Safety and Efficacy of Nivolumab in Brain Metastases From Renal Cell Carcinoma: Results of the GETUG-AFU 26 NIVOREN Multicenter Phase II Study.

Nivolumab is standard of care for patients with metastatic clear cell renal cell carcinoma (ccRCC) after failure of antiangiogenic therapies, but its activity on brain metastases from ccRCC remains unknown, because these patients were excluded from pivotal studies. We aimed to assess the activity of nivolumab in this population.

The GETUG-AFU 26 NIVOREN phase II trial assessed the activity and safety of nivolumab in patients with metastatic ccRCC who failed vascular endothelial growth factor-directed therapies ( ClinicalTrials.gov identifier: NCT03013335 ). Patients with asymptomatic brain metastases were prospectively identified and underwent dedicated brain evaluation. Two cohorts were constituted: cohort A comprised patients with previously untreated brain metastases, and cohort B comprised patients whose brain metastases underwent prior therapy. The primary end point was intracranial response rate in cohort A.

Seventy-three patients with brain metastases were included: 39 in cohort A and 34 in cohort B. Intracranial response rate was 12% in cohort A; no objective response was reported in patients with brain lesions that were multiple or larger than 1 cm. Median intracranial progression-free survival was 2.7 months (95% CI, 2.3 to 4.6 months) in cohort A and 4.8 months (95% CI, 3.0 to 8.0 months) in cohort B, with adjusted hazard ratio of 2.04 (95% CI, 1.08 to 3.83). Overall survival rate at 12 months was 67% (95% CI, 49.6% to 79.1%) in cohort A and 59% (95% CI, 40.6% to 73.2%) in cohort B. Most patients in cohort A (72%) needed subsequent focal brain therapy. Nivolumab was well tolerated, with no unexpected toxicity.

Nivolumab activity is limited in patients with untreated brain metastases from ccRCC. Brain imaging and focal therapy should be considered before immune checkpoint inhibitors in patients with metastatic ccRCC.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology. 2019 Jun 13 [Epub ahead of print]

Ronan Flippot, Cécile Dalban, Brigitte Laguerre, Delphine Borchiellini, Gwénaelle Gravis, Sylvie Négrier, Christine Chevreau, Florence Joly, Lionnel Geoffrois, Sylvain Ladoire, Hakim Mahammedi, Frédéric Rolland, Marine Gross-Goupil, Elise Deluche, Frank Priou, Mathieu Laramas, Philippe Barthélémy, Bérengère Narciso, Nadine Houedé, Stéphane Culine, Stéphane Oudard, Marina Chenot, Florence Tantot, Sylvie Chabaud, Bernard Escudier, Laurence Albiges

1 Gustave Roussy Cancer Campus, Université Paris-Saclay, Villejuif, France., 2 Centre Léon Berard, Lyon, France., 3 Centre Eugene Marquis, Rennes, France., 4 Université Côte d'Azur, Nice, France., 5 Institut Paoli Calmettes, Marseille, France., 6 Institut Universitaire du Cancer Toulouse-Oncopole, Toulouse, France., 7 Centre François Baclesse, Caen, France., 8 Institut de Cancérologie de Lorraine, Vandoeuvre-lès-Nancy, France., 9 Centre Georges François Leclerc, Dijon, France., 10 Centre Jean Perrin, Clermont-Ferrand, France., 11 Centre René Gauducheau, Saint Herblain, France., 12 Bordeaux University Hospital, Bordeaux, France., 13 Limoges University Hospital, Limoges, France., 14 Centre Hospitalier de Vendée, La Roche sur Yon, France., 15 Alpes University Hospital, Grenoble, France., 16 Strasbourg University Hospital, Strasbourg, France., 17 Tours University Hospital, Tours, France., 18 Nimes University Hospital, Nimes, France., 19 Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Paris, France., 20 Hôpital Européen Georges Pompidou, Assistance Publique-Hôpitaux de Paris, Paris, France., 21 Université Montpellier, Montpellier, France., 22 GETUG Group, Unicancer, Paris, France.