Disease Overview

Immune thrombocytopenia (ITP) is a disorder where the body’s immune system mistakenly attacks and destroys its own platelets, which are crucial for blood clotting. This immune reaction leads to a decrease in platelet count (thrombocytopenia), increasing the risk of bleeding.

Epidemiology

ITP can affect individuals of all ages, but it is most commonly seen in two forms:

Acute ITP: Typically affects children between 2 to 6 years old and often follows a viral infection. Symptoms usually resolve within weeks to months without treatment.

Chronic ITP: Occurs in adults and can also affect children. It persists for six months or longer and may require ongoing medical management.

ITP is considered rare, with an estimated annual incidence of 1 to 6 cases per 100,000 children and adults. It affects both genders equally and can occur at any age.

Diagnostic Criteria

Clinical history and physical examination to assess for signs of bleeding such as petechiae, ecchymoses (bruises), and mucosal bleeding. Blood tests to measure platelet count, assess platelet size and morphology, and rule out other causes of thrombocytopenia. Guidelines from organizations such as the American Society of Hematology provide criteria for diagnosing ITP based on clinical findings and laboratory results.

Clinical Presentation

Patients with immune thrombocytopenia (ITP) commonly exhibit symptoms related to low platelet counts, which impair normal blood clotting functions. These manifestations often include small red or purple spots on the skin known as petechiae, as well as larger bruises called ecchymoses. Additionally, patients may experience frequent nosebleeds, bleeding gums, or unusually heavy menstrual bleeding in women. In severe cases of ITP, there is a heightened risk of bleeding into critical internal organs such as the brain, leading to intracranial hemorrhage, or into the gastrointestinal tract. These complications can pose significant health risks and require prompt medical attention to prevent potentially life-threatening outcomes.

Evaluation / Diagnosis  

Healthcare providers rely on a comprehensive approach that involves gathering a detailed medical history to identify symptoms and potential triggers that may contribute to the disease. A thorough physical examination is conducted to assess for signs of bleeding, such as petechiae and ecchymoses, and to evaluate for the enlargement of the spleen, which can occur in some cases of ITP.

Laboratory tests play a crucial role in confirming the diagnosis, including a complete blood count (CBC) to measure the platelet count, which is typically low in patients with ITP. A peripheral blood smear is also performed to examine the morphology of blood cells and identify any abnormalities. In some instances where there is diagnostic uncertainty or suspicion of other underlying conditions causing thrombocytopenia, a bone marrow aspiration may be recommended to further investigate and exclude alternative causes.

Management

The management of immune thrombocytopenia (ITP) focuses on several key strategies to effectively control symptoms and prevent complications. Pharmacological treatment options are central to therapy. The initial treatment approach focuses on reducing bleeding tendencies and boosting platelet counts. Corticosteroids are commonly used to suppress immune activity, while thrombopoietin receptor agonists (TPO-RAs) like Promacta (eltrombopag) and Nplate (romiplostim) stimulate platelet production.

For patients who do not respond adequately to first-line therapies, newer options are being explored. These include inhibitors targeting specific pathways involved in platelet destruction, such as Syk inhibitors and Bruton tyrosine kinase (BTK) inhibitors. These therapies aim to address the underlying immune dysfunction more effectively, potentially offering sustained platelet response and reducing the need for long-term treatment strategies like splenectomy.

Ongoing clinical trials are assessing the safety and efficacy of these experimental treatments, seeking to broaden the therapeutic options available for managing ITP. By advancing our understanding and treatment approaches, researchers aim to improve outcomes and quality of life for individuals living with this challenging autoimmune disorder.

Non-pharmacological approaches include splenectomy, the surgical removal of the spleen, which is considered in cases of severe or refractory ITP where pharmacological treatments have proven ineffective. Regular monitoring of platelet counts and symptoms is crucial to assess treatment effectiveness and adjust therapies as needed. Patients are also advised to avoid activities that increase the risk of bleeding, such as certain medications and contact sports, to minimize potential complications.