Hepatic veno-occlusive disease
diseaseOn this page
Also known as Budd Chiari Syndromehepatic Vodliver veno-occlusive diseasesinusoidal obstruction syndrome
Summary
Hepatic veno-occlusive disease (MONDO:0019514) is a disease and 23 clinical trials. Top therapeutic interventions include glutamine and defibrotide. A subtype of hepatic vascular disorder — broader associated-gene and molecular evidence is on the parent page (see Disease family below).
At a glance
- Prevalence: Unknown (Worldwide) [Orphanet-validated]
- Phenotypes (HPO): 12
- Clinical trials: 23
Clinical features
Signs & symptoms
Clinical features (HPO)
12 HPO clinical features (Orphanet curated; top 12 by frequency):
| HPO ID | Term | Frequency |
|---|---|---|
| HP:0000083 | Renal insufficiency | Very frequent (80-99%) |
| HP:0000952 | Jaundice | Very frequent (80-99%) |
| HP:0001541 | Ascites | Very frequent (80-99%) |
| HP:0002027 | Abdominal pain | Very frequent (80-99%) |
| HP:0002240 | Hepatomegaly | Very frequent (80-99%) |
| HP:0002878 | Respiratory failure | Very frequent (80-99%) |
| HP:0002910 | Elevated circulating hepatic transaminase concentration | Very frequent (80-99%) |
| HP:0003573 | Increased total bilirubin | Very frequent (80-99%) |
| HP:0004324 | Increased body weight | Very frequent (80-99%) |
| HP:0001928 | Abnormality of coagulation | Frequent (30-79%) |
| HP:0003645 | Prolonged partial thromboplastin time | Frequent (30-79%) |
| HP:0002480 | Hepatic encephalopathy | Very rare (<1-4%) |
Identifiers
Disease identifiers
| Field | Value |
|---|---|
| Canonical name | hepatic veno-occlusive disease |
| Mondo ID | MONDO:0019514 |
| MeSH | D006504 |
| Orphanet | 890 |
| DOID | DOID:0080177 |
| ICD-10-CM | K76.5 |
| ICD-11 | 762044088, 901464792 |
| NCIT | C26793 |
| SNOMED CT | 65617004 |
| UMLS | C0019156 |
| MedGen | 5514 |
| GARD | 0013004 |
| MedDRA | 10047216 |
| NORD | 879 |
| Is cancer (heuristic) | no |
Also known as: Budd Chiari Syndrome · hepatic Vod · liver veno-occlusive disease · sinusoidal obstruction syndrome
Disease family
This is a subtype of hepatic vascular 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 › digestive system disorder › hepatobiliary disorder › liver disorder › hepatic vascular disorder › hepatic veno-occlusive disease
Related subtypes (10): portal vein thrombosis, hepatic infarction, nutmeg liver, liver angiosarcoma, peliosis hepatis, portal hypertension, hepatic vein thrombosis, Budd-Chiari syndrome, portosinusoidal vascular disease, Fontan-associated liver disease
Subtypes (1): hepatic veno-occlusive disease-immunodeficiency syndrome
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, 2 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.
| Drug | Development status |
|---|---|
| Defibrotide Sodium | Approved (phase 4) |
| Dalteparin Sodium | Phase 3 (in late-stage trials) |
| Defibrotide | Phase 3 (in late-stage trials) |
Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Acetylcysteine.
Clinical trials & evidence
Clinical trials
Clinical trials: 23.
Phase distribution (across all retrieved trials)
| Phase | Trials |
|---|---|
| Not specified | 14 |
| PHASE2 | 5 |
| PHASE3 | 2 |
| PHASE4 | 1 |
| PHASE2/PHASE3 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT03963999 | PHASE4 | WITHDRAWN | Validating Ultrasound Biomarkers for Hepatic Sinusoidal Obstruction Syndrome in Pediatric Hematopoietic Cell Transplant Patients |
| NCT00272948 | PHASE2/PHASE3 | COMPLETED | Pediatric Trial Investigating the Incidence & Outcome of Veno-Occlusive Disease With the Prophylactic Use of Defibrotide |
| NCT00358501 | PHASE3 | COMPLETED | Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients |
| NCT00628498 | PHASE3 | COMPLETED | Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study |
| NCT05987124 | PHASE2 | RECRUITING | Defibrotide Dose-escalation for SOS Post-HSCT |
| NCT06258525 | PHASE2 | NOT_YET_RECRUITING | SAMe in Prevention of Oxaliplatin-associated Liver Injury |
| NCT01886248 | PHASE2 | UNKNOWN | Antithrombin-III for Patients With Hepatic Veno-occlusive Diseases Following Hematopoietic Stem Cell Transplantation |
| NCT04313036 | PHASE2 | UNKNOWN | 5-day Defibrotide Treatment for Hepatic SOS/VOD |
| NCT04712435 | PHASE2 | UNKNOWN | Efficacy of N-acetylcysteine Versus Placebo as Prophylaxis of Sinusoidal Obstruction Syndrome in Patients Undergoing Hematopoietic Stem Cell Transplantation |
| NCT03865589 | Not specified | ACTIVE_NOT_RECRUITING | Using Ultrasound Elastography to Predict Development of Hepatic Sinusoidal Obstruction Syndrome |
| NCT05782556 | Not specified | RECRUITING | Freiburg TIPS Registry |
| NCT06715046 | Not specified | RECRUITING | Cell Free DNA Profiling As a Tool to Monitor Clinically-Relevant Events in Allogeneic Hematopoietic Stem Cell Transplantation |
| NCT00143546 | Not specified | NO_LONGER_AVAILABLE | Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the Liver |
| NCT00563498 | Not specified | UNKNOWN | Effects on Glutamine on the Outcome of Allogeneic Bone Marrow Transplant Recipients |
| NCT00885950 | Not specified | COMPLETED | Prevention of the Hepatic Sinusoidal Obstruction Syndrome by Means of Anticoagulants |
| NCT02483481 | Not specified | COMPLETED | Using Ultrasound Elastography to Predict Development of SOS |
| NCT03032016 | Not specified | COMPLETED | European VOD Registry |
| NCT03132337 | Not specified | COMPLETED | Sinusoidal Obstruction Syndrome for Stem Cell Transplant Patients Biomarker Study |
| NCT03426358 | Not specified | COMPLETED | Liver Stiffness Measurement Predicts VOD/SOS Development (ELASTOVOD ITALIAN MULTICENTRIC STUDY) |
| NCT03858530 | Not specified | TERMINATED | Ultrasound Elastography to Predict Development of SOS |
| NCT04141735 | Not specified | COMPLETED | Detection of Sinusoidal Obstruction Syndrome With Ultrasound After Allogeneic HSCT |
| NCT04168788 | Not specified | UNKNOWN | Pharmacogenetic Study of Antimitotic Therapies Involved in Hepatic VOD in Children With Nephroblastoma or ALL |
| NCT06054451 | Not specified | UNKNOWN | Clinical Diagnosis and Pathological Spectrum of Porto-sinusoidal Vascular Disease in India |
Drugs tested across these trials (top 30)
| Molecule | Max phase | Trials referencing |
|---|---|---|
| GLUTAMINE | 4 | 1 |
| DEFIBROTIDE | 3 | 6 |
| CHEMBL5435500 | 0 | 1 |
Related Atlas pages
- Drugs: Glutamine, Defibrotide