Prognostic factors and clinical outcomes in children and adolescents with metastatic rhabdomyosarcoma - A report from the intergroup rhabdomyosarcoma study IV
Prognostic factors and clinical outcomes in children and adolescents with metastatic rhabdomyosarcoma - A report from the intergroup rhabdomyosarcoma study IV
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DOI：
10.1200/jco.2003.06.129
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发表时间：
2003-01-01
影响因子：
45.3
通讯作者：
Crist, WM
中科院分区：
文献类型：
--
作者：
Breneman, JC;Lyden, E;Crist, WM
目的：确定与第四次组间横纹肌肉瘤研究 (IRS-IV) 治疗的转移性横纹肌肉瘤 (RMS) 儿童结局相关的危险因素。患者和方法：转移性横纹肌肉瘤患者接受两种方案之一治疗，其中包括异环磷酰胺和依托泊苷 (IE) 与长春新碱、放线菌素和环磷酰胺 (VAC) 或长春新碱、美法仑的窗口期(VM) 和 VAC。研究终点是无失败生存期（FFS）和总生存期（OS）。包括年龄、组织学、原发和转移性疾病部位以及转移性疾病部位数量在内的临床因素与这些终点相关。结果：127 名患者符合分析条件。所有患者的估计 3 年 OS 和 FFS 分别为 39% 和 25%。通过单变量分析，3 年 OS 受到组织学（胚胎为 47%，所有其他为 34%，P = .026）和转移部位数量增加（P = .028）的显着影响。通过多变量分析，存在两个或更少的转移部位是唯一显着的预测因素（分别为 P = .007 和 .006）。具有两个或更少转移部位的胚胎组织学组合确定了一个亚组，其 3 年 FFS 为 40%，OS 为 47%。 结论：接受 IRS-IV 研究治疗的 IV 组 RMS 儿童如果具有两个或更少转移部位和胚胎组织学，则 OS 和 FFS 有所改善。这一有利的患者子集的结果接近于选定的患有局部非转移性疾病的患者中观察到的结果。因此，这些患者可能不适合接受包含具有显着毒性或未经证实的抗肿瘤活性的实验药物的治疗方案。 (C) 2003 年，美国临床肿瘤学会。
Purpose : To identify risk factors associated with outcomes in children with metastatic rhabdomyosarcoma (RMS) treated on the fourth Intergroup Rhabdomyosarcoma Study (IRS-IV).Patients and Methods: Patients with metastatic RMS were treated with one of two regimens that incorporated a window of either ifosfamide and etoposide (IE) with vincristine, dactinomycin, and cyclophosphamide (VAC) or vincristine, melphalan (VM) and VAC. Study end points were failure-free survival (FFS) and overall survival (OS). Clinical factors including age, histology, sites of primary and metastatic disease, and number of sites of metastatic disease were correlated with those end points.Results: One hundred twenty-seven patients were eligible for analysis. The estimated 3-year OS and FFS for all patients were 39% and 25%, respectively. By univariate analysis, 3-year OS was significantly influenced by histology (47% for embryonal v 34% for all others, P = .026) and increasing number of metastatic sites (P = .028). By multivariate analysis, the presence of two or fewer metastatic sites was the only significant predictor (P = .007 and.006, respectively). The combination of embryonal histology with two or fewer metastatic sites identified a subgroup with 3-year FFS of 40% and OS of 47%.Conclusion: Children with group IV RMS treated on the IRS-IV study had improved OS and FFS if they had two or fewer metastatic sites and embryonal histology. This favorable subset of patients has outcomes approaching those observed in selected patients with localized, nonmetastatic disease. Thus, these patients might not be appropriate candidates for regimens that include experimental agents with substantial toxicities or unproven antitumor activity. (C) 2003 by American Society of Clinical Oncology.