What is Immune Thrombocytopenic Purpura (ITP)?

From the Guidelines

Immune Thrombocytopenic Purpura (ITP) is an acquired autoimmune disorder characterized by a low platelet count resulting from platelet destruction and impaired platelet production, as defined by the American Society of Hematology 2019 guidelines for immune thrombocytopenia 1.

Description of ITP

ITP has an incidence of 2 to 5 per 100 000 and can be an isolated primary condition or it may be secondary to other conditions, with a heterogeneous disorder having variable clinical symptoms and remains a diagnosis of exclusion of other causes of thrombocytopenia 1.

Clinical Presentation

The primary symptoms of ITP include easy bruising, petechiae, purpura, nosebleeds, bleeding gums, and in severe cases, internal bleeding, with bleeding events often being unpredictable, and patients with ITP, even in the setting of severe thrombocytopenia, may not exhibit bleeding beyond bruising and petechiae 1.

Treatment

Treatment depends on platelet count, bleeding severity, and patient age, with first-line treatments typically including corticosteroids like prednisone, intravenous immunoglobulin (IVIG), or anti-D immunoglobulin for Rh-positive patients, and second-line options including thrombopoietin receptor agonists, rituximab, or splenectomy for persistent cases 1.

Quality of Life

ITP has a significant impact on health-related quality of life (HRQoL), particularly in the first year after diagnosis, related to restrictions on activities, anxiety due to the risk of bleeding, and the burden of treatment and monitoring, with fatigue being common and reported in 22% to 45% of patients with ITP 1.

Management

The decision as to whether a patient can be observed or requires further intervention is highly complex and varies based on comorbidities, medications, and age, which all impact the risk of bleeding, with management approaches varying based on disease duration, access to care, quality-of-life implications, and patient and provider preferences, among other factors 1.

From the FDA Drug Label

Immune Thrombocytopenia (ITP) Nplate is indicated for the treatment of thrombocytopenia in: Adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy Pediatric patients 1 year of age and older with ITP for at least 6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Immune Thrombocytopenic Purpura (ITP) is a condition characterized by a low platelet count, where the immune system destroys platelets and/or impairs the production of new platelets.

• Key points:
  • ITP is also known as immune thrombocytopenia
  • It is a condition where the immune system attacks and destroys platelets
  • This can lead to a low platelet count, increasing the risk of bleeding
  • Treatment options may include medications such as romiplostim (Nplate) to increase platelet production 2 2

From the Research

Definition of Immune Thrombocytopenic Purpura (ITP)

• Immune Thrombocytopenic Purpura (ITP) is an autoimmune disease characterized by low platelet count and bleeding 3, 4, 5, 6, 7
• It is also known as idiopathic thrombocytopenic purpura, and is a blood disorder in which the body's immune system destroys platelets, leading to low platelet counts in the blood (peripheral blood platelet count < 150 × 10^9/L) 6

Pathophysiology of ITP

• The pathophysiology of ITP is not fully understood, but it is believed to result from a complex interplay between hereditary and environmental variables 6
• Platelet autoantibodies result in accelerated platelet destruction by the reticuloendothelial cells in the spleen and liver, overwhelming the compensatory capability of the bone marrow to increase platelet production 5

Treatment Options for ITP

• Corticosteroids, intravenous immunoglobulin (IVIG), immunosuppressants, rituximab, and thrombopoietin receptor agonists (TPO-RAs) are some of the advanced treatments for ITP 3, 4, 5, 6, 7
• Splenectomy is also a treatment option, although it is usually considered later in the treatment process 4, 7
• New drugs for ITP treatment are now available after steroid failure, and individualized treatment plans should be developed based on the patient's age, platelet count, risk of bleeding, and comorbidities 6, 7

Goals of Therapy

• The goal of treatment for patients with ITP is to raise the platelet count to high enough levels to prevent bleeding using the least toxic therapy, recognizing the generally benign nature of the illness 5
• The goals of therapy involve maintaining a safe platelet count while minimizing toxicities from the treatment 4