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Hepatic Veno-occlusive Disease

Disease Details

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Description: Hepatic veno-occlusive disease is a condition in which the small veins in the liver are obstructed, leading to liver damage and potentially liver failure.
Type: Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome, is typically classified as a vascular disease affecting the liver. The genetic form of this disease can be inherited in an autosomal recessive manner.
Signs And Symptoms: Features of hepatic veno-occlusive disease include weight gain, tender enlargement of the liver, ascites, and yellow discoloration of the skin; it often is associated with acute kidney failure.
Prognosis: Mild disease has a risk of death of about 10% while moderate disease has a risk of death of 20%. When it occurs as a result of bone marrow transplant and multiorgan failure is present, the risk of death is greater than 80%.
Onset: Hepatic veno-occlusive disease (VOD) typically presents with symptoms within the first 2-3 weeks following an inciting event, such as high-dose chemotherapy or bone marrow transplantation. However, onset can vary, with some cases appearing several weeks later.
Prevalence: The prevalence of hepatic veno-occlusive disease (HVOD), also known as sinusoidal obstruction syndrome (SOS), varies depending on the population and underlying risk factors, such as those undergoing hematopoietic stem cell transplantation (HSCT). It is estimated that HVOD affects approximately 5-60% of HSCT patients, with the wide range reflecting differences in conditioning regimens, patient populations, and diagnostic criteria. The disease is much rarer in the general population.
Epidemiology: Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a rare condition characterized by damage to the small hepatic veins, leading to liver dysfunction. It most commonly occurs after hematopoietic stem cell transplantation (HSCT), with an incidence ranging from 5% to 60% depending on risk factors and conditioning regimens. VOD can also occur following certain chemotherapies, radiation therapy, and ingestion of toxic pyrrolizidine alkaloids found in some herbal remedies. Moreover, children and individuals with pre-existing liver disease are at higher risk. Mortality rates vary significantly, with severe cases exhibiting high fatality rates if not treated promptly. Data on the general population are limited due to its rarity outside the context of transplantation and specific toxin exposures.
Intractability: Hepatic veno-occlusive disease (HVOD), also known as sinusoidal obstruction syndrome (SOS), can be challenging to treat, especially if diagnosed late. The disease involves blockage of small hepatic veins, leading to liver damage. The severity can vary from mild, self-limiting cases to severe, life-threatening instances. Early detection and management are crucial, and treatment options like defibrotide can improve outcomes. However, severe cases can be intractable and may require intensive interventions and supportive care.
Disease Severity: Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), can vary in severity. It ranges from mild, with minimal symptoms and normal liver function, to severe, where significant liver damage leads to symptoms such as jaundice, ascites, painful hepatomegaly, and multi-organ failure. Severe cases of VOD can be life-threatening and require urgent medical intervention. Nan (not a number) in this context is not applicable.
Healthcare Professionals: Disease Ontology ID - DOID:0080177
Pathophysiology: In the bone marrow transplant setting, hepatic veno-occlusive disease is felt to be due to injury to the hepatic venous endothelium from the conditioning regimen. Toxic agents causing veno-occlusive disease include plants as well as the medication cyclophosphamide.
Carrier Status: Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is not typically associated with a genetic carrier status. It refers to a condition characterized by the blockage of small veins in the liver. This condition can be triggered by various factors, including high-dose chemotherapy, bone marrow transplantation, or exposure to certain toxins. Genetic predisposition can play a role, but carrier status is not a standard concept in the context of hepatic VOD as it is for single-gene inherited disorders.
Mechanism: Hepatic veno-occlusive disease (HVOD), also known as sinusoidal obstruction syndrome (SOS), primarily affects the liver and is characterized by the occlusion of small hepatic veins. The mechanism involves endothelial cell damage in the liver sinusoids, followed by the activation of various cellular pathways that lead to fibrosis and occlusion of the small hepatic veins.

**Molecular Mechanisms:**
1. **Endothelial Cell Injury:** Chemotherapy, radiation therapy, or toxins (like pyrrolizidine alkaloids) damage the endothelial cells lining the liver sinusoids. This initiates the disease process.
2. **Cytokine Release:** Injured endothelial cells release cytokines such as TNF-α, IL-1, and other pro-inflammatory molecules.
3. **Expression of Adhesion Molecules:** Increased expression of adhesion molecules (e.g., VCAM-1, ICAM-1) on endothelial cells facilitates leukocyte adhesion and infiltration.
4. **Coagulation Cascade Activation:** Endothelial damage leads to the activation of the coagulation cascade, resulting in fibrin deposition and micro-thrombi formation.
5. **Oxidative Stress:** Increased oxidative stress contributes to further endothelial damage and inflammation.
6. **Fibrosis:** Stellate cell activation and subsequent fibrosis occur, leading to the obliteration of small hepatic veins.
7. **Hepatocyte Ischemia:** Resulting impairment in blood flow causes ischemia and necrosis of hepatocytes.

