Vedolizumab for SR GI-aGvHD: A real-world study

Results from a multicenter, retrospective, real-world study evaluating outcomes in 100 patients with steroid-refractory (SR) gastrointestinal (GI) acute graft-versus-host disease (aGvHD) who received vedolizumab between March 2019 and November 2024, were published in MedComm by Cao et al. Patients who received best available treatment (BAT, n = 411) during the same time period served as controls. The primary endpoint was the overall response rate (ORR) on Day 28 for lower GI aGvHD, defined as the proportion of patients achieving a complete response (CR) or partial response (PR).

Key data: The ORRs on Day 28 and at any time after vedolizumab treatment were 68.0% (95% confidence interval [CI], 57.9–77.0) and 81.0% (95% CI, 71.9–88.2), respectively. The 2-year probabilities of overall survival (OS) and non-relapse mortality (NRM) were 58.3% and 31.9%, respectively. The vedolizumab group presented comparable ORRs compared with the BAT group; however, in patients with Stage III–IV SR-GI-aGvHD, the vedolizumab group presented significantly higher CR rates on Day 28 (52.3% vs 24.9%; p < 0.001) and at any time (59.3% vs 41.2%; p = 0.004). The proportion of patients experiencing ≥1 infection was lower in the vedolizumab group compared with BAT (22.2% vs 63.0%; p < 0.001).

Key learning: This real-world analysis confirmed the efficacy of vedolizumab in patients with SR-aGvHD, with a lower infection rate compared with BAT, supporting further evaluation in prospective, randomized studies.