Prognostic Impact of Treatment Modalities, Including Targeted Compartmental Radio-Immunotherapy, in a Cohort of Neuroblastoma Patients With CNS Metastases at Relapse.
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INTRODUCTION: Neuroblastoma (NB) with central nervous system (CNS) metastases is rare at diagnosis, but occurs more often during relapse/progression. Patients with CNS metastases face a dismal prognosis, with no standardized curative treatment available. Novel therapeutic approaches, such as intraventricular radio-immunotherapy with 131I-omburtamab (Omb), have been developed. In this study, we report a retrospective, single-tertiary center analysis of a 23-year cohort of NB patients with CNS metastases, highlighting current treatment strategies. PATIENTS AND METHODS: Retrospective data analysis of all NB patients with CNS metastases treated at Hospital Sant Joan de Déu, Barcelona, from January 2000 to January 2023. Patient characteristics at diagnosis, first-line treatment, relapse patterns, and CNS metastasis management were analyzed in search of risk variables and survival outcomes. RESULTS: CNS metastases at relapse were identified in 39/185 (21.1%) patients. Median age at diagnosis was 2.7 years, and 24/39 were male. Stage 4 NB with multisite compartment metastases accounted for most cases (92.2%). CNS events occurred predominantly at first relapse (29/39, 74.4%) and with neurological symptoms (23/38, 60.5%). MCYN amplification and concomitant extra-CNS metastases at CNS relapse were associated with poorer overall survival (OS) (p = 0.018 and p = 0.0059, respectively). Neurological symptoms upon relapse significantly increased the risk for subsequent CNS events (p = 0.028). Curative-intent treatment was attempted in 34/39 (87.2%) patients. After adjusting for immortal time bias, RT plus Omb significantly improved OS (p < 0.0001). CONCLUSIONS: In our experience, MYCN amplification and concomitant extra-CNS metastases at CNS relapse significantly decrease OS. Multimodal treatment, including 131I-omburtamab radioimmunotherapy, significantly improves survival outcomes.