Graft Versus Host Disease Associated with Immune Checkpoint Inhibitors

A recent systematic literature review, published in Frontiers in Pharmacology, found a significant association between immune checkpoint inhibitor (ICI) treatment and graft-versus-host disease (GVHD). ICI are emerging therapeutic agents used for hematologic malignancies after allogeneic hematopoietic stem cell transplantation (allo-HSCT) or in relapse after allo-HSCT. These drugs target programmed death-1 receptor (anti-PD-1), its ligand (anti-PD-L1) and cytotoxic T lymphocyte antigen-4 (anti-CTLA-4), with the overall goal of enhancing the immune system. Unfortunately, a serious complication of allo-HSCT, GVHD, can be exacerbated by ICI treatment.


This study used VigiBase®, a global database of individual case safety reports (ICSRs), to query cases of GVHD associated with ICI treatment. Their findings yielded a significant association between GVHD events and ICI drugs, with the most prominent drug association being nivolumab. They also found the overall mortality associated with ICI and GVHD to be 25.8%. Furthermore, the authors suggest emerging evidence that the mTOR pathway may be involved in ICI-associated GVHD. Although more research is needed, these findings demonstrate that ICI use in patients with allo-HSCT needs to be carefully monitored.



Nguyen LS, Raia L, Lebrun-Vignes B, Salem JE. Graft Versus Host Disease Associated with Immune Checkpoint Inhibitors: A Pharmacovigilance Study and Systematic Literature Review. Front Pharmacol. 11:619649 (2021).


Tags: GVHD, Immunity, Inhibitors, Association

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