Neuroblastoma metastatic to the central nervous system: Survival analyses from the German Childhood Cancer Registry and the literature.

10555 Background: Therapeutic innovation has resulted in an overall decline in childhood neuroblastoma (NB) mortality; however, metastatic NB to the central nervous system (CNS NB), which has emerged as a sanctuary site for NB metastases, remains difficult to treat and is typically fatal. The objective of this study was to describe the natural course of CNS NB. Methods: Data were sourced from a custom query of the German Childhood Cancer Registry (GCCR) and from the literature. Survival statistics were prepared from a diverse, thus generalizable, pool of CNS NB patients. Data are presented as secondary event-free and overall survival (EFS and OS) after diagnosis of first CNS recurrence from initial high-risk NB. Results: The GCCR query identified 85 patients with CNS NB diagnosed from 1990–2010, including 57 with isolated CNS disease. The median (95% confidence) EFS and OS times were 2.6 (1.5–3.8) and 4.7 (2.1–7.2) months, respectively, for all CNS NB patients, and 2.8 (1.4–4.1) and 6.8 (2.1–11.5) months, respectively, for isolated CNS NB patients. Secondary OS at 12, 18, and 36 months was 29.4%, 18.8%, and 8.2%, respectively, for all CNS NB patients, and 35.1%, 22.8%, and 12.3%, respectively, for isolated CNS NB patients. Thirteen publications were selected with 83 patients treated from 1979–2013. In addition to an inclusive analysis, a restricted analysis was performed, excluding patients who did not receive therapy with curative intent, to assess survival after therapeutic intervention. Median OS (95% confidence) was 5.6 (3.0–8.0) and 8.7 (5.8–11.0) months in the inclusive and restricted analyses, respectively. The proportion of patients surviving 12, 18, and 36 months at reporting were 24%, 12%, and 3.6%, respectively, for the inclusive population, and 33%, 17%, and 5%, respectively, for the restricted population. Conclusions: An assessment of the natural course of CNS NB from two sources arrived at similar conclusions with respect to overall and long-term survival. In general, median secondary OS is < 6 months and < 10% of patients survive 36 months. The findings were consistent across geographic regions and have not changed appreciably in 4 decades.