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A syndrome of immunologically mediated tissue damage that may occur following an allogeneic transplant, usually affecting the skin, liver, and GI tract. The onset is usually within one hundred days of transplantation or immunologic manipulation.
Biomarker and diagnostic research for acute graft versus host disease has been reported in the published literature.
1 FDA-approved treatment is available for acute graft versus host disease, including remestemcel-L-rknd (ryoncil, approved 2024). An additional 10 compounds hold orphan drug designation.
Brand Name | Generic Name | Mechanism | Approved | Market Status |
|---|---|---|---|---|
17 clinical trials registered, 7 recruiting. Interventions under study include drug therapy, other interventions, procedural interventions, and biologic therapy. Pipeline includes 3 PHASE3, 5 PHASE2, 5 PHASE1. Research is primarily sponsored by academic and government institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT05921305](https://clinicaltrials.gov/study/NCT05921305) |
Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 2:58 PM UTC
European rare disease database
Genetic and Rare Diseases Info Center
ryoncil |
remestemcel-L-rknd |
— |
2024 |
Available |
The following drugs have received orphan drug designation from the FDA for acute graft versus host disease. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor | Designated | Exclusivity End | Designation Status |
|---|---|---|---|---|---|
allogeneic-unrelated, CD4+ T cells transduced with lentiviral vector encoding full-length human interleukin-10 gene and a truncated (non-signaling) form of the human nerve growth factor receptor | allogeneic-unrelated, CD4+ T cells transduced with lentiviral vector encoding full-length human interleukin-10 gene and a truncated (non-signaling) form of the human nerve growth factor receptor | Tr1X, Inc. | 2025 | — | Designated |
tregalizumab | tregalizumab | T-Balance Therapeutics GmbH | 2023 | — | Designated |
Teduglutide | Teduglutide | 9 Meters Biopharma Inc. | 2020 | — | Designated |
defibrotide | defibrotide | Jazz Pharmaceuticals Ireland Limited | 2020 | — | Withdrawn |
acazicolcept | acazicolcept | Alpine Immune Sciences | 2020 | — | Designated |
recombinant human interleukin 22-human immunoglobulin Fc fusion protein | recombinant human interleukin 22-human immunoglobulin Fc fusion protein | Evive Biotechnology (Shanghai) Ltd. | 2019 | — | Designated |
itolizumab | itolizumab | Biocon Limited | 2019 | — | Designated |
Allogeneic mesenchymoangioblast-derived mesenchymal stem cells | Allogeneic mesenchymoangioblast-derived mesenchymal stem cells | Cynata Therapeutics Limited | 2018 | — | Designated |
Human alpha-1-antitrypsin | Human alpha-1-antitrypsin | Paul Maher, MD | 2017 | — | Designated |
Recombinant human monoclonal antibody against human complement component C5a | Recombinant human monoclonal antibody against human complement component C5a | Alexion Pharmaceuticals, Inc. | 2016 | — | Withdrawn |
Gene therapy approaches for acute graft versus host disease have been reported in the published literature.
17 trials found
Mini-dose MTX Plus Standard-dose Steroid for the Initial Treatment of Acute GVHD |
PHASE3 |
Peking University People's Hospital |
RECRUITING |
[NCT06904599](https://clinicaltrials.gov/study/NCT06904599) | Efficacy of Human Chorionic Gonadotropin in Acute GVHD Treatment | PHASE2 | Hospital Universitario Dr. Jose E. Gonzalez | RECRUITING |
[NCT06083129](https://clinicaltrials.gov/study/NCT06083129) | Phase III Study Comparing GVHD Prophylaxis With ATG-thymoglobulin to ATLG-grafalon in Elderly Patients With Acute Myeloid Leukemia or Myelodysplasic Syndrome and Receiving an Allogeneic Hematopoietic Stem Cell Transplantation With a 10/10 HLA Matched Unrelated Donor | PHASE3 | Assistance Publique - Hôpitaux de Paris | RECRUITING |
[NCT05443425](https://clinicaltrials.gov/study/NCT05443425) | Leflunomide in Combination With Steroids for the Treatment of Acute Graft-versus-Host Disease After Donor Stem Cell Transplant for Hematologic Malignancies | PHASE1 | City of Hope Medical Center | RECRUITING |
[NCT04373057](https://clinicaltrials.gov/study/NCT04373057) | Prebiotic Galacto-oligosaccharide and Acute GVHD | PHASE1 | University of Kansas Medical Center | RECRUITING |
255 publications have been identified in PubMed for acute graft versus host disease. Research spans Basic Science / Preclinical (22%), Clinical Trial Publication (21%), and Review / Meta-Analysis (20%).
Research Type | Count | % of Total |
|---|---|---|
Laboratory research | 55 | 22% |
Clinical study results | 54 | 21% |
Research summaries | 51 | 20% |
Disease patterns and progression | 42 | 16% |
Testing and diagnosis research | 26 | 10% |
New treatment approaches | 13 | 5% |
Patient case studies | 12 | 5% |
Other research | 2 | 1% |
Han JE (2026). [PMID: 41319282](https://pubmed.ncbi.nlm.nih.gov/41319282/). *Adv Sci (Weinh)*. [Gene Therapy / Novel Therapeutics]
Ghimire S (2026). [PMID: 41828729](https://pubmed.ncbi.nlm.nih.gov/41828729/). *Int J Mol Sci*. [Clinical Trial Publication]
Wei ZF (2026). [PMID: 42159898](https://pubmed.ncbi.nlm.nih.gov/42159898/). *Inflammopharmacology*. [Gene Therapy / Novel Therapeutics]
Mootoosamy C (2026). [PMID: 41775174](https://pubmed.ncbi.nlm.nih.gov/41775174/). *Leuk Res*. [Epidemiology / Natural History]
Niu Y (2026). [PMID: 41187423](https://pubmed.ncbi.nlm.nih.gov/41187423/). *International immunopharmacology*. [Basic Science / Preclinical]
Braun LM (2026). [PMID: 41802111](https://pubmed.ncbi.nlm.nih.gov/41802111/). *Blood*. [Epidemiology / Natural History]
Ulschmid CM (2026). [PMID: 41498421](https://pubmed.ncbi.nlm.nih.gov/41498421/). *Blood Adv*. [Epidemiology / Natural History]
Mahmoudjafari Z (2026). [PMID: 42180389](https://pubmed.ncbi.nlm.nih.gov/42180389/). *Ther Adv Hematol*. [Review / Meta-Analysis]
Zeiser R (2026). [PMID: 41858687](https://pubmed.ncbi.nlm.nih.gov/41858687/). *Transfus Med Hemother*. [Clinical Trial Publication]
Gohil D (2026). [PMID: 38584000](https://pubmed.ncbi.nlm.nih.gov/38584000/). *Br J Pharmacol*. [Basic Science / Preclinical]
AI-curated news mentioning acute graft versus host disease
Updated Feb 8, 2026
A recent case report highlights the occurrence of acute graft-versus-host disease following deceased-donor liver transplantation. This finding may inform future clinical practices and patient management strategies in transplant settings.