Pembrolizumab as first-line treatment for metastatic uveal melanoma

Ernesto Rossi, Monica Maria Pagliara, Daniela Orteschi, Tommaso Dosa, Maria Grazia Sammarco, Carmela Grazia Caputo, Gianluigi Petrone, Guido Rindi, Marcella Zollino, Maria Antonietta Blasi, Alessandra Cassano, Emilio Bria, Giampaolo Tortora, Giovanni Schinzari

Research output: Contribution to journalArticle


BACKGROUND: No standard treatment has been defined for metastatic uveal melanoma (mUM). Although clinical trials testing Nivolumab/Pembrolizumab for cutaneous melanoma did not include mUM, anti PD-1 agents are commonly used for this disease.

PATIENTS AND METHODS: In this prospective observational cohort single arm study, we investigated efficacy and safety of Pembrolizumab as first-line therapy for mUM. The efficacy was evaluated in terms of progression-free survival (PFS), response rate and overall survival (OS). Toxicity was also assessed.

RESULTS: Seventeen patients were enrolled. A median of 8 cycles were administered (range 2-28). Two patients achieved partial response (11.7%), 6 a disease stabilization (35.3%), whereas 9 (53%) had a progression. No complete response was observed. PFS of the overall population was 3.8 months. PFS was 9.7 months for patients with an interval higher than 5 years from diagnosis of primary tumor to metastatic disease and 2.6 months for patients with an interval lower than 5 years [p = 0.039, HR 0.2865 (95% CI 0.0869-0.9443)]. Median OS was not reached. The two responding patients were still on treatment with Pembrolizumab at the time of data analysis. Survival was 12.8 months for patients with clinical benefit, while OS for progressive patients was 3.1 months. PD-L1 expression and genomic abnormalities predictive of relapse after diagnosis of primary tumor were not associated with PFS. Toxicity was mild, without grade 3-4 side effects.

CONCLUSIONS: The efficacy of Pembrolizumab does not seem particularly different when compared to other agents for mUM, but responding patients had a remarkable disease control.

Original languageEnglish
Pages (from-to)1179-1185
Number of pages7
JournalCancer Immunology and Immunotherapy
Issue number7
Publication statusPublished - Jul 2019


  • Adult
  • Aged
  • Aged, 80 and over
  • Antibodies, Monoclonal, Humanized/therapeutic use
  • Antineoplastic Agents, Immunological/therapeutic use
  • Female
  • Humans
  • Male
  • Melanoma/drug therapy
  • Middle Aged
  • Programmed Cell Death 1 Receptor/antagonists & inhibitors
  • Progression-Free Survival
  • Prospective Studies
  • Uveal Neoplasms/drug therapy

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  • Cite this

    Rossi, E., Pagliara, M. M., Orteschi, D., Dosa, T., Sammarco, M. G., Caputo, C. G., Petrone, G., Rindi, G., Zollino, M., Blasi, M. A., Cassano, A., Bria, E., Tortora, G., & Schinzari, G. (2019). Pembrolizumab as first-line treatment for metastatic uveal melanoma. Cancer Immunology and Immunotherapy, 68(7), 1179-1185.