Vedolizumab for steroid-refractory gastrointestinal acute graft-versus-host disease: A real-world VAST study

Abstract Background: Steroid-refractory (SR) acute graft-versus-host disease (aGVHD) is the major cause of early mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT), particularly for those with gastrointestinal (GI) involvement. Vedolizumab which can disrupt the homing of T cells to gut lymphoid tissue has the potential effect for the treatment of GI-aGVHD. This study aims to evaluate the efficacy, safety, and prognostic factors of vedolizumab treatment for SR-GI-aGVHD in real-world settings. Methods: This was a multicenter, retrospective analysis included SR-GI-aGVHD patients who received vedolizumab at 5 hospitals across China. The primary endpoint was the 28-day overall response rate (ORR) for GI-aGVHD. This study also included an independent historical SR-GI-aGVHD cohort treated without vedolizumab (n = 411) as control. Results: In total, 100 SR-GI-aGVHD patients were enrolled. Vedolizumab was administered either as monotherapy (n = 23) or in combination with other second-line treatments (n = 77). The ORR for GI-aGVHD was 68.0% on day 28 and 81.0% at any time after vedolizumab treatment. Particularly, patients receiving vedolizumab within 7 days showed higher ORR compared with those receiving vedolizumab beyond 7 days. The ORR at day 28 and at any time for overall aGVHD was 68.0% and 81.0%, respectively, after vedolizumab treatment. The 2-year probabilities of overall survival and non-relapse mortality (NRM) was 58.3% (95% CI 46.5 – 70.1%) and 33.3% (95% CI 22.1 – 44.5%), respectively. Vedolizumab-based therapy also showed some superiority over therapy without vedolizumab in historical cohort. Conclusions: Our real-world findings suggest that vedolizumab is an effective and safe treatment for SR-GI-aGVHD.