Abstract 1080P
Background
Based on early data, compassionate use of pembrolizumab (PEM) was granted to patients (pts) with advanced melanoma (unresectable or metastatic) in a French early access program (06/2014 – 09/2015). To explore real-world evidence (RWE), enrolment for this multicenter retro and prospective cohort study began in 06/2016. We present final results for effectiveness among this population.
Methods
Primary objective was 5 year (y) overall survival (OS) after PEM initiation. Other endpoints included yearly overall survival rates (OSR); real-world progression free survival (rwPFS; PEM initiation to progression); overall response rate (ORR, % of pts with complete or partial response [CR, PR]); disease control rate (DCR, % of pts with CR, PR or stable disease ≥ 24 weeks); duration of response (DoR; 1st objective tumor response until tumor progression or death) and long term safety data. Kaplan Meier survival analyses were used.
Results
Of 913 pts in the program, 705 pts (377 men) were enrolled in HORIZON with a mean delay of 1.2 y between diagnosis of advanced melanoma and 1st PEM infusion. Median follow up was 12.52 months (m; 95%CI [11.08; 15.87]), ranging from 0 to 70.1 m. At PEM initiation, mean age was 63.2 y, 52.1% had ≥ 3 metastatic sites and 71.3% were BRAF wild type. Only 179 pts (25.4%) were melanoma treatment (ttt) naïve, while 51.5% received prior ipilimumab. In this particularly severe population, OSR at 1 y to 5 y were 54.3%, 37.9%, 31.2%, 26.5% and 25.2%. At the 5 y follow up, median OS was 14.72 m (95%CI [12.2; 17.67]), rwPFS 3.21 m (95%CI [2.85 ; 3.64]). DoR was 30.07 m (95%CI [17.34; 43.41]), with 33.9% ORR and 42.4% DCR. Results will be presented by subgroups, including lines of ttt, presence of brain metastases, LDH levels, age and performance status.
Conclusions
Horizon is a large study, giving a real life long term landscape of non selected melanoma patients. Median OS and rwPFS are consistent with studies enrolling pts with prior therapy, the ORR and the 5Y OSR were respectively 33.9% and 25.2%. This is also a realistic estimation of the challenge to be filled by complementary therapeutic strategies in melanoma. We kindly thank all RIC-Mel network investigators.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
MSD France.
Funding
MSD France.
Disclosure
J.J. Grob: Financial Interests, Personal and Institutional, Advisory Role: MSD France; Financial Interests, Personal and Institutional, Advisory Role: BMS; Financial Interests, Personal and Institutional, Advisory Role: Roche; Financial Interests, Personal and Institutional, Advisory Role: Novartis; Financial Interests, Personal and Institutional, Advisory Role: Amgen; Financial Interests, Personal and Institutional, Advisory Role: Pierre Fabre; Financial Interests, Personal and Institutional, Advisory Role: Merck; Non-Financial Interests, Personal and Institutional, Advisory Role: MSD France; Non-Financial Interests, Personal and Institutional, Advisory Role: BMS; Non-Financial Interests, Personal and Institutional, Advisory Role: Roche; Non-Financial Interests, Personal and Institutional, Advisory Role: Novartis; Non-Financial Interests, Personal and Institutional, Advisory Role: Amgen; Non-Financial Interests, Personal and Institutional, Advisory Role: Pierre Fabre; Non-Financial Interests, Personal and Institutional, Advisory Role: Merck; Non-Financial Interests, Personal and Institutional, Advisory Role: Sanofi; Non-Financial Interests, Personal and Institutional, Advisory Role: Philogen; Non-Financial Interests, Personal and Institutional, Advisory Role: Pfizer. F. Aubin: Non-Financial Interests, Personal and Institutional, Advisory Role: MSD France; Non-Financial Interests, Personal, Invited Speaker: Novartis; Non-Financial Interests, Personal, Advisory Board: Novartis; Non-Financial Interests, Personal, Invited Speaker: BMS; Non-Financial Interests, Personal, Advisory Board: BMS. L. Benmahammed-Bellagha: Financial Interests, Personal and Institutional, Full or part-time Employment: MSD France; Financial Interests, Personal and Institutional, Stocks/Shares: MSD France. F. Brunet-Possenti: Non-Financial Interests, Personal and Institutional, Advisory Role: MSD France; Non-Financial Interests, Personal and Institutional, Advisory Board: