Graft versus host disease

Summary

Graft versus host disease (MONDO:0013730) is a disease and 550 clinical trials. Top therapeutic interventions include cyclophosphamide anhydrous, sirolimus, and busulfan. A subtype of immune system disorder — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Prevalence: 1-9 / 100 000 (Europe) [Orphanet-validated]
• Phenotypes (HPO): 41
• Clinical trials: 550

Clinical features

Epidemiology

Prevalence records

1 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Point prevalence	1-9 / 100 000	5	Europe	Validated

Signs & symptoms

Clinical features (HPO)

41 HPO clinical features (Orphanet curated; top 41 by frequency):

HPO ID	Term	Frequency
HP:0011123	Inflammatory abnormality of the skin	Very frequent (80-99%)
HP:0000155	Oral ulcer	Frequent (30-79%)
HP:0002014	Diarrhea	Frequent (30-79%)
HP:0002719	Recurrent infections	Frequent (30-79%)
HP:0002910	Elevated circulating hepatic transaminase concentration	Frequent (30-79%)
HP:0010280	Stomatitis	Frequent (30-79%)
HP:0040186	Maculopapular exanthema	Frequent (30-79%)
HP:0200041	Skin erosion	Frequent (30-79%)
HP:0200123	Chronic hepatitis	Frequent (30-79%)
HP:0000737	Irritability	Occasional (5-29%)
HP:0000952	Jaundice	Occasional (5-29%)
HP:0001369	Arthritis	Occasional (5-29%)
HP:0001433	Hepatosplenomegaly	Occasional (5-29%)
HP:0001649	Tachycardia	Occasional (5-29%)
HP:0002013	Vomiting	Occasional (5-29%)
HP:0002018	Nausea	Occasional (5-29%)
HP:0002027	Abdominal pain	Occasional (5-29%)
HP:0002090	Pneumonia	Occasional (5-29%)
HP:0002113	Pulmonary infiltrates	Occasional (5-29%)
HP:0002904	Hyperbilirubinemia	Occasional (5-29%)
HP:0002996	Limited elbow movement	Occasional (5-29%)
HP:0003155	Elevated circulating alkaline phosphatase concentration	Occasional (5-29%)
HP:0003202	Skeletal muscle atrophy	Occasional (5-29%)
HP:0004386	Gastrointestinal inflammation	Occasional (5-29%)
HP:0006467	Limited shoulder movement	Occasional (5-29%)
HP:0012156	Hemophagocytosis	Occasional (5-29%)
HP:0031123	Recurrent gastroenteritis	Occasional (5-29%)
HP:0031359	Cutaneous sclerotic plaque	Occasional (5-29%)
HP:0031452	Lichenoid skin lesion	Occasional (5-29%)
HP:0040189	Scaling skin	Occasional (5-29%)
HP:0100533	Inflammatory abnormality of the eye	Occasional (5-29%)
HP:0100537	Fasciitis	Occasional (5-29%)
HP:0100614	Myositis	Occasional (5-29%)
HP:0200119	Acute hepatitis	Occasional (5-29%)
HP:0000211	Trismus	Very rare (<1-4%)
HP:0000633	Decreased lacrimation	Very rare (<1-4%)
HP:0001508	Failure to thrive	Very rare (<1-4%)
HP:0002716	Lymphadenopathy	Very rare (<1-4%)
HP:0005198	Stiff interphalangeal joints	Very rare (<1-4%)
HP:0005679	Dupuytren contracture	Very rare (<1-4%)
HP:0009125	Lipodystrophy	Very rare (<1-4%)

Identifiers

Disease identifiers

Field	Value
Canonical name	graft versus host disease
Mondo ID	MONDO:0013730
MeSH	D006086
Orphanet	39812
DOID	DOID:0081267
ICD-10-CM	D89.81
ICD-11	437372167
NCIT	C3063
SNOMED CT	234646005
UMLS	C0018133
MedGen	9082
GARD	0016642
MedDRA	10018651
NORD	1203
Is cancer (heuristic)	no

Also known as: disease, graft-versus-host · disease, graft-vs-host · disease, homologous wasting · disease, runt · diseases, graft-versus-host · diseases, graft-vs-host · graft versus host disease · graft vs host disease · graft vs. host disease · graft-versus-host disease · graft-versus-host disease, susceptibility to · graft-versus-host diseases · graft-versus-host-disease · graft-vs-host disease · graft-VS-host diseases · GVH · GVHD · GvHD · homologous wasting disease · runt disease

Disease family

This is a subtype of immune system disorder. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.

