BIOLOGY AND TREATMENT OF PEDIATRIC SARCOMA

儿科肉瘤的研究正在两个领域进行：生物学研究 和治疗试验。 生物学研究包括：（1）体外研究 组织培养评价和细胞系的表征 这些肿瘤;（2）来自这些肿瘤的细胞系的体外分化 肿瘤;（3）这些肿瘤的细胞遗传学特征。 治疗 研究试图：（1）改善新诊断患者的治疗 通过强化诱导治疗， 联合治疗包括自体骨髓移植;（2） 开发治疗肉瘤患者的新疗法， 复发患者化疗药物的II期研究。 以来 在这个项目开始的时候，我们已经证明了尤文氏肉瘤 外周神经上皮瘤细胞系具有易位- t（11.22）- 和相似的癌基因表达模式。 我们还证明 随着分化，尤文肉瘤细胞系形成了一个神经元， 表型 自1983年以来，我们已经治疗了70多名新的 确诊为高危肉瘤 这个83-C-73的完全缓解率 方案中的缓解率为93%，约50%的患者仍处于缓解状态。 这些数字代表了在此期间使用的先前方案的改善。 机构。 基于之前的儿科分支II期试验， 治疗复发性肉瘤， 我们于1985年6月开始了一项使用异环磷酰胺的试验， 依托泊苷与美司钠尿保护。 我们现在已经证明， 联合用药在治疗复发性 儿童和年轻人的肉瘤。 到目前为止，有25到49名患者 目前可评价的患者有完全或部分缓解。 这些结果 表明异环磷酰胺和依托泊苷的组合将是一种 在儿科恶性肿瘤治疗中的重要治疗组合。

The study of pediatric sarcomas is ongoing in two areas: biological studies and therapeutic trials. The biological studies include: (1) in vitro tissue culture evaluation and characterization of cell lines derived from these tumors; (2) in vitro differentiation of cell lines derived from these tumors; (3) cytogenetic characterization of these tumors. The therapeutic studies are attempting: (1) to improve therapy for newly diagnosed patients by intensifying the induction therapy and replacing maintenance with a conslidation therapy including autologous bone marrow transplantation; (2) to develop new therapies for treatment of patients with sarcomas, using Phase II studies of chemotherapeutic agents in relapsed patients. Since the inception of this program, we have demonstrated that Ewing's sarcoma and peripheral neuroepithelioma cell lines have translocation - t (11.22) - and a similar pattern of oncogene expression. We have also demonstrated that with differentiation, the Ewing's sarcoma cell lines develop a nerual phenotype. Since 1983, we have treated over 70 patients with newly diagnosed high risk sarcomas. The complete remission rate on this 83-C-73 protocol is 93% and approximately 50% of the patients remain in remission. These figures represent improvement over previous regimen utilized at this institution. Based on a previous Pediatric Branch Phase II Trial for the treatment of recurrent sarcomas that demonstrated the activity of Ifosfamide, we began a trial in June 1985 utilizing Ifosfamide and Etoposide with Mesna uroprotection. We have now demonsrated that this combination has a high degree of activity in the treatment of recurrent sarcomas in children and young adults. To date, 25 to 49 patients who are presently evaluable have had a complete or partial response. These results suggest that the combination of Ifosfamide and Etoposide will be an important therapeutic combination in the treatment of pediatric malignancy.