Autoimmune Thrombocytopenic Purpura
Disease Details
Family Health Simplified
- Description
- Autoimmune thrombocytopenic purpura (ITP) is a disorder where the immune system mistakenly attacks and destroys platelets, leading to excessive bruising and bleeding.
- Type
- Autoimmune thrombocytopenic purpura (ITP) is primarily an acquired autoimmune disorder, not typically associated with a specific type of genetic transmission. While there might be some genetic predispositions that can influence susceptibility, ITP is not inherited in a simple Mendelian fashion.
- Signs And Symptoms
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Signs of ITP include the spontaneous formation of bruises (purpura) and petechiae (tiny bruises), especially on the extremities. Additionally, bleeding from the nostrils and/or gums, as well as menorrhagia (excessive menstrual bleeding), may occur if the platelet count falls below 20,000 per μl. A platelet count below 10,000 per μl can lead to the spontaneous formation of hematomas (blood masses) in the mouth or on other mucous membranes. Furthermore, bleeding time from minor lacerations or abrasions is usually prolonged.
In cases where platelet counts drop to extremely low levels (<5,000 per μl), serious and potentially fatal complications may arise. These complications include subarachnoid or intracerebral hemorrhage (bleeding inside the skull or brain), lower gastrointestinal bleeding, or other internal bleeding. A person with ITP with an extremely low platelet count is susceptible to internal bleeding resulting from blunt abdominal trauma, such as in a motor vehicle crash. These complications are unlikely to occur when the platelet count is less than 20,000 per μl. - Prognosis
- In general patients with acute ITP will only rarely have life-threatening bleeding. most of the patients ultimately have stable but lower platelet counts which is hemostatic for a person. Unlike in pediatric patients who can be cured, most of the adults will run a chronic course even after splenectomy.
- Onset
- Autoimmune thrombocytopenic purpura (ITP) can have a sudden or gradual onset. It may develop quickly over a few days to weeks or more slowly over several months. The exact timing can vary significantly from person to person.
- Prevalence
- The prevalence of autoimmune thrombocytopenic purpura (also known as immune thrombocytopenia, ITP) varies. It is estimated at approximately 3.3 cases per 100,000 adults annually. In children, the annual incidence is about 1.9 to 6.4 cases per 100,000, with a slight predilection for females in adults and no strong gender bias in children.
- Epidemiology
- A normal platelet count is considered to be in the range of 150,000–450,000 per microlitre (μl) of blood for most healthy individuals. Hence one may be considered thrombocytopenic below that range, although the threshold for a diagnosis of ITP is not tied to any specific number.The incidence of ITP is estimated at 50–100 new cases per million per year, with children accounting for half of that number. At least 70 percent of childhood cases will end up in remission within six months, even without treatment. Moreover, a third of the remaining chronic cases will usually remit during follow-up observation, and another third will end up with only mild thrombocytopenia (defined as a platelet count above 50,000). A number of immune related genes and polymorphisms have been identified as influencing predisposition to ITP, with FCGR3a-V158 allele and KIRDS2/DL2 increasing susceptibility and KIR2DS5 shown to be protective.ITP is usually chronic in adults and the probability of durable remission is 20–40 percent. The male to female ratio in the adult group varies from 1:1.2 to 1.7 in most age ranges (childhood cases are roughly equal for both sexes) and the median age of adults at the diagnosis is 56–60. The ratio between male and female adult cases tends to widen with age. In the United States, the adult chronic population is thought to be approximately 60,000—with women outnumbering men approximately 2 to 1, which has resulted in ITP being designated an orphan disease.The mortality rate due to chronic ITP varies but tends to be higher relative to the general population for any age range. In a study conducted in Great Britain, it was noted that ITP causes an approximately 60 percent higher rate of mortality compared to sex- and age-matched subjects without ITP. This increased risk of death with ITP is largely concentrated in the middle-aged and elderly. Ninety-six percent of reported ITP-related deaths were individuals 45 years or older. No significant difference was noted in the rate of survival between males and females.
- Intractability
- Autoimmune thrombocytopenic purpura (ITP) can be challenging to treat in some cases, but it is not universally intractable. Treatments such as corticosteroids, immunoglobulins, and newer therapies can lead to remission in many patients. However, some individuals may experience chronic or refractory ITP that is difficult to manage and may require ongoing or additional therapeutic strategies.