Classification path: disease › human disease › disease by body system or component › immune system disorder › graft versus host disease

Related subtypes (46): hypersensitivity reaction disease, immune system cancer, immune system organ benign neoplasm, bone marrow disorder, thymus gland disorder, inborn error of immunity, leukocyte disorder, psoriasis, spondyloarthropathy, aggressive insulitis, benign insulitis, inflammatory bowel disease, autoimmune disease, TNF receptor 1-associated periodic fever syndrome, epidermodysplasia verruciformis, Vici syndrome, proteosome-associated autoinflammatory syndrome, hyperimmunoglobulinemia D with periodic fever, transcobalamin II deficiency, pyogenic arthritis-pyoderma gangrenosum-acne syndrome, granulomatosis with polyangiitis, autosomal recessive osteopetrosis 7, congenital sideroblastic anemia-B-cell immunodeficiency-periodic fever-developmental delay syndrome, Roifman syndrome, cryopyrin-associated periodic syndrome, anti-HLA hyperimmunization, acquired immunodeficiency, erythroderma desquamativum, autoinflammatory syndrome with pyogenic bacterial infection and amylopectinosis, familial Mediterranean fever, 22q11.2 deletion syndrome, T-cell large granular lymphocyte leukemia, twin to twin transfusion syndrome, immunodeficiency disease, immunoproliferative disorder, cytokine receptor deficiency, immunodeficiency-related disorder, phagocytic cell dysfunction, thrombocytopenic purpura, lymphoid system disorder, immune reconstitution inflammatory syndrome, growth hormone insensitivity with immune dysregulation 1, autosomal recessive, cytokine release syndrome, early-onset autoimmunity-autoinflammation-immunodeficiency syndrome, CADINS disease, autoinflammation, panniculitis, and dermatosis syndrome

Subtypes (2): acute graft versus host disease, chronic graft versus host disease

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).

Function

No pathway enrichment — requires an associated-gene cohort.

Therapeutics

Drugs indicated for this disease

1 approved, 45 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
ALLOGENEIC T CELLS GENETICALLY MODIFIED WITH A RETROVIRAL VECTOR ENCODING FOR A TRUNCATED FORM OF THE HUMAN LOW AFFINITY NERVE GROWTH FACTOR RECEPTOR (DELTALNGFR) AND THE HERPES SIMPLEX I VIRUS THYMIDINE KINASE (HSV-TK MUT2)	Approved (phase 4)
Acetylcysteine	Phase 3 (in late-stage trials)
Basiliximab	Phase 3 (in late-stage trials)
Beclomethasone Dipropionate	Phase 3 (in late-stage trials)
Begelomab	Phase 3 (in late-stage trials)
Belatacept	Phase 3 (in late-stage trials)
Budesonide	Phase 3 (in late-stage trials)
Busulfan	Phase 3 (in late-stage trials)
Clobetasol	Phase 3 (in late-stage trials)
Cyclosporine	Phase 3 (in late-stage trials)
Cytarabine	Phase 3 (in late-stage trials)
Daclizumab	Phase 3 (in late-stage trials)
Decitabine	Phase 3 (in late-stage trials)
Dexamethasone	Phase 3 (in late-stage trials)
Epoetin Beta	Phase 3 (in late-stage trials)
Everolimus	Phase 3 (in late-stage trials)
Filgrastim	Phase 3 (in late-stage trials)
Human Immunoglobulin G	Phase 3 (in late-stage trials)
Hydroxychloroquine	Phase 3 (in late-stage trials)
Ibrutinib	Phase 3 (in late-stage trials)
Iguratimod	Phase 3 (in late-stage trials)
Imatinib	Phase 3 (in late-stage trials)
Infliximab	Phase 3 (in late-stage trials)
Inolimomab	Phase 3 (in late-stage trials)
Itacitinib	Phase 3 (in late-stage trials)
Itolizumab	Phase 3 (in late-stage trials)
Methotrexate	Phase 3 (in late-stage trials)
Methoxsalen	Phase 3 (in late-stage trials)
Methylprednisolone	Phase 3 (in late-stage trials)
Mycophenolate Mofetil	Phase 3 (in late-stage trials)
Mycophenolic Acid	Phase 3 (in late-stage trials)
Pentostatin	Phase 3 (in late-stage trials)
Prednisolone	Phase 3 (in late-stage trials)
Prednisone	Phase 3 (in late-stage trials)
Recombinant Human Thrombopoietin	Phase 3 (in late-stage trials)
Remestemcel-L	Phase 3 (in late-stage trials)
Rituximab	Phase 3 (in late-stage trials)
Ruxolitinib	Phase 3 (in late-stage trials)
Semustine	Phase 3 (in late-stage trials)
Sirolimus	Phase 3 (in late-stage trials)
Sitagliptin	Phase 3 (in late-stage trials)
Sizavaleucel	Phase 3 (in late-stage trials)
Sodium Chloride	Phase 3 (in late-stage trials)
Sodium Citrate	Phase 3 (in late-stage trials)
Tacrolimus Anhydrous	Phase 3 (in late-stage trials)
Thiotepa	Phase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): .ALPHA.1-PROTEINASE INHIBITOR HUMAN, Abatacept, Acetaminophen, Albumin Human, Aldesleukin, Alemtuzumab, Belumosudil, Bortezomib, Brentuximab Vedotin, Cannabidiol, Carmustine, Clofarabine, Conestat Alfa, Defibrotide, Denileukin Diftitox, Diphenhydramine, Eltrombopag, Emapalumab, Etanercept, Etoposide, FE 203799, Fludarabine, Fludarabine Phosphate, Ganciclovir, Gavilimomab, INDIUM IN 111 IBRITUMOMAB TIUXETAN, Imlifidase, Lenalidomide, Leronlimab, Letermovir, Maraviroc, Melphalan, Natalizumab, Nilotinib, Nystatin, Obinutuzumab, Panobinostat, Piperacillin, Polymyxin B, Pomalidomide, Tazobactam, Tocilizumab, Treosulfan, Ustekinumab, Vancomycin, Visilizumab, YTTRIUM Y 90 IBRITUMOMAB TIUXETAN.

