Gururangan Sridharan, Fisher Michael J, Allen Jeffrey C, Herndon James E, Quinn Jennifer A, Reardon David A, Vredenburgh James J, Desjardins Annick, Phillips Peter C, Watral Melody A, Krauser Jeanne M, Friedman Allan H, Friedman Henry S
Preston Robert Tisch Brain Tumor Center, Duke University Medical Center, Box 3624, Durham, NC 27710, USA.
Neuro Oncol. 2007 Apr;9(2):161-8. doi: 10.1215/15228517-2006-030. Epub 2007 Mar 8.
We conducted a phase II study to assess the efficacy of oral temozolomide (TMZ) in children with progressive low-grade glioma. Thirty eligible patients were enrolled on this study. Median age at enrollment was 10 years (range, 4-18 years). Eligible patients received TMZ (200 mg/m(2) per day) by mouth for five days every four weeks. Patients received a median of nine cycles (range, 2-12 cycles) of treatment. Best responses in the 26 patients (86%) with optic pathway glioma (OPG)/pilocytic astrocytoma (PA) included partial response in 3 patients (11%), minor response in 1 (4%), stable disease in 10 (38%), and progressive disease in 12 (46%). Only one of four patients with fibrillary astrocytoma had stable disease for 29 months after TMZ. The overall disease stabilization rate in patients with OPG/PA was 54%, and disease control was maintained for a median interval of 34 months. Seventeen of 26 patients had progressive disease either on or off therapy, and three have died of disease. The two-year progression-free and overall survivals in patients with OPG/PA were 49% (95% CI, 30%-67%) and 96% (95% CI, 89%-100%), respectively. Worst toxicity related to TMZ in all 30 patients included grade 2-4 thrombocytopenia in seven patients, grade 2-4 neutropenia in seven, grade 2 skin rash in one, and intratumor hemorrhage in one. TMZ given in this schedule was successful in stabilizing disease in a significant proportion of the patients with OPG/PA, with manageable toxicity.
我们开展了一项II期研究，以评估口服替莫唑胺（TMZ）治疗进展性低级别胶质瘤患儿的疗效。30例符合条件的患者入组本研究。入组时的中位年龄为10岁（范围4 - 18岁）。符合条件的患者每四周口服TMZ（200 mg/m²/天），持续五天。患者接受的治疗周期中位数为9个周期（范围2 - 12个周期）。26例患有视路胶质瘤（OPG）/毛细胞型星形细胞瘤（PA）的患者（86%）的最佳反应包括3例（11%）部分缓解、1例（4%）轻微反应、10例（38%）疾病稳定和12例（46%）疾病进展。4例纤维型星形细胞瘤患者中只有1例在接受TMZ治疗后疾病稳定了29个月。OPG/PA患者的总体疾病稳定率为54%，疾病控制维持的中位时间为34个月。26例患者中有17例在治疗期间或治疗后出现疾病进展，3例死于疾病。OPG/PA患者的两年无进展生存率和总生存率分别为49%（95%CI，30% - 67%）和96%（95%CI，89% - 100%）。所有30例患者中与TMZ相关的最严重毒性包括7例2 - 4级血小板减少、7例2 - 4级中性粒细胞减少、1例2级皮疹和1例肿瘤内出血。按此方案给予TMZ成功地使相当一部分OPG/PA患者的疾病得到稳定，且毒性可控。
