Meyers Paul A
Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA.
Curr Opin Oncol. 2004 Mar;16(2):120-5. doi: 10.1097/00001622-200403000-00006.
The principal pediatric sarcomas are Ewing sarcoma, rhabdomyosarcoma, and osteosarcoma. While the prognosis for these diseases is generally favorable, it is possible to identify groups of patients with each diagnosis whose prognosis remains unacceptably poor. Based on the chemotherapy sensitivity of these tumors, many investigators have suggested that consolidation with high dose chemotherapy with or without radiation therapy followed by stem cell rescue could improve the prognosis for these high risk patients. This review evaluates the results of high dose therapy with stem cell reconstitution used to consolidate treatment of high risk patients with sarcomas.
The majority of the trials which have been conducted to assess this hypothesis have treated relatively small numbers of patients. They do not use consistent definitions of high risk. They have used different regimens for cytoreduction. They have used a variety of sources for stem cells. In many series, the consolidation therapy is applied only to patients who have achieved a significant response to prior therapy and the results have then been compared to a cohort of patients with similar presenting features, not all of whom responded to initial therapy.
The published results do not demonstrate an unequivocal benefit for consolidation with high-dose therapy. Future trials of high-dose therapy must define rigorous eligibility criteria, must have an appropriate, preferably randomized, control group, and must be designed with sufficient power to evaluate the hypothesis.
主要的儿童肉瘤包括尤因肉瘤、横纹肌肉瘤和骨肉瘤。虽然这些疾病的总体预后较好，但对于每种诊断的患者群体而言，仍有可能识别出预后差到无法接受的患者。基于这些肿瘤对化疗的敏感性，许多研究者提出，采用含或不含放射治疗的高剂量化疗进行巩固治疗，随后进行干细胞救援，可能会改善这些高危患者的预后。本综述评估了采用干细胞重建的高剂量治疗用于巩固高危肉瘤患者治疗的结果。
为评估这一假设而进行的大多数试验所治疗的患者数量相对较少。它们对高危没有统一的定义。它们采用了不同的细胞减灭方案。它们使用了多种干细胞来源。在许多系列研究中，巩固治疗仅应用于对先前治疗有显著反应的患者，然后将结果与一组具有相似表现特征的患者进行比较，而这些患者并非都对初始治疗有反应。
已发表的结果并未表明高剂量治疗巩固有明确益处。未来的高剂量治疗试验必须定义严格的入选标准，必须有一个合适的、最好是随机的对照组，并且必须设计有足够的效力来评估该假设。
