Rhabdomyosarcoma: New windows of opportunity
Rhabdomyosarcoma: New windows of opportunity
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DOI：
10.1634/theoncologist.10-7-518
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发表时间：
2005-08-01
期刊：
影响因子：
5.8
通讯作者：
Meyer, WH
中科院分区：
文献类型：
--
作者：
Breitfeld, PP;Meyer, WH
横纹肌肉瘤是一种高度恶性的蓝色小细胞肿瘤，以肌肉分化为特征。通过现代治疗，超过70%的儿童和青少年患有这种疾病被治愈。适当的活检以获得足够的组织用于准确诊断和分子表征是至关重要的。必须评估患者的肿瘤范围;组间横纹肌肉瘤研究（IRS）临床分组和分期系统在北美普遍应用。多学科治疗是必要的，以最大限度地提高治愈率。局部控制依赖于完全手术切除，如果可能的话;那些肿瘤没有完全切除和那些与肺泡组织学肿瘤需要局部照射，以最大限度地局部控制。在北美，长春新碱（Oncovin（R）; Eli Lilly and Company，Indianapolis，http://www.lilly。com）、放线菌素D（Cosmegen（R）; Merck & Co.，股份有限公司、怀特豪斯站，NJ，http://www.merck.com）和环磷酰胺是标准的化疗剂。IRS使用治疗窗研究来确认临床前异种移植模型的预测性质，并确定几种新的单一药物和在高风险患者群体中具有活性的药物组合。尽管做出了这些努力，但这些高危患者的结局仍然很差。下一代儿童肿瘤学研究将评估拓扑异构酶I抑制剂和剂量压缩治疗方法的疗效。肿瘤分子表征的新进展，包括基因表达分析，可以确定新的治疗靶点，这些靶点可以通过扩大临床前药物发现工作来利用，并有望彻底改变基于风险的治疗。
Rhabdomyosarcoma is a highly malignant, small blue cell tumor characterized by muscle differentiation. With modern treatment, more than 70% of children and adolescents with this disease are cured. Adequate biopsy to obtain sufficient tissue for accurate diagnosis and molecular characterization is critical. Patients must be assessed for tumor extent; the Intergroup Rhabdomyosarcoma Study (IRS) clinical group and Staging system is universally applied in North America. Multidisciplinary therapy is necessary to maximize cure rates. Local control relies on complete surgical excision when possible; those whose tumors are not completely excised and those with alveolar histology tumors require local irradiation to maximize local control. In North America, vincristine (Oncovin (R); Eli Lilly and Company, Indianapolis, http://www.lilly. com), dactinomycin (Cosmegen (R); Merck & Co., Inc., Whitehouse Station, NJ, http://www.merck.com), and cyclophosphamide are the standard chemotherapy agents. The IRS has used therapeutic window studies to confirm the predictive nature of preclinical xenograft models and to identify several new single agents and combinations of agents with activity in high-risk patient groups. Despite these efforts, the outcome for these high-risk patients remains poor. The next generation of Children's Oncology Group studies will evaluate the efficacy of topoisomerase-I inhibitors and dose-compression therapy approaches. New advances in molecular characterization of tumors, including gene-expression analysis, may identify new therapeutic targets that can be exploited by expanded preclinical drug discovery efforts, and hold the promise of revolutionizing risk-based therapies.