- Disease Severity
- Autoimmune thrombocytopenic purpura (often referred to as idiopathic thrombocytopenic purpura or ITP) varies in severity among individuals. Severity can range from mild, with few symptoms, to severe, with significant bleeding issues. The condition is characterized by a low platelet count, which can lead to easy or excessive bruising and bleeding. In severe cases, it can cause internal bleeding, which may be life-threatening and require urgent medical attention.
- Healthcare Professionals
- Disease Ontology ID - DOID:8924
- Pathophysiology
- Autoimmune thrombocytopenic purpura (ITP) is a disorder characterized by an abnormally low platelet count. The pathophysiology involves the immune system mistakenly identifying the body's platelets as foreign. The immune system produces autoantibodies against these platelets, marking them for destruction. This destruction primarily occurs in the spleen, leading to thrombocytopenia. The reduced platelet count results in impaired blood clotting, manifesting as purpura (bruising), petechiae (small red or purple spots), and increased bleeding tendency.
- Carrier Status
- Autoimmune thrombocytopenic purpura (ATP), also known as immune thrombocytopenic purpura (ITP), does not involve a carrier status. It is an autoimmune condition where the immune system mistakenly attacks and destroys platelets, which are crucial for blood clotting. The disease is not inherited in a typical carrier sense like some genetic disorders.
- Mechanism
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Autoimmune thrombocytopenic purpura (ITP) is a disorder characterized by the immune system attacking and destroying platelets, which are essential for normal blood clotting. This leads to bleeding and bruising.
Mechanism:
In ITP, autoantibodies, typically of the IgG type, are produced against platelet surface antigens, such as glycoproteins IIb/IIIa or Ib/IX. These autoantibodies bind to platelets, marking them for destruction. The platelets are then recognized and ingested by macrophages in the spleen and liver through Fc receptor-mediated phagocytosis. This process reduces the number of circulating platelets, causing thrombocytopenia.
Molecular Mechanisms:
1. **Autoantibody Production**: Dysfunction in regulatory T cells (Tregs) or an imbalance between Th17 cells and Tregs can lead to loss of immune tolerance and production of autoantibodies against platelet surface antigens.
2. **Fc Receptor-Mediated Phagocytosis**: Autoantibody-coated platelets engage Fc gamma receptors on macrophages, leading to phagocytosis and destruction of the platelets in the spleen and liver.
3. **Complement Activation**: Autoantibodies can activate the complement system, leading to the formation of the membrane attack complex, which can lyse platelets directly.
4. **Cytokines and Inflammatory Mediators**: Elevated levels of inflammatory cytokines such as IL-17 and IFN-γ can amplify the immune response, contributing to the ongoing destruction of platelets.
Understanding these mechanisms helps in developing therapeutic strategies for ITP, such as immunosuppressive drugs, intravenous immunoglobulin (IVIG), and splenectomy. - Treatment
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With rare exceptions, there is usually no need to treat based on platelet counts. Many older recommendations suggested a certain platelet count threshold (usually somewhere below 20.0/µl) as an indication for hospitalization or treatment. Current guidelines recommend treatment only in cases of significant bleeding.
Treatment recommendations sometimes differ for adult and pediatric ITP. - Compassionate Use Treatment
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Autoimmune thrombocytopenic purpura (ITP), also known as immune thrombocytopenia, largely involves standard treatments, but there are compassionate use, off-label, or experimental options available for certain patients.
1. **Compassionate Use Treatments**: This is typically considered when patients have exhausted standard treatment options. It can include investigational drugs not yet approved by regulatory agencies. These treatments require special approval and are accessed on a case-by-case basis.
2. **Off-label Treatments**:
- **Rituximab**: Originally approved for B-cell non-Hodgkin lymphoma and rheumatoid arthritis, it is used off-label to reduce antibody production against platelets.
- **Eltrombopag and Romiplostim**: Thrombopoietin receptor agonists approved for chronic ITP can sometimes be used in acute cases off-label both in adults and children.
3. **Experimental Treatments**:
- **Fostamatinib**: Originally for rheumatoid arthritis, it is being investigated for ITP based on its ability to inhibit spleen tyrosine kinase, which is involved in platelet destruction.
