Immune Thrombocytopenic Purpura (ITP)

Also known as: Idiopathic Thrombocytopenic Purpura, Autoimmune Thrombocytopenic Purpura

Immune Thrombocytopenic Purpura (ITP) is an autoimmune disorder characterized by a low platelet count (thrombocytopenia) due to the immune system attacking and destroying platelets. This can lead to increased risk of bleeding and bruising. ITP can be acute (short-term) or chronic (long-term).

Sometimes, your body's defense system gets confused and starts attacking your platelets, which are like tiny bandages in your blood. This makes you bruise and bleed more easily. Doctors can give you medicine to help your body stop attacking the platelets.

Signs & Symptoms

Easy or excessive bruising (purpura)
Superficial bleeding into the skin that appears as pinpoint-sized reddish-purple spots (petechiae), usually on the lower legs
Prolonged bleeding from cuts
Spontaneous bleeding from gums or nose
Blood in urine or stool
Unusually heavy menstrual flow
Fatigue
Enlarged spleen (splenomegaly) - rare

Treatment Options

MEDICATION FDA Approved

Corticosteroids (e.g., Prednisone)

HIGHLY EFFECTIVE

MEDICATION FDA Approved

Intravenous Immunoglobulin (IVIG)

HIGHLY EFFECTIVE

MEDICATION FDA Approved

Anti-RhD Immunoglobulin (WinRho)

MODERATELY EFFECTIVE

MEDICATION FDA Approved

Thrombopoietin Receptor Agonists (TPO-RAs) (e.g., Romiplostim, Eltrombopag, Avatrombopag)

HIGHLY EFFECTIVE

SURGERY

Splenectomy (surgical removal of the spleen)

HIGHLY EFFECTIVE

MEDICATION FDA Approved

Rituximab (anti-CD20 monoclonal antibody)

MODERATELY EFFECTIVE

MEDICATION FDA Approved

Fostamatinib (Syk inhibitor)

MODERATELY EFFECTIVE Approved 2018

SUPPORTIVE

Supportive Care (e.g., avoiding NSAIDs, managing bleeding risks)

SUPPORTIVE

Diagnosis

Complete Blood Count (CBC) to assess platelet count
Peripheral blood smear to examine platelet morphology
Bone marrow aspiration and biopsy (to rule out other causes of thrombocytopenia)
Testing for anti-platelet antibodies (though not always reliable)
Ruling out other conditions (e.g., infections, drug-induced thrombocytopenia)

History

ITP was first described in the 18th century, but its autoimmune nature was not fully understood until the mid-20th century. Early treatments focused on splenectomy, while more recent advances have led to the development of immunomodulatory therapies and TPO-RAs.

Recent Breakthroughs

2018

FDA Approves Fostamatinib for Chronic ITP

Fostamatinib, a spleen tyrosine kinase (Syk) inhibitor, was approved by the FDA for the treatment of adults with chronic ITP who have had an insufficient response to previous treatment. This provides a new oral treatment option for patients with refractory ITP.

2023

Avatrombopag Demonstrates Sustained Platelet Response in ITP

Clinical trials have shown that avatrombopag, another TPO-RA, can achieve sustained platelet response and reduce the need for rescue therapies in patients with chronic ITP.