Department of Pediatric Surgery, Children's Hospital of Pittsburgh, University of Pittsburgh, Pittsburgh, Pennsylvania.
Cancer. 2011 Jun 1;117(11):2541-50. doi: 10.1002/cncr.25719. Epub 2010 Dec 14.
The objectives of this study were to compare tumor volume and patient weight versus traditional factors of tumor size (greatest dimension) and patient age and to determine which parameters best discriminated outcome among pediatric patients with intermediate-risk rhabdomyosarcoma (RMS).
Complete information was available for 370 patients with nonmetastatic RMS who were enrolled in the Children's Oncology Group (COG) intermediate-risk study D9803 (1999-2005). The Kaplan-Meier method was used to estimate survival distributions. A recursive partitioning model was used to identify prognostic factors that were associated with event-free survival (EFS). Cox proportional hazards regression models were used to estimate the association between patient characteristics and the risk of failure or death.
For all patients with intermediate-risk RMS, a recursive partitioning algorithm for EFS suggested that prognostic groups should be defined optimally by tumor volume (with a transition point at 20 cm(3) ), patient weight (with a transition point at 50 kg), and embryonal histology. Tumor volume and patient weight added significant outcome information to the standard prognostic factors, including greatest tumor dimension and patient age (P = .02). The ability to resect the tumor completely was not associated significantly with the size of the patient, and patient weight did not significantly modify the association between tumor volume and EFS after adjustment for standard risk factors (P = .2).
The factors that had the strongest association with EFS were tumor volume, patient weight, and histology. On the basis of regression modeling, tumor volume and patient weight were superior predictors of outcome compared with greatest tumor dimension and patient age in children with intermediate-risk RMS. The current results indicated that the prognostic performance of tumor volume and patient weight should be assessed in an independent prospective study.
本研究的目的是比较肿瘤体积和患者体重与传统肿瘤大小因素（最大径）和患者年龄，并确定哪些参数能最好地区分患有中危横纹肌肉瘤（RMS）的儿科患者的结局。
共有 370 名无转移 RMS 患儿完整信息可用于 COG 中危研究 D9803（1999-2005 年）。采用 Kaplan-Meier 法估计生存分布。递归分区模型用于确定与无事件生存（EFS）相关的预后因素。Cox 比例风险回归模型用于估计患者特征与失败或死亡风险之间的关系。
对于所有患有中危 RMS 的患者，EFS 的递归分区算法提示，最佳预后组应通过肿瘤体积（以 20 cm3 为转折点）、患者体重（以 50 kg 为转折点）和胚胎组织学来定义。肿瘤体积和患者体重为标准预后因素增加了显著的预后信息，包括最大肿瘤直径和患者年龄（P =.02）。完全切除肿瘤的能力与患者的大小没有显著相关，而在调整标准危险因素后，患者体重并未显著改变肿瘤体积与 EFS 之间的关系（P =.2）。
与 EFS 相关性最强的因素是肿瘤体积、患者体重和组织学。基于回归建模，与最大肿瘤直径和患者年龄相比，肿瘤体积和患者体重是中危 RMS 儿童预后的更好预测指标。目前的结果表明，应在独立的前瞻性研究中评估肿瘤体积和患者体重的预后性能。
