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Updated: Sep 10, 2025

Bone Marrow Transplantation Platform to Investigate the Role of Dendritic Cells in Graft-versus-Host Disease
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Bone Marrow Transplantation Platform to Investigate the Role of Dendritic Cells in Graft-versus-Host Disease

Published on: March 17, 2020

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Emerging novel therapies for steroid-refractory acute graft-versus-host disease: recent advances and future

Yishan Ye1, Wenjing Hao2, Florent Malard2

  • 1Bone Marrow Transplantation Center, The First Affiliated Hospital, Zhejiang University School of Medicine, 310000 Hangzhou, China; Service d'Hématologie Clinique et Thérapie Cellulaire, Hôpital Saint-Antoine, Sorbonne Université, INSERM UMRs 938, Paris, France.

Current Opinion in Immunology
|August 27, 2025
PubMed

Acute graft-versus-host disease (aGVHD) is a significant complication of allogeneic hematopoietic cell transplantation (allo-HCT), characterized by immune-mediated tissue damage from donor immune cells. Standard treatment of aGVHD involves systemic corticosteroids, but many patients do not respond adequately, with 30%-50% of patients being steroid refractory (SR-aGVHD) leading to poor outcomes. This highlights the need for effective second-line therapies. Ruxolitinib, a JAK1/2 inhibitor, has emerged as a key treatment, demonstrating superior overall response rates in SR-aGVHD compared to best available therapies. However, resistance and intolerance to ruxolitinib necessitate exploring novel and combination therapies such as apraglutide, neihulizumab, and other targeted agents, which have shown promising results in clinical trials. Additionally, nonpharmacologic approaches, including microbiotherapy, mesenchymal stromal cells, or alpha-1 antitrypsin, are also being investigated for their immunomodulatory potential. The future direction is to develop personalized treatment strategies that incorporate biomarkers and diverse therapeutic modalities. The aim is to enhance disease management, aiming for sustainable control and improved quality of life in patients facing the challenges of SR-aGVHD.

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