Verrill M W, Judson I R, Wiltshaw E, Thomas J M, Harmer C L, Fisher C
Sarcoma Unit, Royal Marsden NHS Trust, London, UK.
Ann Oncol. 1997 Nov;8(11):1099-105. doi: 10.1023/a:1008264902857.
Ewing's sarcoma and primitive neuroectodermal tumour (ES/PNET) are rare, limiting opportunities for therapy studies in adults. Chemotherapy regimens adapted from paediatric studies are often used for adults but concerns about poor outcome and treatment toxicity may adversely affect drug dose intensity. We present our experience using a paediatric protocol at full dose.
Records of 34 patients with ES/PNET who received the IVAD chemotherapy regimens were reviewed. Received drug dose intensity, toxicity and survival data were collected.
Received dose intensity in 30 evaluable patients was 0.92 compared to the standard IVAD schedule. Myelosuppression was the major toxicity, 83% of patients experienced grade 4 neutropenia. There was no major renal or cardiac toxicity. In patients without metastases at presentation, five-year overall survival was 63% and progression free survival was 39%. Tumour burden at presentation was statistically significantly associated with survival (P = 0.002). The five-year survival rate of 80% in patients presenting with low volume non metastatic disease was equivalent to published paediatric series.
Although the IVAD chemotherapy regimens are myelotoxic in adults, they can be given safely. We recommend that adults with ES/PNET should be included in current multicentre, multidisciplinary treatment studies directed at children.
尤因肉瘤和原始神经外胚层肿瘤（ES/PNET）较为罕见，限制了成人治疗研究的机会。源自儿科研究的化疗方案常被用于成人，但对疗效不佳和治疗毒性的担忧可能会对药物剂量强度产生不利影响。我们介绍了我们以全剂量使用儿科方案的经验。
回顾了34例接受IVAD化疗方案的ES/PNET患者的记录。收集了接受的药物剂量强度、毒性和生存数据。
30例可评估患者的接受剂量强度与标准IVAD方案相比为0.92。骨髓抑制是主要毒性，83%的患者出现4级中性粒细胞减少。未出现严重的肾脏或心脏毒性。初诊时无转移的患者，五年总生存率为63%，无进展生存率为39%。初诊时的肿瘤负荷与生存具有统计学显著相关性（P = 0.002）。低负荷非转移性疾病患者80%的五年生存率与已发表的儿科系列相当。
尽管IVAD化疗方案在成人中具有骨髓毒性，但可以安全给药。我们建议，患有ES/PNET的成人应纳入当前针对儿童的多中心、多学科治疗研究。
