vincristine, cisdiaminedichloroplatinum, and cyclophosphamide) and IEV for the intermediate-risk group and high-risk group. Peripheral blood stem cell transplantation was suggested for the high-risk group. Radiotherapy was only given for RMS and ESFT. Differences in survival between the groups were determined by comparison of entire survival curves and tested by the Kaplan-Meier method and the log-rank tests.
The 5-year event-free survival (EFS) for the whole group (RMS, ESFT and GCT) was 60%. The 5-year EFS for children with RMS was 35% (95% CI 16-54), GCT was 79% (95% CI 70-88) and ESFT was 72% (95% CI 58-86). The 5-year EFS showed that the patients with RMS in the retroperitoneum-pelvis did not have a better result than those with tumors in other sites (P=0.604). The histological classification of RMS exerted prognostic influence on the estimated 5-year EFS (P=0.04). Tumor stage and risk group were also contributive to prognosis (P=0.008). For GCT patients, the primary sites of tumors and their histological classification did not influence the therapeutic result (P=0.814). The 5-year EFS was 100% in stage I and II versus 62% in stage III and IV patients (P=0.02). Because of the small number of patients, we did not analyze the prognostic factors for patients with ESFT. No organ failure or functional impairment occurred in the patients enrolled in the RS-99 protocol. One ESFT patient developed a second cancer.
The RS-99 protocol is well tolerated and is reasonable for the 3 different tumors. Risk-based grouping protocol design is needed and the protocol for high risk RMS should be revised.
直到上世纪末，中国对横纹肌肉瘤（RMS）和其他恶性肿瘤的治疗效果知之甚少。很少有前瞻性临床研究结果报告。我们在包括外科医生、放射科医生、病理学家和儿科肿瘤学家在内的多学科团队的密切合作下，设计了 RS-99 方案。本研究旨在提高儿童实体瘤的预后，并分析同一方案治疗不同肿瘤的结果，包括 RMS、尤文肉瘤家族肿瘤（ESFT）和颅外生殖细胞瘤（GCT）。
1998 年 10 月至 2006 年 10 月，66 例恶性实体瘤患儿[RMS（n=30）、GCT（n=22）和 ESFT（n=14）]按 RS-99 方案入组。患儿年龄 9-194 个月，其中女 34 例，男 32 例。方案包括手术、放疗和化疗，低危组采用 VCP（长春新碱、顺铂和环磷酰胺）和 IEV（依托泊苷、长春新碱和异环磷酰胺），中危组和高危组采用 AVCP（阿霉素、长春新碱、顺铂和环磷酰胺）和 IEV。高危组建议行外周血干细胞移植。放疗仅用于 RMS 和 ESFT。通过比较整个生存曲线，采用 Kaplan-Meier 方法和对数秩检验来确定各组之间的生存差异。
全组（RMS、ESFT 和 GCT）5 年无事件生存率（EFS）为 60%。RMS 患儿 5 年 EFS 为 35%（95%CI 16-54），GCT 为 79%（95%CI 70-88），ESFT 为 72%（95%CI 58-86）。5 年 EFS 显示，腹膜后-盆腔 RMS 患儿的预后并不优于其他部位肿瘤患儿（P=0.604）。RMS 的组织学分类对估计的 5 年 EFS 有预后影响（P=0.04）。肿瘤分期和危险组也与预后有关（P=0.008）。对于 GCT 患儿，肿瘤的原发部位和组织学分类对治疗结果无影响（P=0.814）。Ⅰ期和Ⅱ期患者 5 年 EFS 为 100%，Ⅲ期和Ⅳ期患者为 62%（P=0.02）。由于病例数较少，我们未分析 ESFT 患者的预后因素。RS-99 方案入组患者无器官衰竭或功能损害。1 例 ESFT 患者发生二次癌症。
RS-99 方案耐受性良好，适用于 3 种不同的肿瘤。需要进行基于风险的分组方案设计，高危 RMS 的方案需要修订。
