OBJECTIVES

: To access the effectiveness and safety of pembrolizumab in advanced or metastatic melanoma in real world context.

METHODS

: An ambidirectional observational cohort study was constituted on 01/06/2017. For this analysis, data cutoff was set on (04/06/2018). Adult patients (≥18 years) diagnosed with advanced or metastatic melanoma and treated with pembrolizumab were identified using the National Oncology Registry. The primary outcome was overall survival (OS) and the secondary outcomes were progression-free survival (PFS) and adverse events (AEs). Data were registered by two authors (FCB; CR). Disease progression (DP) were assessed per RECIST v1.1 and validated by an independent oncologist (MF). AEs were coded with MedDRA and graded with CTCAE v4.03.

RESULTS

: Ninety-eight patients were identified. Fifty-three were female (54.1%). At treatment initiation, the median age was 66.5 years (IQR 57.8-76.3), 69 patients (70.4%) had PS≤1, 81 (82.7%) had metastatic disease and 67 (68.4%) had not received previous systemic therapy. Median follow-up was 6.6 months (IQR 3.4-11.6) and there were no lost to follow-up.

Median treatment duration was 5.0 months (IQR 2.2-7.9). At the cutoff date, treatment was ongoing for 36 patients (36.7%), 58 (59.2%) had discontinued (58.6% due to DP and 14.0% due to AEs). Sixty-seven patients (68.4%) had DP and 44 (44.9%) died. Median OS was 16.6 months (95%CI 8.2-NR) and 1-year OS was 53.5% (95%CI 36.2-64.0). Median PFS was 4.7 months (95%CI 3.9-6.9).

A total of 450 AEs were reported in 77 patients (78.6%) and the most frequent were asthenia, hyperglycemia and diarrhea. Nineteen AEs (4.2%) were grade 3-4.

CONCLUSIONS

: Comparison between our preliminary data and clinical trials suggests a lower 1-year OS (53.5% vs 68.4%) and an equivalence for PFS (4.7 vs 4.1 months (95%CI 2.9-7.2)) and prevalence of AEs (78.6% vs 77%). Notwithstanding, incidence of grade 3-4 AEs in real life was lower (4.2% vs 17.0%).