Kirkman Matthew A, Hayward Richard, Phipps Kim, Aquilina Kristian
Department of Neurosurgery, Great Ormond Street Hospital for Children NHS Trust, London, WC1N 3JH, UK.
Victor Horsley Department of Neurosurgery, The National Hospital for Neurology and Neurosurgery, Queen Square, London, UK.
Childs Nerv Syst. 2018 Nov;34(11):2259-2267. doi: 10.1007/s00381-018-3871-1. Epub 2018 Jun 24.
Children with disseminated central nervous system (CNS) tumors have worse outcomes than those with solitary disease, but outcomes of disease dissemination at initial presentation have not been systematically studied and compared across tumor groups to date. We evaluated the impact of tumor dissemination at presentation on management and clinical outcomes in a cohort of consecutively treated children in a single neurosurgical unit over a 14-year period.
Method used was a retrospective review of data on children presenting to Great Ormond Street Hospital, London, UK, with medulloblastoma, primitive neuroectodermal tumor, atypical teratoid rhabdoid tumor, pilocytic astrocytoma, and ependymoma between 2003 and 2016 inclusive. Uni- and multi-variate analyses were performed to evaluate a range of outcome measures.
Three-hundred sixty-one children were identified in total, 53 with disease dissemination at presentation (M:F = 34:19, median age = 3.8 years, range = 7 days-15.6 years) and 308 with solitary tumors (M:F = 161:147, median age = 5.8 years, range = 1 day-16.9 years). Median follow-up was similar irrespective of dissemination status (disseminated tumor 64.0 months, range = 5.2-152.0 months; solitary tumor 74.5 months, range = 4.7-170.1 months; P > 0.05). In multivariate analyses, tumor type and dissemination status at presentation were significantly associated with overall survival (P < 0.0001), risk of recurrence/disease progression (P < 0.01), and event-free survival (P < 0.0001). Subtotal resection was associated with shorter time to recurrence/disease progression (P < 0.01) and worse event-free (P < 0.0001) but not overall survival, whereas treatment with chemotherapy and radiotherapy were associated with improved overall (Ps < 0.0001) and event-free survival (Ps < 0.05). Differences between tumor groups were evident.
Dissemination status at initial presentation significantly affects outcomes in children with CNS tumors.
弥漫性中枢神经系统（CNS）肿瘤患儿的预后比单发肿瘤患儿更差，但疾病在初次就诊时的播散情况及其预后尚未得到系统研究，且至今未在不同肿瘤组间进行比较。我们评估了在14年期间于单一神经外科连续治疗的一组患儿中，初次就诊时肿瘤播散对治疗及临床预后的影响。
采用回顾性研究方法，分析2003年至2016年期间（含）就诊于英国伦敦大奥蒙德街医院的髓母细胞瘤、原始神经外胚层肿瘤、非典型畸胎样横纹肌样肿瘤、毛细胞型星形细胞瘤和室管膜瘤患儿的数据。进行单因素和多因素分析以评估一系列预后指标。
共纳入361例患儿，其中53例初次就诊时存在疾病播散（男∶女 = 34∶19，中位年龄 = 3.8岁，范围 = 7天至15.6岁），308例为单发肿瘤（男∶女 = 161∶147，中位年龄 = 5.8岁，范围 = 1天至16.9岁）。无论播散状态如何，中位随访时间相似（播散性肿瘤64.0个月，范围 = 5.2至152.0个月；单发肿瘤74.5个月，范围 = 4.7至170.1个月；P > 0.05）。在多因素分析中，肿瘤类型和初次就诊时的播散状态与总生存期（P < 0.0001）、复发/疾病进展风险（P < 0.01）和无事件生存期（P < 0.0001）显著相关。次全切除与复发/疾病进展时间较短（P < 0.01）和无事件生存期较差（P < 0.0001）相关，但与总生存期无关，而化疗和放疗与总生存期改善（P < 0.0001）和无事件生存期改善（P < 0.05）相关。不同肿瘤组之间的差异明显。
初次就诊时的播散状态显著影响中枢神经系统肿瘤患儿的预后。
