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Chronic graft versus host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted donor cells attack the transplant recipient's body. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. The exact cause of chronic GVHD is unknown.It likely results from a complex immune-mediated interaction between the donor and recipient cells.Chronic GVHD is treated with prednisone or other similar anti-inflammatory or immunosuppressive medications.
Features include very common findings: Poor wound healing, Thickened skin, and Intermittent generalized erythematous papular rash; and common findings: Xerostomia, Recurrent corneal erosions, Photophobia, and Skin color changes (abnormality of skin pigmentation) and others. 53 total HPO annotations.
Organ System | Phenotype Count | Example Features |
|---|---|---|
Digestive system | 12 | Elevated circulating hepatic transaminase concentration, Ascites, Diarrhea |
Biomarker and diagnostic research for chronic graft versus host disease has been reported in the published literature.
2 FDA-approved treatments are available for chronic graft versus host disease, including AXATILIMAB-CSFR (NIKTIMVO, approved 2024) and IBRUTINIB (IMBRUVICA, approved 2022). An additional 1 compound holds orphan drug designation.
Brand Name | Generic Name | Mechanism | Approved | Market Status |
|---|---|---|---|---|
Phenotype severity distribution: 3 very common features, 13 common features.
25 clinical trials registered, 13 recruiting. Interventions under study include other interventions, drug therapy, medical devices, and procedural interventions. Pipeline includes 2 PHASE3, 8 PHASE2, 9 PHASE1. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT05675930](https://clinicaltrials.gov/study/NCT05675930) |
Data assembled from 6 of 12 sources · Last updated Sep 19, 2026, 6:05 PM UTC
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
European rare disease database
Genetic and Rare Diseases Info Center
Skin | 8 | Thickened skin, Intermittent generalized erythematous papular rash, Skin color changes (abnormality of skin pigmentation) |
Lungs and breathing | 8 | Bronchiectasis, Dyspnea, Pneumothorax |
Muscles | 3 | Muscle weakness, Flexion contracture, Myalgia |
Brain and nerves | 3 | Difficulty swallowing (dysphagia), Entrapment neuropathy, Pain |
Blood and immune system | 2 | Recurrent infections, Low blood cell counts (all types) (pancytopenia) |
Kidneys and urinary system | 2 | Blood in the urine (hematuria), Urinary bladder inflammation |
Bones and joints | 2 | Joint inflammation (arthritis), Arthralgia |
Eyes | 1 | Recurrent corneal erosions |
Lab test results | 1 | Elevated circulating hepatic transaminase concentration |
Growth and development | 1 | Weight loss |
Heart and blood vessels | 1 | Chest pain |
NIKTIMVO |
AXATILIMAB-CSFR |
— |
2024 |
Available |
IMBRUVICA | IBRUTINIB | — | 2022 | Available |
The following drugs have received orphan drug designation from the FDA for chronic 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 |
|---|---|---|---|---|---|
autologous lymphocytes depleted ex vivo of immunoreactive T cells using 4,5 | autologous lymphocytes depleted ex vivo of immunoreactive T cells using 4,5 | Kiadis Pharma Netherlands B.V. | 2008 | — | Withdrawn |
Gene therapy approaches for chronic graft versus host disease have been reported in the published literature.
25 trials found
A Study of Photobiomodulation (PBM) Therapy in People With Oral Graft-Versus-Host Disease (GVHD) After Stem Cell Transplant |
PHASE2 |
Memorial Sloan Kettering Cancer Center |
RECRUITING |
[NCT01804686](https://clinicaltrials.gov/study/NCT01804686) | A Long-term Extension Study of PCI-32765 (Ibrutinib) | PHASE3 | Janssen Research & Development, LLC | RECRUITING |
[NCT06247150](https://clinicaltrials.gov/study/NCT06247150) | Tissue Immune Landscape of Graft Versus Host Disease After Allogeneic Stem Cell Transplantation (TIL-GVHD) | NA | University Hospital, Bordeaux | RECRUITING |
[NCT06233110](https://clinicaltrials.gov/study/NCT06233110) | Ruxolitinib Plus Fostamatinib for Steroid Refractory cGvHD | PHASE1 | Stefanie Sarantopoulos, MD, PhD. | RECRUITING |
[NCT07302776](https://clinicaltrials.gov/study/NCT07302776) | TACrolimus Targeted Immunosuppression Cessation in ALlogeneic HCT | PHASE1 | Stanford University | RECRUITING |
278 publications have been identified in PubMed for chronic graft versus host disease. Kisho has analyzed 111 by research type. Research spans Review / Meta-Analysis (40%), Epidemiology / Natural History (19%), and Clinical Trial Publication (15%).
