Abstract
BACKGROUND: Immune checkpoint inhibitor-related esophagitis (IME) is often managed with proton pump inhibitors (PPIs). Severe or refractory cases may require steroids and/or selective immunosuppressive therapies (SIT). However, large-scale studies assessing IME treatment strategies are lacking. This study evaluated their efficacy. METHOD: This retrospective study at a tertiary cancer center included patients with malignancy who received immune checkpoint inhibitor (ICIs) from 2010-2024 and developed IME, defined as new or worsening upper gastrointestinal (GI) symptoms post-ICI initiation with other causes excluded. RESULTS: Among 148 patients, 75% received PD-1/PD-L1 inhibitors for 4.9 months; 50.7% received concurrent chemotherapy. Isolated IME was present in 27.7% of patients, while the remainder had concurrent immune-mediated GI conditions. Only 24.4% of isolated IME cases were treated with PPIs, and there was no significant difference between the PPI and non-PPI groups in steroid administration, outcomes or recurrence. Corticosteroids were used in 27.7% of cases, significantly shortening the time to symptom resolution (12 vs. 45 days; P=0.015). Nausea (87.8% vs. 57%, P<0.001) and emesis (58.5% vs. 34.6%, P=0.008) were more frequently observed in the steroid group, along with higher rates of hospitalization (73.2% vs. 36.4%, P<0.001), need for intravenous steroids (30% vs. 0%, P<0.001), and ICI discontinuation (74.4% vs. 44.6%, P=0.002). SIT were required for other concomitant GI adverse events in 41.5% of the steroid-treated patients. No significant differences in clinical improvement, ICI resumption or all-cause mortality were noted between the corticosteroid and non-corticosteroid groups. CONCLUSION: Our findings showed faster clinical improvement with steroids, while PPIs demonstrated no significant effectiveness.