- **Splenectomy alternatives**: Research on less invasive procedures or new pharmacological agents to achieve long-term remission.
Participation in clinical trials might also be an option for accessing novel therapies in experimental phases, and patients should consult with their healthcare provider to explore these possibilities. - Lifestyle Recommendations
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Lifestyle recommendations for autoimmune thrombocytopenic purpura (ITP) focus on managing symptoms and avoiding activities that could exacerbate the condition. Key suggestions include:
1. **Avoid Contact Sports and High-Risk Activities**: To prevent injuries that could lead to bleeding.
2. **Monitor and Report Symptoms**: Keep track of any unusual bruising, bleeding, or petechiae and report these to your healthcare provider.
3. **Healthy Diet**: Maintain a balanced diet to support overall health and immune function. Avoid alcohol as it can affect platelet function.
4. **Medication Management**: Take medications as prescribed and avoid over-the-counter medications like aspirin or ibuprofen, which can impair platelet function.
5. **Regular Check-ups**: Have consistent medical follow-ups to monitor your platelet counts and overall health.
6. **Stress Management**: Incorporate stress-reducing practices such as meditation, yoga, or gentle exercise to help manage the condition better.
7. **Infection Prevention**: Practice good hygiene and stay up-to-date with vaccinations, as infections can sometimes trigger or worsen ITP.
Consulting with healthcare professionals for personalized advice is crucial. - Medication
- Autoimmune thrombocytopenic purpura (ITP) is typically treated with medications such as corticosteroids (e.g., prednisone) to reduce the autoimmune response. Other treatments may include intravenous immunoglobulin (IVIG), rituximab, and thrombopoietin receptor agonists (e.g., romiplostim, eltrombopag) to increase platelet production. In some cases, immunosuppressive drugs (e.g., azathioprine, mycophenolate mofetil) may also be used.
- Repurposable Drugs
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Autoimmune thrombocytopenic purpura (ITP), also known as immune thrombocytopenia, involves the body's immune system attacking and destroying platelets. Some drugs that are used for other conditions but may be repurposable for ITP include:
1. **Rituximab** - Originally for non-Hodgkin's lymphoma and rheumatoid arthritis.
2. **Eltrombopag and Romiplostim** - Thrombopoietin receptor agonists developed for chronic ITP that stimulate platelet production.
3. **IVIG (Intravenous Immunoglobulin)** - Used for a variety of immune deficiencies and autoimmune disorders.
4. **Dapsone** - Commonly used for dermatitis herpetiformis and leprosy.
5. **Azathioprine** - An immunosuppressant used in organ transplantation and autoimmune diseases.
These medications work through various mechanisms to modulate the immune response or increase platelet production. - Metabolites
- Autoimmune thrombocytopenic purpura (ITP) is characterized primarily by a low platelet count, but specific metabolites directly associated with it are not well-defined. It's more of an immunological disorder impacting platelet survival than a metabolic one. The focus is on antibodies attacking platelets. Indirectly, there can be elevated levels of metabolites related to altered bone marrow activity or as a response to increased cell turnover. However, no specific metabolites are unique identifiers of ITP.
- Nutraceuticals
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For autoimmune thrombocytopenic purpura (ITP), there is no established evidence that nutraceuticals directly treat the condition. However, some nutraceuticals and dietary supplements may support general immune function and overall health:
1. **Vitamin D:** May help regulate the immune system, potentially beneficial for autoimmune disorders.
2. **Omega-3 Fatty Acids:** Known for their anti-inflammatory properties.
3. **Probiotics:** Can support gut health, which is linked to immune function.
4. **Curcumin (Turmeric):** Has anti-inflammatory and immune-modulating properties.
5. **Vitamin B12 and Folate:** Essential for overall blood health.
Always consult with a healthcare professional before starting any new supplement regimen, particularly with a condition like ITP. - Peptides
- Autoimmune thrombocytopenic purpura (ITP) involves the immune system attacking and destroying platelets, leading to bleeding and easy bruising. Peptides, short chains of amino acids, can sometimes be involved in therapeutic strategies for modulating the immune response. Researchers are exploring nanoparticle (nan) technologies to deliver drugs or therapeutic agents more precisely to improve outcomes in ITP. These innovations aim to enhance the efficacy and safety of treatments by targeting specific cells or tissues involved in the disease process.