Novartis; Non-Financial Interests, Personal and Institutional, Advisory Board: BMS; Non-Financial Interests, Personal and Institutional, Advisory Board: Sanofi. C. Dutriaux: Financial Interests, Personal and Institutional, Advisory Role: MSD France; Financial Interests, Personal and Institutional, Advisory Role: BMS; Financial Interests, Personal and Institutional, Advisory Role: Novartis; Financial Interests, Personal and Institutional, Advisory Role: Pierre Fabre; Non-Financial Interests, Personal and Institutional, Advisory Role: MSD France; Non-Financial Interests, Personal and Institutional, Advisory Role: BMS; Non-Financial Interests, Personal and Institutional, Advisory Role: Pierre Fabre; Non-Financial Interests, Personal and Institutional, Advisory Role: Novartis. C. Jarvis: Financial Interests, Personal and Institutional, Full or part-time Employment: ClinSearch. N. Kramkimel: Non-Financial Interests, Personal and Institutional, Advisory Role: MSD France; Non-Financial Interests, Personal and Institutional, Advisory Role: BMS; Non-Financial Interests, Personal and Institutional, Advisory Role: Sanofi. M. Leccia: Non-Financial Interests, Personal and Institutional, Advisory Role: MSD France. N. Meyer: Financial Interests, Personal and Institutional, Funding: MSD France; Non-Financial Interests, Personal and Institutional, Advisory Role: MSD France; Non-Financial Interests, Personal and Institutional, Advisory Role: BMS; Non-Financial Interests, Personal and Institutional, Advisory Role: Pierre Fabre; Non-Financial Interests, Personal and Institutional, Advisory Role: Novartis; Non-Financial Interests, Personal and Institutional, Advisory Role: Incyte. L. Mortier: Financial Interests, Personal and Institutional, Advisory Role: MSD France; Financial Interests, Personal and Institutional, Advisory Role: BMS; Financial Interests, Personal and Institutional, Advisory Role: Roche; Financial Interests, Personal and Institutional, Advisory Role: Novartis; Financial Interests, Personal and Institutional, Advisory Role: Pierre Fabre. E. Neidhardt: Non-Financial Interests, Personal and Institutional, Advisory Role: MSD France; Non-Financial Interests, Personal and Institutional, Invited Speaker: BMS. C. Robert: Financial Interests, Personal, Advisory Board: BMS; Financial Interests, Personal, Advisory Board: Roche; Financial Interests, Personal, Advisory Board: Pierre Fabre; Financial Interests, Personal, Advisory Board: Novartis; Financial Interests, Personal, Advisory Board: Sanofi; Financial Interests, Personal, Advisory Board: MSD; Financial Interests, Personal, Advisory Board: AstraZeneca. E. Scherrer: Financial Interests, Personal and Institutional, Full or part-time Employment: MSD; Financial Interests, Personal and Institutional, Stocks/Shares: MSD. A. Spampinato: Financial Interests, Personal and Institutional, Full or part-time Employment: MSD France; Financial Interests, Personal and Institutional, Stocks/Shares: MSD France. L. Verdoni: Financial Interests, Personal and Institutional, Full or part-time Employment: MSD France; Financial Interests, Personal and Institutional, Stocks/Shares: MSD France. P. Saiag: Financial Interests, Personal and Institutional, Funding: MSD France; Financial Interests, Personal and Institutional, Funding: Merck Serono; Financial Interests, Personal and Institutional, Funding: BMS; Financial Interests, Personal and Institutional, Funding: Pierre Fabre; Financial Interests, Personal and Institutional, Funding: Novartis; Financial Interests, Personal and Institutional, Funding: Roche; Financial Interests, Personal and Institutional, Funding: Array; Non-Financial Interests, Personal and Institutional, Advisory Role: MSD France; Non-Financial Interests, Personal and Institutional, Advisory Role: BMS; Non-Financial Interests, Personal and Institutional, Advisory Role: Pierre Fabre; Non-Financial Interests, Personal and Institutional, Advisory Role: Novartis; Non-Financial Interests, Personal and Institutional, Advisory Role: Incyte; Non-Financial Interests, Personal and Institutional, Advisory Role: Sanofi; Non-Financial Interests, Personal and Institutional, Advisory Role: Philogen; Non-Financial Interests, Personal and Institutional, Advisory Role: Pfizer. All other authors have declared no conflicts of interest.