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Immune thrombocytopenic purpura (ITP)

Immune thrombocytopenic purpura (ITP) is an autoimmune pathology characterized by a low platelet count, purpura, and hemorrhagic episodes caused by antiplatelet autoantibodies

Prevalence

15-18 / 10 000

49,650-59,580

US Estimated

77,025-92,430

Europe Estimated

Age of Onset

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ICD-10

D69.3

Inheritance

Immune thrombocytopenic purpura and other autoimmune disorders can run in families, but the inheritance pattern is usually unknown. People with a first-degree relative (such as a parent or sibling) with immune thrombocytopenia likely have an increased risk of developing the disorder themselves.

Autosomal dominant

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Autosomal recessive

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Mitochondrial/Multigenic

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Not applicable

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X-linked dominant

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X-linked recessive

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Rare View

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5 Facts you should know

FACT

1

ITP is defined as a low platelet count - <100,000 - with the presence of a generalized petechial rash, bruising, or bleeding in an otherwise healthy patient

FACT

2

The chronic form of ITP affects individuals between ages 20-50 years; there is a female/male ratio of 3 to 1, and It is usually not preceded by a viral infection

FACT

3

First line therapy: ITP patients with active bleeding require corticosteroids to stop further destruction of platelets - about 60% of patients respond well within two weeks

FACT

4

For adults with ITP who are dependent or unresponsive to corticosteroids for 3+ months are considered chronic ITP, guidelines recommend 2nd-line therapies including thrombopoietin receptor agonists (TPO-RA), rituximab, or splenectomy

FACT

5

Fostamatinib, a spleen tyrosine kinase (Syk) inhibitor is an option for patients whose disease is refractory to second-line treatments

Immune thrombocytopenic purpura is also known as...

Immune thrombocytopenic purpura

Immune thrombocytopenia

ITP

Autoimmune thrombocytopenic purpura

Idiopathic thrombocytopenic purpura

Werlhof disease

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Common Signs & Symptoms

Thrombocytopenia

(low platelet count)

Thromboembolism

(obstructive blood clot)

Epistaxis

(nose bleeding)

Gastrointestinal hemorrhage

(gastrointestinal bleeding)

Arterial thrombosis

(blood clot in artery)

Bruising susceptibility

(easy bruisability)

Gingival bleeding

(bleeding gums)

Cerebral hemorrhage

(bleeding in brain)

Current treatments

The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.

Fostamatinib disodium hexahydrate (Brand name: Tavalisse)

Manufactured by Rigel Pharmaceuticals, Inc.
FDA-approved indication: April 2018 approved for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

MOA from Rigel Pharmaceuticals, Inc. on Vimeo.

Human immunoglobulin (human) (Brand name: Gammaplex)

Manufactured by Bio Products Laboratory
FDA-approved indication: In adults for treatment of chronic immune thrombocytopenic purpura (ITP)

Immune globulin injection [human], 10% caprylate/chromatography purified (Brand name: Gamunex-C)

Manufactured by Grifols
FDA-approved indication: Treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability and impairment and for maintenance therapy to prevent relapse. Also for use for the treatment of primary immunodeficiency disease (PIDD) in patients 2 years of age and older and idiopathic thrombocytopenic purpura (ITP).

Romiplostim (Brand name: Nplate)

Manufactured by Amgen, Inc
FDA-approved indication: December 2018, romiplostim (Nplate) received expanded approval for the treatment of thrombocytopenia in 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. Previously in August 2008, it was approved for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Immune globulin intravenous (human), 10% liquid (Brand name: Privigen)

Manufactured by CSL Behring
FDA-approved indication: September 2017, immune globulin intravenous (human), 10% liquid (Privigen) was approved for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability and impairment, however it was not studied for use longer than 6 months. It was also approved for the treatment of patients age 15 years and older with chronic immune thrombocytopenic purpura (ITP) to raise platelet counts, and as replacement therapy for primary humoral immunodeficiency (PI).

Eltrombopag (Brand name: Promacta)

Manufactured by GlaxoSmithKline
FDA-approved indication: Treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Immune Globulin Injection (Human) 10% Caprylate/Chromatography Purified (Brand name: Gammaked)

Manufactured by Kedrion Biopharma
FDA-approved indication: The treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability and impairment and for maintenance therapy to prevent relapse, primary humoral immunodeficiency (PI) in patients 2 years of age and older, and idiopathic thrombocytopenic purpura (ITP) to raise platelet counts to prevent bleeding or to allow a patient with ITP to undergo surgery.

Rho (D) immunoglobulin intravenous (human) (Brand name: WinRho SD)

Manufactured by Baxter
FDA-approved indication: Treatment of adults and children with chronic and acute immune thrombocytopenic purpura.

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