Cabozantinib in Renal Cell Carcinoma With Brain Metastases

Giorgia Peverelli, Alessandra Raimondi, Raffaele Ratta, Elena Verzoni, Marco Bregni, Enrico Cortesi, Giacomo Cartenì, Giuseppe Fornarini, Gaetano Facchini, Sebastiano Buti, Luca Galli, Marcello Tucci, Michele Prisciandaro, Giuseppe Procopio

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Background: Cabozantinib showed efficacy and manageable toxicity in patients with metastatic renal cell carcinoma (mRCC). In this study we aimed to describe the safety and to collect evidence on the potential efficacy of cabozantinib in mRCC patients with brain metastases (BM) in a real-world experience. Materials and Methods: We retrospectively collected data of patients treated with cabozantinib within the Italian Managed Access Program. Patients were selected for the presence of BM before the start of treatment and for at least 1 previous tyrosine kinase inhibitor (TKI) treatment regimen for metastatic disease. Safety data were reported, and overall response rate (ORR), brain-specific response, progression-free survival (PFS), and median overall survival (OS) were analyzed. Results: Overall, 12 patients treated with cabozantinib were evaluated. Any grade adverse events (AEs) accounted for 92%, Grade 3/4 AEs rated at 36% with no major neurological side effects. The most common AEs included hypertension (33%), fatigue (24%), aminotransferase elevation (25%), hypothyroidism (16%), and gastrointestinal toxicity (16%). The ORR was 50% with a disease control rate of 75%. All 5 patients treated with a combined systemic and brain-directed approach obtained intracranial disease control, without increased toxicity. Median PFS and median OS were 5.8 and 8.8 months, respectively. Comparable safety and tolerability results for other TKI regimens were reported from the literature. Conclusion: Cabozantinib showed safety, acceptable tolerability, and promising antitumor activity in a population of mRCC patients with BM from a real-world experience. A combined modality approach for renal cell carcinoma with BM, whenever feasible, could be recommended to improve oncological outcomes.
Original languageEnglish
JournalClinical Genitourinary Cancer
Publication statusPublished - 2019



  • Brain-specific response
  • Metastatic renal cell carcinoma
  • Neurological toxicity
  • Systemic and loco-regional treatment integration
  • Tyrosine kinase inhibitors

Cite this

Peverelli, G., Raimondi, A., Ratta, R., Verzoni, E., Bregni, M., Cortesi, E., Cartenì, G., Fornarini, G., Facchini, G., Buti, S., Galli, L., Tucci, M., Prisciandaro, M., & Procopio, G. (2019). Cabozantinib in Renal Cell Carcinoma With Brain Metastases. Clinical Genitourinary Cancer.