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. 2015 Jun 30;113(1):12-9.
doi: 10.1038/bjc.2015.196. Epub 2015 Jun 18.

Final results from the large sunitinib global expanded-access trial in metastatic renal cell carcinoma

M E Gore  1 , C Szczylik  2 , C Porta  3 , S Bracarda  4 , G A Bjarnason  5 , S Oudard  6 , S-H Lee  7 , J Haanen  8 , D Castellano  9 , E Vrdoljak  10 , P Schöffski  11 , P Mainwaring  12 , R E Hawkins  13 , L Crinò  14 , T M Kim  7 , G Carteni  15 , W E E Eberhardt  16 , K Zhang  17 , K Fly  18 , E Matczak  19 , M J Lechuga  20 , S Hariharan  19 , R Bukowski  21
Affiliations

Final results from the large sunitinib global expanded-access trial in metastatic renal cell carcinoma

M E Gore et al. Br J Cancer. .

Abstract

Background: We report final results with extended follow-up from a global, expanded-access trial that pre-regulatory approval provided sunitinib to metastatic renal cell carcinoma (mRCC) patients, ineligible for registration-directed trials.

Methods: Patients ⩾18 years received oral sunitinib 50 mg per day on a 4-weeks-on-2-weeks-off schedule. Safety was assessed regularly. Tumour measurements were scheduled per local practice.

Results: A total of 4543 patients received sunitinib. Median treatment duration and follow-up were 7.5 and 13.6 months. Objective response rate was 16% (95% confidence interval (CI): 15-17). Median progression-free survival (PFS) and overall survival (OS) were 9.4 months (95% CI: 8.8-10.0) and 18.7 months (95% CI: 17.5-19.5). Median PFS in subgroups of interest: aged ⩾65 years (33%), 10.1 months; Eastern Cooperative Oncology Group performance status ⩾2 (14%), 3.5 months; non-clear cell histology (12%), 6.0 months; and brain metastases (7%), 5.3 months. OS was strongly associated with the International Metastatic Renal-Cell Carcinoma Database Consortium prognostic model (n=4065). The most common grade 3/4 treatment-related adverse events were thrombocytopenia (10%), fatigue (9%), and asthenia, neutropenia, and hand-foot syndrome (each 7%).

Conclusion: Final analysis of the sunitinib expanded-access trial provided a good opportunity to evaluate the long-term side effects of a tyrosine kinase inhibitor used worldwide in mRCC. Efficacy and safety findings were consistent with previous results.

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Conflict of interest statement

MEG reported receiving consultancy fees from Pfizer and Astellas, and honoraria from Roche, Pfizer, Novartis, and Bristol-Myers Squibb. CS reported receiving consultancy fees from Pfizer, Bayer, GlaxoSmithKline, and Wyeth Pharmaceuticals. CP reported receiving consultancy fees from Pfizer, Bayer Schering Pharma, GlaxoSmithKline, Novartis, Boehringer-Ingelheim, and AVEO/Astellas, honoraria from Pfizer, Bayer Schering Pharma, GlaxoSmithKline, Novartis, and Astellas, and research funding from Pfizer, Bayer Schering Pharma, and Novartis. SB reported receiving consultancy fees from Pfizer, Bayer Schering Pharma, GlaxoSmithKline, Novartis, Boehringer-Ingelheim, AVEO/Astellas, and Genentech, and honoraria from Novartis, Pfizer, Bayer Schering Pharma, and GlaxoSmithKline. GAB reported receiving consultancy fees and honoraria from Pfizer, Novartis, and GlaxoSmithKline, and research funding from Pfizer. SO reported receiving consultancy fees and honoraria from Pfizer, Novartis, GlaxoSmithKline, Bayer Schering Pharma, and Boehringer-Ingelheim. S-HL reported receiving consultancy fees from Pfizer, Novartis, Bayer, and Roche, honoraria from Pfizer, Novartis, GlaxoSmithKline, and Bayer, and research funding from Pfizer, AstraZeneca, and Novartis. JH reported receiving consultancy fees from Pfizer. EV reported receiving consultancy fees and honoraria from Pfizer, Novartis, GlaxoSmithKline, and Roche, and research funding from Pfizer. PS reported receiving consultancy fees and honoraria from Pfizer. PM reported receiving consultancy fees from Janssen. REH reported receiving honoraria from Pfizer, Novartis, GlaxoSmithKline, and Bristol-Myers Squibb, and research funding from Pfizer, Novartis, and GlaxoSmithKline. GC reported receiving honoraria and research funding from Pfizer, Novartis, and GlaxoSmithKline. WEEE reported receiving consultancy fees and honoraria from Pfizer, Novartis, GlaxoSmithKline, Roche, Bayer, Boehringer Ingelheim, Astellas, and Bristol-Myers Squibb. KZ, KF, EM, MJL, and SH are full-time employees of Pfizer and hold Pfizer stock. RB reported receiving consultancy fees from Pfizer, Novartis, GlaxoSmithKline, Genentech, and Exelixis, honoraria from Pfizer, Novartis, GlaxoSmithKline, Bayer, and Genentech, and expert testimony fees from Pfizer and Novartis. DC, TMK, and LC declared no conflict of interest.

Figures

Figure 1
Figure 1
Kaplan–Meier estimates of (A) PFSa and (B) OS for the overall population. aIn the PFS plot, 324 modified intent-to-treat patients were excluded from the evaluable population due to non-RECIST tumour assessment.
Figure 2
Figure 2
Kaplan–Meier estimates of OS for the overall population, according to prognostic risk group based on the IMDC model (Heng et al, 2009, 2013). N=4065. a aIn total, 478 patients were excluded due to missing data for one or more risk factors.
See this image and copyright information in PMC

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