Natural history and management of low-grade glioma in NF-1 children
Natural history and management of low-grade glioma in NF-1 children
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DOI：
10.1007/s11060-010-0159-z
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发表时间：
2010-11-01
影响因子：
3.9
通讯作者：
Gnekow, Astrid K.
中科院分区：
文献类型：
--
作者：
Driever, Pablo Hernaiz;von Hornstein, Stephan;Gnekow, Astrid K.
儿童1型神经纤维瘤病(NF-1)患者易发生低级别胶质瘤(LGG)。1996年的HIT-LGG研究旨在观察儿童LGG的自然病史，并在需要非手术治疗的情况下推迟使用卡铂和长春新碱对较小儿童的照射。在1044名方案患者中，共有109名(10.4%)具有遗传的核因子-1特征[57名女性患者；中位年龄5.1岁(范围1-15.4岁)]。83名患者(76%)患有视路肿瘤。神经成像只对67名患者进行了诊断。组织学检查显示42例活检患者中有38例为WHO I级毛细胞性星形细胞瘤。65例(60%)患者接受非手术治疗，包括化疗(n=55)或放疗(n=10)。中位观察时间为5.25年的总存活率为96%，而5年的无事件存活率(EFS)为0.24。甚至在11岁以上的儿童中也观察到进行性LGG。交叉/交叉后定位是进行性疾病的单变量危险因素。在化疗组，我们观察到5年无进展生存率(PFS)为0.73。照射组的PFS率为0.78。多变量分析显示手术干预和视路内的定位是增加肿瘤进展风险的因素。在这项大型的前瞻性多国研究中，在75%的患者中，NF-1患者的LGG确实有进展。化疗产生了可接受的PFS。决定进展的生物学因素仍然知之甚少。
Pediatric neurofibromatosis type 1 (NF-1) patients are prone to developing low-grade glioma (LGG). The HIT-LGG study 1996 aimed to observe the natural history of pediatric LGG and to postpone irradiation in younger children by using carboplatinum and vincristine in case non-surgical treatment was required. A total of 109 of 1,044 (10.4%) protocol patients had a genetic NF-1 trait [57 female patients; median age 5.1 years (range 1-15.4 years)]. Eighty-three patients (76%) suffered from an optic pathway tumor. Neuroimaging only allowed diagnosis in 67 patients. Histology revealed pilocytic astrocytoma WHO grade I in 38 of 42 biopsied patients. Sixty-five (60%) patients received non-surgical treatment, either chemotherapy (n = 55) or irradiation (n = 10). The overall survival rate of 96% after a median observation time of 5.25 years contrasts with an event free survival rate (EFS) of 0.24 at 5 years. Progressive LGG were observed even in children older than 11 years. Chiasmatic/postchiasmatic localization was a univariate risk factor for progressive disease. In the chemotherapy group, we observed a 5-year progression-free survival (PFS) rate of 0.73. Similarly, the PFS rate in the irradiation group was 0.78. Multivariate analysis revealed surgical intervention and localization within the optic pathway as factors that increased the risk of tumor progression. In this large prospective multinational study, LGG in NF-1 patients did progress in 75% of patients. Chemotherapy yielded acceptable PFS. The biological factors determining progression remain poorly understood.