Durable responses upon short-term addition of targeted therapy to anti-PD1 in advanced melanoma patients: 5-year progression-free and overall survival update of the IMPemBra trial

Eur J Cancer. 2025 Jun 3:222:115431. doi: 10.1016/j.ejca.2025.115431. Epub 2025 Apr 19.

Abstract

Background: The addition of targeted therapy (TT) to immune checkpoint inhibitors has been shown to transiently increase immune infiltration in melanoma. This formed the rationale for the IMPemBra trial, which showed a numerical increase in progression-free survival (PFS) in patients treated with short-term/intermittent TT and anti-PD1 compared to anti-PD1 alone. In this report, the final toxicity-analysis, 5-year PFS and exploratory analysis of overall survival (OS) will be reported, together with an analysis of subsequent therapies.

Patients and methods: 32 treatment-naïve patients with a BRAFV600E/K-mutated advanced melanoma were treated with 2 cycles of pembrolizumab 200 mg every 3 weeks, followed by randomization to continue pembrolizumab monotherapy for six weeks in cohort-1 versus pembrolizumab plus intermittent dabrafenib 150 mg BID + trametinib 2 mg QD 2×1-week (cohort 2), 2×2-weeks (cohort 3), or 1×6-weeks (cohort 4). After week 12, all patients continued pembrolizumab monotherapy for a maximum of 2 years.

Results: With a median follow-up of 73 months, final grade 3-4 immune-related adverse events are 12 % (cohort 1), 12 % (cohort 2), 38 % (cohort 3) and 63 % (cohort 4). Estimated 5-year PFS and OS rates were 25 % and 50 % for pembrolizumab monotherapy (cohort-1) and 46 % and 71 % for pembrolizumab + intermittent TT (cohorts 2-4). Estimated 5-year PFS and OS were 63 % and 63 % (cohort 2), 38 % and 75 % (cohort 3), and 38 % and 75 % (cohort 4), respectively. The subsequent therapies were balanced between cohorts. Patients treated with short-term/intermittent schemes achieved durable responses upon subsequent TT again.

Conclusion: This survival update from the IMPemBra trial demonstrates that combination of short-term TT and checkpoint inhibition can induce long-lasting responses, warranting further analyses in larger cohorts, and in a randomized design.

Keywords: BRAF; Checkpoint inhibition; Intermittent; Melanoma; Targeted therapy.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • Adult
  • Aged
  • Antibodies, Monoclonal, Humanized* / administration & dosage
  • Antibodies, Monoclonal, Humanized* / adverse effects
  • Antibodies, Monoclonal, Humanized* / therapeutic use
  • Antineoplastic Combined Chemotherapy Protocols* / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols* / therapeutic use
  • Female
  • Humans
  • Imidazoles / administration & dosage
  • Imidazoles / adverse effects
  • Immune Checkpoint Inhibitors* / administration & dosage
  • Immune Checkpoint Inhibitors* / adverse effects
  • Immune Checkpoint Inhibitors* / therapeutic use
  • Male
  • Melanoma* / drug therapy
  • Melanoma* / genetics
  • Melanoma* / immunology
  • Melanoma* / mortality
  • Melanoma* / pathology
  • Middle Aged
  • Oximes / administration & dosage
  • Oximes / adverse effects
  • Programmed Cell Death 1 Receptor* / antagonists & inhibitors
  • Progression-Free Survival
  • Proto-Oncogene Proteins B-raf / genetics
  • Pyridones / administration & dosage
  • Pyridones / adverse effects
  • Pyrimidinones / administration & dosage
  • Pyrimidinones / adverse effects
  • Skin Neoplasms* / drug therapy
  • Skin Neoplasms* / genetics
  • Skin Neoplasms* / immunology
  • Skin Neoplasms* / mortality
  • Skin Neoplasms* / pathology

Substances

  • Antibodies, Monoclonal, Humanized
  • dabrafenib
  • Imidazoles
  • Immune Checkpoint Inhibitors
  • Oximes
  • PDCD1 protein, human
  • pembrolizumab
  • Programmed Cell Death 1 Receptor
  • Proto-Oncogene Proteins B-raf
  • Pyridones
  • Pyrimidinones
  • trametinib