Abstract

Abstract Background: Immune checkpoint blockade (ICB) and BRAFV600-targeted therapy have demonstrated substantial clinical efficacy for patients with stage 4 melanoma in clinical trials; however, their impact on survival and barriers to treatment in the “real-life” setting remains unknown. Methods: Patients who presented with cutaneous melanoma during 2004-2015 using the National Cancer Database, which comprises >70% of all newly-diagnosed cancers in the U.S., were evaluated for predictors of presenting with stage 4 disease and receiving ICB, and the associated unadjusted and risk-adjusted overall survival (OS). Results: 17,975 patients presented with stage 4 metastatic cutaneous melanoma. Overall, patients who presented after the FDA’s initial approvals (starting in 2011) for ICB and BRAFV600-targeted therapy demonstrated a 31% relative improvement in 4-yr OS (p<0.001), compared to pre-2011. Following the initial approvals in 2011, improved OS was associated in risk-adjusted analyses with ICB (HR 0.57, 95CI: 0.52-0.63). ICB demonstrated improved median and 4-yr OS of 16.9 months (95CI: 15.6-19.3; vs. 7.7 months, 95CI: 7.2-8.4) and 32.4% (95CI: 29.5-35.3; vs. 21.0%, 95CI: 19.6-22.2, all p<0.001), respectively; improved OS was persistent in unadjusted and risk-adjusted landmark survival analyses. Uninsured patients and management in the community setting were less likely to receive ICB in multivariable analyses. Conclusions: In a national “real-life” treatment population, we show that the wide availability of the novel treatment modalities ICB and BRAFV600-targeted therapy has significantly improved the survival of patients with stage 4 melanoma. Our findings additionally suggest that there are opportunities for expanding coverage and access to these novel immunotherapies in community practice. Citation Format: Allison Dobry, Cheryl Zogg, Stephen Hodi, Timothy Smith, Patrick Ott, Bryan Iorgulescu. Management of advanced melanoma: improved survival and barriers to access in a national cohort following the approvals of checkpoint blockade immunotherapies and targeted therapies [abstract]. In: Proceedings of the AACR Special Conference on Tumor Immunology and Immunotherapy; 2018 Nov 27-30; Miami Beach, FL. Philadelphia (PA): AACR; Cancer Immunol Res 2020;8(4 Suppl):Abstract nr A99.

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