Clinical trials & evidence

Clinical trials

Clinical trials: 550.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	179
Not specified	157
PHASE1/PHASE2	65
PHASE1	65
PHASE3	51
PHASE4	15
PHASE2/PHASE3	10
EARLY_PHASE1	8

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02386800	PHASE4	ACTIVE_NOT_RECRUITING	CINC424A2X01B Rollover Protocol
NCT03229200	PHASE4	ACTIVE_NOT_RECRUITING	Extended Treatment Protocol for Subjects Continuing to Benefit From Ibrutinib.
NCT06824103	PHASE4	RECRUITING	Study of Efficacy and Safety of Ruxolitinib in Chinese Participants With Corticosteroid-refractory Chronic Graft vs. Host Disease
NCT07240649	PHASE4	NOT_YET_RECRUITING	Outcomes From Hyperbaric Oxygen (HBO2) Treatment for Emerging Indications
NCT07569653	PHASE4	RECRUITING	Letermovir Prophylaxis Duration Guided by CMV-Specific T-cell Monitoring After Allo-HSCT.
NCT00003398	PHASE4	COMPLETED	Bone Marrow Transplantation in Treating Patients With Hematologic Cancer
NCT00044720	PHASE4	COMPLETED	Study Evaluating Sirolimus in End Stage Renal Disease in High Risk Kidney Transplant Recipients
NCT00195429	PHASE4	COMPLETED	A Study Comparing the Withdrawal of Steroids or Tacrolimus in Kidney Transplant Recipients
NCT00195468	PHASE4	COMPLETED	Study Comparing Cyclosporine Dose Reduction vs. Cyclosporine Elimination in Kidney Transplant Recipients Taking Sirolimus
NCT00195481	PHASE4	COMPLETED	Study Evaluating Sirolimus in Kidney Transplant Recipients in India
NCT00574470	PHASE4	COMPLETED	Anti Cytokine Combination Therapy With Daclizumab and Infliximab for Treatment of Steroid Refractory Acute GVHD
NCT01282879	PHASE4	TERMINATED	Evaluation of Antifungal Prophylaxis on Graft-versus-host Disease (GVHD) Patients
NCT02342145	PHASE4	COMPLETED	Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major
NCT02660684	PHASE4	COMPLETED	Study to Assess the Safety and Efficacy of Tacrolimus (Prograf Capsule/Injection) and Methotrexate (MTX) Combination Therapy for Prevention of Graft Versus Host Disease (GVHD) in Patients Who Received Peripheral Hematopoietic Stem Cell Transplantation From a Sibling Donor
NCT04728646	PHASE4	WITHDRAWN	Evaluation of Dextenza in Patients With Ocular GVHD and Effects on Ocular Surface Disease Outcomes
NCT03818334	PHASE2/PHASE3	RECRUITING	Post Transplant Cyclophosphamide in Matched Unrelated Donor Stem Cell Transplantation for Hematological Malignancies
NCT05153226	PHASE3	ACTIVE_NOT_RECRUITING	GvHD Prophylaxis in Unrelated Donor HCT: Randomized Trial Comparing PTCY Versus ATG
NCT06083129	PHASE3	RECRUITING	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
NCT06615050	PHASE3	RECRUITING	A Study of Tacrolimus/Methotrexate/Ruxolitinib Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation (BMT CTN 2203)
NCT06682169	PHASE3	RECRUITING	Evaluation of Rovadicitinib Compared to the Protocol Selected by Researchers in Third Line and Subsequent Studies of Moderate to Severe Chronic Graft-versus-host Disease
NCT06705062	PHASE3	RECRUITING	Study Comparing Reduced Versus Standard Dose Post-transplantation Cyclophosphamide in Combination With Post-engraftment Anti-thymoglobin as Graft Versus Host Disease Prophylaxis in Alternative Donor Peripheral Stem Cell Transplantation
