Assessing the Safety and Efficacy of Two Starting Doses of Lenvatinib Plus Everolimus in Patients with Renal Cell Carcinoma: A Randomized Phase 2 Trial.

Lenvatinib (18 mg) plus everolimus (5 mg) is approved for patients with advanced renal cell carcinoma (RCC) after one or more prior antiangiogenic therapies.

To assess whether a lower starting dose of lenvatinib has comparable efficacy with improved tolerability for patients with advanced RCC treated with lenvatinib plus everolimus.

A randomized, open-label, phase 2 global trial was conducted in patients with advanced clear cell RCC and disease progression after one prior vascular endothelial growth factor-targeted therapy (prior anti-programmed death-1/programmed death ligand-1 therapy permitted).

Patients were randomly assigned 1:1 to the 14- or 18-mg lenvatinib starting dose, both in combination with everolimus 5 mg/d. Patients in the 14-mg arm were to be uptitrated to lenvatinib 18 mg at cycle 2, day 1, barring intolerable grade 2 or any grade ≥3 treatment-emergent adverse events (TEAEs) requiring dose reduction occurring in the first 28-d cycle.

The primary efficacy endpoint was investigator-assessed objective response rate (ORR) as of week 24 (ORRwk24); the noninferiority threshold of the 14- versus 18-mg arm was p ≤ 0.045. The primary safety endpoint was the proportion of patients with intolerable grade 2 or any grade ≥3 TEAEs within 24 wk of randomization.

The ORRwk24 for the 14-mg arm (32% [95% confidence interval {CI} 25-39]) was not noninferior to the ORRwk24 in the 18-mg arm (35% [95% CI 27-42]; odds ratio: 0.88; 90% CI 0.59-1.32; p = 0.3). The proportion of intolerable grade 2 or any grade ≥3 TEAEs was similar between the two arms (14 mg, 83% vs 18 mg, 80%; p = 0.5). The secondary endpoints of overall ORR, progression-free survival, and overall survival numerically favored the 18-mg arm. A limitation of this study was that the study design did not allow for a full comparison of progression-free survival between treatment arms.

The study findings support the approved dosing regimen of lenvatinib 18 mg plus everolimus 5 mg daily for patients with advanced RCC.

In this report, we examined two doses of lenvatinib (the approved 18-mg dose and a lower dose of 14 mg) in people with advanced renal cell carcinoma to determine whether the lower dose (which was increased to the approved 18-mg dose after the first treatment cycle) could improve safety without affecting efficacy. The results showed that the efficacy of the lower lenvatinib dose (14 mg) was not the same as that of the approved (18 mg) dose, although safety results were similar, so the approved lenvatinib 18-mg dose should still be used.

European urology. 2022 Feb 21 [Epub ahead of print]

Sumanta K Pal, Javier Puente, Daniel Y C Heng, Hilary Glen, Piotr Koralewski, Daniil Stroyakovskiy, Boris Alekseev, Francis Parnis, Daniel Castellano, Tudor Ciuleanu, Jae Lyun Lee, Kaisa Sunela, Karen O'Hara, Terri A Binder, Lixian Peng, Alan D Smith, Sun Young Rha

Department of Medical Oncology & Therapeutics, City of Hope Comprehensive Cancer Center, Duarte, CA, USA. Electronic address: ., Medical Oncology Department, Hospital Clinico San Carlos, Instituto de Investigación Sanitaria del Hospital Clínico San Carlos (IdISSC), CIBERONC, Madrid, Spain., Department of Oncology, Tom Baker Cancer Center, Calgary, Alberta, Canada., Medical Oncology, Beatson West of Scotland Cancer Centre, Glasgow, UK., Department of Oncology, Rydygier Hospital, Krakow, Poland., Chemotherapeutic Department, Moscow City Oncology Hospital, Moscow, Russian Federation., Moscow Hertzen Oncology Institute, Moscow, Russian Federation (Alekseev)., Medical Oncology, Adelaide Cancer Center, Adelaide, Australia., Medical Oncology Department, Hospital Universitario 12 de Octubre (CIBERONC), Madrid, Spain., Medical Oncology, Iuliu Hatieganu University of Medicine and Pharmacy, Ion Chiricuta Institute of Oncology, Cluj-Napoca, Romania., Department of Oncology, University of Ulsan College of Medicine, Asan Medical Center, Seoul, South Korea., Department of Oncology, Tampere University Hospital, Tampere, Finland., Eisai Europe Ltd., Hatfield, UK., Eisai Inc., Nutley, NJ, USA., Department of Medical Oncology, Yonsei Cancer Center, Yonsei University Health System, Seoul, South Korea. Electronic address: .