A single-institution experience with the pembrolizumab (PEM) Expanded Access Program (EAP).

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e20088 Background: PEM is contingently approved in the US to treat patients (pts) with advanced melanoma (MEL) whose disease progressed following ipilimumab (IPI) and a BRAF inhibitor (if BRAFV600-mutated MEL). We report our clinical experience of MEL pts enrolled in the PEM EAP. Methods: 62 pts were enrolled and treated on PEM EAP from May to September 2014 at MD Anderson Cancer Center. PEM was administered at 2 mg/kg every 3 wk until unacceptable toxicity, disease progression, or for up to 2 yr. Toxicity was assessed before each dosing. Response was evaluated every 12 wk (after every 4 doses) using immune-related response criteria. Results: Table showsbaseline characteristics of 60 pts included in the efficacy analysis (excluding 2 pts with uveal MEL). Median number of PEM doses administered was 6 (range 1-12). At a median follow-up of 5.4 mo (range 0.2-7.8), the overall response rate (ORR) was 22% (26% in 51 pts who received ≥ 4 doses), with 17% (20%) stable disease. Median progression-free survival (PFS) was 3.5 mo (95% CI 2.8-4.8); median overall survival (OS) was not reached (95% CI 6.7-not evaluable). Univariate analysis showed a statistically significant association between OS and ECOG PS and LDH; PFS and ORR were associated with ECOG PS, LDH, and stage. BRAF mutation status was not associated with treatment outcome. Adverse drug events (ADE) occurred in 87% of pts (n = 62), all grade 1/2. ADE of interest were pruritus (11%), rash (18%), diarrhea (18%), elevated liver function tests (42%) and abnormal thyroid function tests (9.6%). One pt developed grade 2 pneumonitis. Grade 3-4 toxicities and treatment-related deaths were not observed. Conclusions: Our experience with PEM EAP was similar to published data of PEM in metastatic MEL. PEM is safe and effective in pts with advanced MEL refractory to standard-of-care therapy including IPI and BRAF inhibitor. Clinical trial information: NCT02083484.Patient characteristics. Number of patients N Age Median (Range) Stage M1c N (%) Prior Therapies Median (Range) Elevated LDH N (%) History of Brain Metastases N (%) Mutations N (%) ECOG PS N (%) BRAF NRAS 0 1 60 60.5 (16-85) 45 (75) 2 (1-7) 25 (42) 15 (25) 15 (25) 20 (33.3) 40 (66.7) 20 (33.3)

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