This cascade of molecular events culminates in the clinical manifestations of HVOD, which include hepatomegaly, jaundice, ascites, and potentially liver failure. Treatment focuses on managing symptoms and preventing further endothelial damage.
Treatment: Treatment generally includes supportive care including pain management and possibly diuretics. In those with severe disease due to a bone marrow transplant, defibrotide is a proposed treatment. It has been approved for use in severe cases in Europe and the United States. A placebo controlled trial, however, has not been done as of 2016.
Compassionate Use Treatment: For hepatic veno-occlusive disease (HVOD), also known as sinusoidal obstruction syndrome (SOS), compassionate use treatments and off-label or experimental options may include:

1. **Defibrotide**: This is often used on a compassionate use basis or as an investigational agent, particularly for severe cases of HVOD/SOS. It has been shown to improve survival in patients with severe forms of the disease.

2. **Anticoagulants and thrombolytics**: Agents like low-molecular-weight heparin or tissue plasminogen activator (tPA) have been explored off-label, though their efficacy and safety profiles in HVOD are not well-established.

3. **Supportive care**: Advanced supportive measures including paracentesis for ascites, and in some cases, renal replacement therapy for hepatorenal syndrome, though these are adjunctive rather than direct treatments for the disease.

Please consult with a healthcare provider for further guidance and individual assessment.
Lifestyle Recommendations: For hepatic veno-occlusive disease (VOD), lifestyle recommendations can help manage symptoms and support overall liver health:

1. Avoid Alcohol: Alcohol can worsen liver damage and should be strictly avoided.
2. Balanced Diet: Eat a diet rich in fruits, vegetables, lean proteins, and whole grains. Avoid fried and fatty foods to reduce liver strain.
3. Hydration: Drink plenty of water to support kidney function and overall health.
4. Avoid Toxins: Limit exposure to chemicals that could damage the liver. Use medications only as prescribed and avoid over-the-counter drugs that could be harmful.
5. Regular Check-ups: Maintain regular follow-up with healthcare providers to monitor liver function and overall health.
6. Physical Activity: Engage in regular, moderate exercise to improve general well-being but avoid strenuous activities if advised by your healthcare provider.
7. Weight Management: Maintain a healthy weight to prevent additional stress on the liver.

Consulting a healthcare provider for personalized recommendations is strongly advised.
Medication: For hepatic veno-occlusive disease (VOD), there are limited medication options. One of the primary treatments is defibrotide, which is specifically approved for the treatment of severe VOD. Defibrotide works by protecting the cells lining the blood vessels and reducing inflammation and clot formation. Treatment typically also includes supportive care such as managing fluid balance, avoiding hepatotoxic drugs, and addressing any organ dysfunction that may occur. For milder cases, treatment may be mostly supportive without specific medications.
Repurposable Drugs: Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a condition characterized by the blockage of small veins in the liver. For repurposable drugs, defibrotide is a medication that has shown promise. It is not originally developed for VOD but has been repurposed to treat and prevent this condition by protecting the endothelial cells and reducing inflammation.
Metabolites: Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is characterized by the occlusion of small hepatic veins. Specific metabolites involved in its pathogenesis include pyrroles, which are reactive metabolites of pyrrolizidine alkaloids found in certain plants. These metabolites can cause endothelial cell damage in the liver. Without further context or specific metabolites in question (as "nan" is ambiguous), it is difficult to provide additional detail. Generally, damage to hepatic venules can lead to fibrosis, hepatic congestion, and ultimately liver failure if not managed promptly.
Nutraceuticals: For hepatic veno-occlusive disease (HVOD), also known as sinusoidal obstruction syndrome, the role of nutraceuticals has not been well established. Nutraceuticals generally refer to food-derived products that offer health benefits, but their specific effects on HVOD are largely unproven. Main treatments typically focus on supportive care to manage symptoms and complications.
Peptides: Hepatic veno-occlusive disease (HVOD), also known as sinusoidal obstruction syndrome (SOS), is a condition characterized by blockage of the small veins in the liver. It often occurs as a complication of high-dose chemotherapy or radiation therapy before bone marrow transplantation.

**Peptides**:
In the context of hepatic veno-occlusive disease, peptides are being investigated for their potential therapeutic benefits. For example, defibrotide is a polydisperse oligonucleotide mixture that has shown promise in treating HVOD by protecting the vascular endothelium and promoting fibrinolysis, thereby reducing clot formation and improving the blood flow in the small hepatic veins.

**Nan**:
The term "nan" is not directly associated with hepatic veno-occlusive disease. If "nan" was meant to refer to nanotechnology or nanoparticles, these areas are an emerging field of research. Nanotechnology could potentially be used in the future to deliver targeted therapies to the liver, enhancing the efficacy and reducing the side effects of treatment for HVOD. However, this research is still in exploratory stages and not yet widely applied in clinical practice for HVOD.