Overview
Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a condition where the tiny blood vessels inside the liver become blocked or damaged. This blockage restricts blood flow out of the liver, causing it to swell and not work properly. The disease most commonly occurs as a complication after bone marrow or stem cell transplantation, particularly when patients receive high-dose chemotherapy or radiation therapy as part of their conditioning treatment. It can also be caused by certain toxins found in herbal teas or bush teas containing pyrrolizidine alkaloids, and rarely by other medications. The main symptoms include painful swelling of the liver, sudden weight gain from fluid retention, yellowing of the skin and eyes (jaundice), and a buildup of fluid in the belly (ascites). In severe cases, the disease can lead to multi-organ failure, including kidney problems and difficulty breathing. Symptoms typically appear within the first few weeks after transplant. Treatment depends on severity. Mild cases may resolve on their own with supportive care such as managing fluid balance and pain. For more severe cases, a medication called defibrotide (Defitelio) has been approved and can significantly improve outcomes. Early recognition and treatment are critical because severe VOD can be life-threatening. Advances in prevention strategies and earlier diagnosis have improved survival rates over the years.
Also known as:
Key symptoms:
Painful or tender enlarged liverYellowing of the skin and eyes (jaundice)Rapid weight gain from fluid retentionSwollen belly from fluid buildup (ascites)Dark-colored urineNausea and vomitingFatigue and weaknessDecreased urine outputSwelling in the legs and feetConfusion or mental fogginess in severe casesEasy bruising or bleedingDifficulty breathingLow platelet counts that do not respond to transfusions
Clinical phenotype terms (12)— hover any for plain English
Variable
Can be inherited in different ways depending on the underlying gene
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
6 eventsNew York Medical College — PHASE2
University of Turin, Italy
Beijing Tsinghua Chang Gung Hospital
Duke University
Children's Mercy Hospital Kansas City — NA
Defitelio: FDA approved
For the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT).
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableDefitelio
For the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopo…
For the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT).
Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Hepatic veno-occlusive disease.
Community
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Start the conversation →Latest news about Hepatic veno-occlusive disease
Disease timeline:
New recruiting trial: Cell Free DNA Profiling As a Tool to Monitor Clinically-Relevant Events in Allogeneic Hematopoietic Stem Cell Transplantation
A new clinical trial is recruiting patients for Hepatic veno-occlusive disease
New recruiting trial: Defibrotide Dose-escalation for SOS Post-HSCT
A new clinical trial is recruiting patients for Hepatic veno-occlusive disease
New recruiting trial: A Prospective Registry of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS).
A new clinical trial is recruiting patients for Hepatic veno-occlusive disease
New recruiting trial: Differential Impact of Pringle and Portal Vein Occlusion on Myocardial Injury After Non-Cardiac Surgeries.
A new clinical trial is recruiting patients for Hepatic veno-occlusive disease
Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Questions for your doctor
Bring these to your next appointment
- Q1.How severe is my VOD, and what does that mean for my treatment plan?,Am I a candidate for defibrotide treatment, and when would it be started?,What are the signs that my condition is getting worse, and when should I seek emergency care?,What are my chances of full recovery, and how long might recovery take?,Are there any dietary or fluid restrictions I need to follow?,Could this condition cause long-term liver damage, and will I need ongoing liver monitoring?,If I need another transplant in the future, what can be done to prevent VOD from happening again?
Common questions about Hepatic veno-occlusive disease
What is Hepatic veno-occlusive disease?
Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a condition where the tiny blood vessels inside the liver become blocked or damaged. This blockage restricts blood flow out of the liver, causing it to swell and not work properly. The disease most commonly occurs as a complication after bone marrow or stem cell transplantation, particularly when patients receive high-dose chemotherapy or radiation therapy as part of their conditioning treatment. It can also be caused by certain toxins found in herbal teas or bush teas containing pyrrolizidine alkaloi
Are there clinical trials for Hepatic veno-occlusive disease?
Yes — 4 recruiting clinical trials are currently listed for Hepatic veno-occlusive disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Hepatic veno-occlusive disease?
25 specialists and care centers treating Hepatic veno-occlusive disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.