Outcomes in children with central nervous system tumors disseminated at presentation: a large single-center experience

Purpose 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. Methods 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. Results 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. Conclusions Dissemination status at initial presentation significantly affects outcomes in children with CNS tumors.