NCT07297914	PHASE2/PHASE3	NOT_YET_RECRUITING	Framework for Optimizing, Refining, and Unifying Management of HSCT in Pediatric ALL
NCT00000591	PHASE3	COMPLETED	T-Cell Depletion in Unrelated Donor Marrow Transplantation
NCT00001561	PHASE3	COMPLETED	Active Immunization of Sibling Bone Marrow Transplant Donors Against Purified Myeloma Protein of the Recipient Undergoing Allogeneic Bone Marrow Transplantation
NCT00002456	PHASE3	COMPLETED	Graft-Versus-Host Disease Prevention in Treating Patients Who Are Undergoing Bone Marrow Transplantation
NCT00003056	PHASE3	TERMINATED	Prevention of Graft-Versus-Host Disease in Patients With Advanced Leukemia or Lymphoma Who Are Eligible for Peripheral Stem Cell Transplantation
NCT00003414	PHASE3	COMPLETED	Graft-Versus-Host Disease in Treating Patients With Recurrent or Refractory Lymphoma or Hodgkin’s Disease
NCT00004255	PHASE2/PHASE3	COMPLETED	Treatment of Bone Marrow to Prevent Graft-Versus-Host Disease in Patients With Acute or Chronic Leukemia Undergoing Bone Marrow Transplantation
NCT00006451	PHASE3	WITHDRAWN	Bone Marrow Transplantation in Treating Patients With Leukemia
NCT00031824	PHASE3	COMPLETED	Hydroxychloroquine in Treating Patients With Newly Diagnosed Chronic Graft-Versus-Host Disease
NCT00035880	PHASE2/PHASE3	COMPLETED	Study Comparing ABX-CBL (Monoclonal Antibody) Versus Atgam in Patients With Steroid Resistant Acute Graft Versus Host Disease
NCT00037531	PHASE3	COMPLETED	Study Evaluating Sirolimus (Rapamune™) in Solid Organ Transplant Recipients
NCT00043147	PHASE3	COMPLETED	Beclomethasone Plus Prednisone in Treating Patients With Graft-Versus-Host Disease
NCT00053976	PHASE3	COMPLETED	Methylprednisolone With or Without Daclizumab in Treating Patients With Acute Graft-Versus-Host Disease
NCT00117702	PHASE2/PHASE3	TERMINATED	Prevention of the Graft-Versus-Host-Disease in Patients After Stem Cell Transplantation With Tacrolimus and Everolimus
NCT00128739	PHASE3	COMPLETED	Treatment of Gastro-Intestinal and/or Hepatic Graft Versus Host Disease With Budesonide in Patients Following Peripheral Blood Stem Cell Transplantation
NCT00130754	PHASE3	COMPLETED	Thymoglobuline in Non-myeloablative Allogeneic Stem-cell Transplantation
NCT00167947	PHASE3	COMPLETED	Study Evaluating Sirolimus in Kidney Transplant Recipients.
NCT00179855	PHASE2/PHASE3	COMPLETED	Extracorporeal Photopheresis for Acute Graft Versus Host Disease
NCT00180089	PHASE3	COMPLETED	Budesonide for Prevention of Acute Gastrointestinal GVHD Following Allogenic Stem Cell Transplantation

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
CYCLOPHOSPHAMIDE ANHYDROUS	4	56
SIROLIMUS	4	7
BUSULFAN	4	6
RUXOLITINIB	4	5
THIOTEPA	4	5
BECLOMETHASONE DIPROPIONATE	4	4
IBRUTINIB	4	4
DACLIZUMAB	4	3
PANOBINOSTAT	4	3
BUDESONIDE	4	2
CYCLOSPORINE	4	2
FILGRASTIM	4	2
METHOTREXATE	4	2
TACROLIMUS ANHYDROUS	4	2
ALDESLEUKIN	4	1
ALEFACEPT	4	1
CLOBETASOL PROPIONATE	4	1
EVEROLIMUS	4	1
FLUDARABINE PHOSPHATE	4	1
HYDROXYCHLOROQUINE	4	1
INFLIXIMAB	4	1
ITRACONAZOLE	4	1
MYCOPHENOLATE MOFETIL	4	1
MYCOPHENOLATE SODIUM	4	1
OXYGEN	4	1
PREDNISONE	4	1
BEGELOMAB	3	1
CLOBETASOL	3	1
GAVILIMOMAB	3	1
REGRAMOSTIM	3	1