Tandem high-dose therapy in rapid sequence for children with high-risk neuroblastoma
Tandem high-dose therapy in rapid sequence for children with high-risk neuroblastoma
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DOI：
10.1200/jco.2000.18.13.2567
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发表时间：
2000-07-01
影响因子：
45.3
通讯作者：
Diller, L
中科院分区：
文献类型：
--
作者：
Grupp, SA;Stern, JW;Diller, L
目的：化疗和支持性治疗的进展缓慢地提高了高危神经母细胞瘤患者的存活率。许多化疗进展的焦点一直是剂量强化。在这项涉及晚期神经母细胞瘤儿童的II期试验中，我们采用了诱导化疗后大剂量、清髓治疗(高剂量治疗)和干细胞拯救(HDT/SCR)的快速顺序。患者和方法：患者接受诱导化疗，在此期间采集外周血干细胞和祖细胞并采取局部控制措施。患者随后接受HDT/SCR串联疗程，间隔4至6周。39例患者(年龄1-12岁)可评估，完成了70个周期的HDT/SCR。结果：所有患者尽管年龄较小，但仍有可能发生分离，平均每个周期可支持7.2×10(6)CD34(+)细胞/公斤。植入迅速；中性粒细胞植入的中位时间为11天。完成第一个HDT疗程的四名患者没有完成第二个疗程，有三名患者因毒性死亡。中位随访时间为22个月(从诊断开始)，39名患者中有26名仍无事件发生。这些患者的3年无事件生存率为58%。结论：HDT/SCR串联方案治疗高危神经母细胞瘤是一种可行的治疗策略，可能会提高无病生存率。J Clin Oncol18：2567-2575。(C)2000年，由美国临床肿瘤学会提供。
Purpose: Advances in chemotherapy and supportive care have slowly improved survival rates for patients with high-risk neuroblastoma. The focus of many of these chemotherapeutic advances has been dose intensification. In this phase II trial involving children with advanced neuroblastoma, we used a program of induction chemotherapy followed by tandem high-dose, myeloablative treatments (high-dose therapy) with stem-cell rescue (HDT/SCR) in rapid sequence.Patients and Methods: Patients underwent induction chemotherapy during which peripheral-blood stem and progenitor cells were collected and local control measures undertaken. Patients then received tandem courses of HDT/SCR, 4 to 6 weeks apart. Thirty-nine patients (age 1 to 12 years) were assessable, and 70 cycles of HDT/SCR were completed.Results: Pheresis wets possible in the case of all patients, despite their young ages, with an average of 7.2 x 10(6) CD34(+) cells/kg available to support each cycle. Engraftment was rapid; median time to neutrophil engraftment was 11 days. Four patients who completed the first HDT course did not complete the second, and there were three deaths due to toxicity. With a median follow-up of 22 months (from diagnosis), 26 of 39 patients remained event-free. The 3-year event-free survival rate for these patients was 58%.Conclusion: A tandem HDT/SCR regimen for highrisk neuroblastoma is a feasible treatment strategy for children and may improve disease-free survival. J Clin Oncol 18:2567-2575. (C) 2000 by American Society of Clinical Oncology.