The safety and short-term efficacy of nivolumab plus ipilimumab for advanced esophageal squamous cell carcinoma.

[BACKGROUNDS] Nivolumab plus ipilimumab (Nivo + Ipi) is recommended as the first-line standard treatment for patients with advanced esophageal squamous cell carcinoma (aESCC), taking into consideration the patient's general condition and Programmed cell death ligand 1 (PD-L1) expression. In the CheckMate 648 (CM648) trial, the incidence of treatment-related adverse events (TRAEs) of any grade and grade ≥ 3 were reported to be 80% and 32%, respectively, among patients who received Nivo + Ipi Still, it is occasionally given to patients with frail general conditions or as salvage-line treatment. However, there was little data on safety and efficacy in real-world settings.

[METHODS] We retrospectively analyzed the data of patients who received Nivo + Ipi for aESCC between 2022 and 2023 in our hospital. We evaluated immune-related AEs (irAEs), overall response rate (ORR), progression-free survival (PFS), and overall survival (OS).

[RESULTS] Thirty patients were subjected to this study. The patients' characteristics were as follows: median age (range): 63.5 (36-80) years, ECOG PS 0/1 ≤ : 16/14, treatment-line 1st/2nd or later: 6/24. Eight of 30 patients who experienced grade 2 or higher irAE required systemic steroid therapy. Four of 8 patients required additional treatment following the initial steroid therapy, two with increased steroid dose, one with mycophenolate mofetil (MMF), and one with steroid pulse therapy plus MMF. The ORR, median PFS, and median OS were 66.7%, 11.0 months, and 15.4 months in the 1st-line group and 36.8%, 2.6 months, and 10.2 months in the 2nd or later-line group, respectively.

[CONCLUSIONS] Our study showed a safety profile comparable to that of CM648 trial. Nivo + Ipi as 2nd or later-line treatment demonstrated promising efficacy.