Wolff J E, Mölenkamp G, Westphal S, Pietsch T, Gnekow A, Kortmann R D, Kuehl J
University of Calgary, c/o Alberta Children's Hospital, Alberta, Canada.
Cancer. 2000 Nov 15;89(10):2131-7. doi: 10.1002/1097-0142(20001115)89:10<2131::aid-cncr14>3.0.co;2-j.
Glioblastoma multiforme in childhood is rare, and the prognosis for patients with the disease is poor. The Pediatric Oncology Society of the Germanic language group (GPOH) enrolls patients in a series of pilot trials, the first of which is reported here (HIT-GBM-A).
Twenty-two patients with glioblastoma multiforme, World Health Organization Grade 4, between the ages of 3-15 years (45% male) were enrolled during the period 1995-1997. There were 13 supratentorial tumors, 8 brainstem tumors, and 1 cerebellar tumor. The patients underwent the following procedures: stereotactic biopsy (n = 3 patients), open biopsy (n = 1 patient), partial resection (n = 6 patients), subtotal resection (n = 4 patients), and macroscopic total resection (n = 8 patients). Adjuvant treatment consisted of oral chemotherapy with trofosfamide, 100 mg/m(2), and etoposide, 25 mg/m(2), daily or for 21-day cycles interrupted by 1-week rests. Standard fractionated radiation (54 grays) was started concurrently with the first cycle.
The chemotherapy was well tolerated, with no treatment-related deaths and only minor side effects. The responses in 12 evaluable patients after two cycles were as follows: 1 complete response, 1 partial response, 3 patients with stable disease, and 7 patients with progressive disease. The median overall survival was 12 months. The 1-year, 2-year, and 4-year overall survival rates were 52%, 26%, and 22%, respectively, and the event free survival rates were 26%, 22%, and 4%, respectively. None of the four surviving patients (3.2 years, 3.4 years, 4.0 years, and 4.2 years after diagnosis) is event free. Two patients are alive after tumor progression: One patient was diagnosed with a medulloblastoma, and one patient was diagnosed with an osteosarcoma as second malignancies. A control group extracted from the Surveillance, Epidemiology, and End Results data had lower survival rates: the difference between the groups was not statistically significant (P = 0.26).
This chemotherapy will not be used in a randomized trial of patients with glioblastoma; however, it may be evaluated for patients with tumors that have more chemoresponsive histologies.
儿童多形性胶质母细胞瘤罕见，该病患者预后较差。日耳曼语系儿童肿瘤学会（GPOH）让患者参与了一系列试点试验，本文报告的是首个试验（HIT-GBM-A）。
1995年至1997年期间，纳入了22例3至15岁的世界卫生组织4级多形性胶质母细胞瘤患者（45%为男性）。有13例幕上肿瘤、8例脑干肿瘤和1例小脑肿瘤。患者接受了以下治疗：立体定向活检（3例患者）、开放活检（1例患者）、部分切除（6例患者）、次全切除（4例患者）和肉眼全切（8例患者）。辅助治疗包括口服化疗，曲磷胺100mg/m²，依托泊苷25mg/m²，每日一次或每21天为一个周期，期间休息1周。标准分割放疗（54戈瑞）与第一个周期同时开始。
化疗耐受性良好，无治疗相关死亡，仅有轻微副作用。12例可评估患者在两个周期后的反应如下：1例完全缓解，1例部分缓解，3例病情稳定，7例病情进展。中位总生存期为12个月。1年、2年和4年总生存率分别为52%、26%和22%，无事件生存率分别为26%、22%和4%。四名存活患者（诊断后3.2年、3.4年、4.0年和4.2年）均未无事件生存。两名患者在肿瘤进展后仍存活：一名患者被诊断为髓母细胞瘤，一名患者被诊断为骨肉瘤作为第二原发恶性肿瘤。从监测、流行病学和最终结果数据中提取的对照组生存率较低：两组之间差异无统计学意义（P = 0.26）。
这种化疗方案不会用于胶质母细胞瘤患者的随机试验；然而，对于具有更多化疗反应性组织学类型的肿瘤患者，可能需要对其进行评估。