Research Type | Count | % of Total |
|---|---|---|
Research summaries | 44 | 40% |
Disease patterns and progression | 21 | 19% |
Clinical study results | 17 | 15% |
Laboratory research | 14 | 13% |
Patient case studies | 8 | 7% |
Other research | 3 | 3% |
New treatment approaches | 3 | 3% |
Testing and diagnosis research | 1 | 1% |
Malard F (2026). [PMID: 41795054](https://pubmed.ncbi.nlm.nih.gov/41795054/). *Bone Marrow Transplant*. [Review / Meta-Analysis]
Wolff D (2026). [PMID: 41207381](https://pubmed.ncbi.nlm.nih.gov/41207381/). *Transplant Cell Ther*. [Review / Meta-Analysis]
Choe H (2026). [PMID: 41238758](https://pubmed.ncbi.nlm.nih.gov/41238758/). *Bone Marrow Transplant*. [Review / Meta-Analysis]
Saadeh A (2026). [PMID: 41525301](https://pubmed.ncbi.nlm.nih.gov/41525301/). *Acta Haematol*. [Review / Meta-Analysis]
Gem H (2026). [PMID: 40905099](https://pubmed.ncbi.nlm.nih.gov/40905099/). *Haematologica*. [Epidemiology / Natural History]
Kong X (2026). [PMID: 41735346](https://pubmed.ncbi.nlm.nih.gov/41735346/). *Nat Commun*. [Basic Science / Preclinical]
Martens MJ (2026). [PMID: 41697751](https://pubmed.ncbi.nlm.nih.gov/41697751/). *J Clin Invest*. [Clinical Trial Publication]
Fujiki T (2026). [PMID: 41389151](https://pubmed.ncbi.nlm.nih.gov/41389151/). *J Immunol*. [Basic Science / Preclinical]
Rathje K (2026). [PMID: 41241260](https://pubmed.ncbi.nlm.nih.gov/41241260/). *Transplant Cell Ther*. [Review / Meta-Analysis]
Yang K (2025). [PMID: 40859356](https://pubmed.ncbi.nlm.nih.gov/40859356/). *BMC Med*. [Clinical Trial Publication]
AI-curated news mentioning chronic graft versus host disease
Updated Aug 26, 2026
Graviton BioScience reports positive Phase 1b trial results for GV101, a selective ROCK2 inhibitor, showing an 86.2% best overall response rate at 400 mg in chronic graft versus host disease patients. This therapy addresses significant unmet needs in a patient population that has failed multiple prior treatments.
A recent study discusses the need for standardized screening protocols for chronic graft-versus-host disease (GVHD) based on insights from the Engraft Learning Network. This research aims to improve early detection and management of chronic GVHD in patients.
A case report highlights the successful use of ruxolitinib in a patient with steroid-refractory chronic graft-versus-host disease (GvHD) affecting the central nervous system. This finding may inform future treatment strategies for similar cases.
A new proposal outlines diagnostic criteria for chronic graft-versus-host disease manifestations in the peripheral nervous system and muscles. This initiative aims to enhance the understanding and diagnosis of this complex condition.
Ibrutinib (Imbruvica) has received both Breakthrough Therapy and Orphan Drug designations for treating chronic graft-versus-host disease (cGVHD) as a monotherapy. This designation highlights its potential for patients who have not responded to previous therapies.