Abstract 1435: Cabozantinib for relapsed neuroblastoma: Case series from one academic medical center
Authors: Perisa Michael P. , Streby Keri , Ranalli Mark , Skeens Micah A. , Shah Nilay 
Published: September 2020

Neuroblastoma represents 7% of pediatric cancers in the United States but disproportionately 15% of pediatric cancer-related deaths.1 For patients with high-risk disease, as determined by anatomic, histologic, clinical and molecular staging, aggressive multimodal therapy results in 5-year overall survival of 50-65%.2 Outcomes for relapsed neuroblastoma are worse, with 10% 2-year event free survival and 20% 4-year overall survival.3 These data emphasize the necessity of novel therapeutic approaches for relapsed neuroblastoma. Cabozantinib is a multitargeted tyrosine kinase inhibitor (TKI) approved by the US Food and Drug Administration (FDA) for use in adults for medullary thyroid cancer,4 renal cell carcinoma,5 and hepatocellular carcinoma.5

Cabozantinib is effective preclinically against neuroblastoma tumor cells alone and in combination therapies,6 and it has been shown to be safe and tolerable in a phase I study for pediatric cancers.7 Given these data, we used cabozantinib in four patients with relapsed neuroblastoma for whom other therapeutic options were not available. We report the outcomes of these patients with advanced neuroblastoma treated with cabozantinib, including use of a novel liquid formulation with clinical evidence of pharmacologic activity.

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Citation Format: Michael P. Perisa, Keri Streby, Mark Ranalli, Micah A. Skeens, Nilay Shah. Cabozantinib for relapsed neuroblastoma: Case series from one academic medical center [abstract]. In: Proceedings of the Annual Meeting of the American Association for Cancer Research 2020; 2020 Apr 27-28 and Jun 22-24. Philadelphia (PA): AACR; Cancer Res 2020;80(16 Suppl):Abstract nr 1435.