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Updated: Dec 20, 2025

Bone Marrow Transplantation Platform to Investigate the Role of Dendritic Cells in Graft-versus-Host Disease
Published on: March 17, 2020
Evolving Therapeutic Options for Chronic Graft-versus-Host Disease
Rebecca M Gonzalez1,2, Joseph Pidala1
1Department of Blood & Marrow Transplant and Cellular Immunotherapy (BMT CI), Moffitt Cancer Center, Tampa, Florida, USA.
Insights
Chronic graft-versus-host disease (cGVHD) significantly impacts allogeneic transplant patients despite treatment advances. New strategies, including novel agents and expanded cyclophosphamide use, are improving prevention and treatment outcomes for this late complication.
Area of Science:
- Hematology
- Immunology
- Oncology
Background:
- Chronic graft-versus-host disease (cGVHD) remains a major cause of morbidity and mortality in allogeneic transplant recipients.
- It is influenced by donor, patient, and hematopoietic cell transplant (HCT) factors, presenting significant post-transplant complications.
- Understanding cGVHD pathophysiology is crucial for developing improved clinical interventions.
Purpose of the Study:
- To review recent advances in understanding cGVHD pathophysiology.
- To highlight new approaches for the prevention and treatment of cGVHD.
- To discuss the impact of novel agents and expanded therapeutic strategies.
Main Methods:
- Review of current literature on cGVHD.
- Analysis of recent clinical trials and therapeutic advancements.
- Discussion of emerging biological insights into cGVHD.
Main Results:
- Posttransplantation cyclophosphamide shows promise beyond haploidentical HCTs, transforming outcomes.
- Novel agents are being investigated in combination with corticosteroids for upfront therapy.
- Ibrutinib is the first FDA-approved agent for cGVHD, marking a significant therapeutic advance.
- Several treatment options for steroid-refractory cGVHD are under investigation.
Conclusions:
- Advances in understanding cGVHD pathophysiology are driving innovation in prevention and treatment.
- Expanded use of posttransplantation cyclophosphamide and novel therapeutic agents offer improved outcomes.
- Future research focuses on mitigating steroid exposure and managing refractory disease.
Abstract:
Despite improvements in prevention and treatment of acute graft-versus-host disease (GVHD), chronic GVHD (cGVHD) remains a significant contributor to morbidity and mortality of allogeneic transplant patients. Chronic GVHD remains a leading cause of late complications posttransplant and is impacted by donor-, patient-, and transplant-related (hematopoietic cell transplant [HCT]) factors. Advances in the biological understanding of cGVHD have provided opportunities to improve clinical interventions for prevention and treatment. Expansion of posttransplantation cyclophosphamide beyond haploidentical HCTs has transformed alternative donor, matched, and mismatch GVHD outcomes and is currently being investigated in two upcoming clinical trials network prophylaxis studies. Although corticosteroids remain the cornerstone therapy, several clinical trials are prospectively investigating the utility of using novel agents in combination with corticosteroids as upfront therapy to mitigate prolonged steroid exposure. Several treatment options for patients with steroid-refractory cGVHD are currently being investigated, and advances have resulted in ibrutinib becoming the first cGVHD agent approved by the U.S. Food and Drug Administration. We review recent advances in understanding of cGVHD pathophysiology and new approaches for the prevention and treatment of cGVHD.
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