High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors
High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors
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DOI：
10.1002/cncr.23305
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发表时间：
2008-03-15
期刊：
影响因子：
6.2
通讯作者：
Fouladi, Maryam
中科院分区：
文献类型：
--
作者：
Shih, Chie-Schin;Hale, Gregory A.;Fouladi, Maryam
背景资料。方法回顾分析~2004年间圣犹大儿童研究医院收治的27例儿童复发性中枢神经系统肿瘤患者的临床资料。结果：初诊年龄中位数为4.5岁(0.4~16.6岁)，ASCR中位数为6.7岁(1.1~18.5岁)。其中髓母细胞瘤13例，原始神经外胚层瘤3例，松果体母细胞瘤2例，不典型畸胎样横纹肌瘤2例，室管膜瘤3例，间变性星形细胞瘤2例，多形性胶质母细胞瘤2例。5年总生存率和无进展生存率分别为28.2%和18.5%。初诊时年龄<3岁的患者的5年PFS率(57.1%)明显好于年长患者(5.0%)(P=.019)。在6名长期幸存者中(5名确诊时患有MO病，1名确诊时患有M3病)，5名患者同时接受了放射治疗和高密度CT检查，作为抢救方案的一部分；4名患者确诊时年龄为3岁，作为一线治疗的一部分仅接受过化疗。两名患者死于移植相关的毒性反应；44%的患者经历了3级或4级的移植相关毒性反应(毒性根据国家癌症研究所共同毒性标准进行分级)。结论：对于患有复发的中枢神经系统恶性肿瘤的大龄儿童，HDCT联合ASCR不是一种有效的抢救策略。年轻人群中显著较好的结果很可能与使用放射治疗作为抢救策略的一部分有关。
BACKGROUND. High-dose chemotherapy (HDCT) with autologous stem cell rescue (ASCR) has been reported to be effective in treating children with recurrent central nervous system (CNS) malignancies.METHODS. To evaluate the efficacy and toxicities of HDCT and ASCR, the medical records of 27 children with recurrent CNS malignancies who received such therapy at St. Jude Children's Research Hospital between 1989 and 2004 were reviewed.RESULTS. The median age at diagnosis was 4.5 years (range, 0.4-16.6 years) and that at ASCR was 6.7 years (range, 1.1-18.5 years). Diagnoses included medulloblastoma (13 patients), primitive neuroectodermal tumor (3 patients), pineoblastoma (2 patients), atypical teratoid rhabdoid tumor (2 patients), ependymoma (3 patients), anaplastic astrocytoma (2 patients), and glioblastoma multiforme (2 patients). The 5-year overall and progression-free survival (PFS) rates were 28.2% and 18.5%, respectively. The 5-year PFS rate for patients aged < 3 years at diagnosis (57.1%) was significantly better than older patients (5.0%) (P = .019). Among the 6 long-term survivors (5 with MO disease and I with M3 disease at diagnosis), 5 received both radiotherapy and HDCT as part of their salvage regimen; 4 were aged < 3 years at diagnosis and had received chemotherapy only as part of frontline therapy. Two patients died of transplant-related toxicities; 44% experienced grade 3 or 4 transplant- related toxicities (toxicities were graded according to the National Cancer Institute Common Toxicity Criteria).CONCLUSIONS. HDCT with ASCR is not an effective salvage strategy for older children with recurrent CNS malignancies. The significantly better outcome in the younger cohort was most likely related to the use of radiotherapy as part of the salvage strategy.