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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
Age of Onset
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
Autosomal recessive
Mitochondrial/Multigenic
X-linked dominant
X-linked recessive
5 Facts you should know
FACT
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
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
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
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
Fostamatinib, a spleen tyrosine kinase (Syk) inhibitor is an option for patients whose disease is refractory to second-line treatments
Interest over time
Google searches
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)
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)
FDA-approved indication: Treatment of adults and children with chronic and acute immune thrombocytopenic purpura.
Top Clinical Trials
| Title | Description | Phases | Status | Interventions | More Information |
|---|---|---|---|---|---|
| Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) | This is a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Patients will receive rilzabrutinib or placebo 400mg twice daily. | Phase 3 | Recruiting | Drug: Rilzabrutinib|Drug: Placebo | More Info |
| Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children | This is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old. | Phase 3 | Recruiting | Drug: Eltrombopag|Drug: Steroids|Drug: IVIG|Drug: Rho(D) Immune Globulin | More Info |
| Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months | A Phase 3b Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months | Phase 3 | Recruiting | Drug: Avatrombopag|Drug: Placebo | More Info |
| A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). | This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP. | Phase 3 | Active, not recruiting | Biological: efgartigimod | More Info |
| A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia | This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP. | Phase 3 | Recruiting | Biological: Efgartigimod PH20 SC|Other: Placebo PH20 SC | More Info |
| A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia | A Phase 3 study to evaluate the safety and efficacy of efgartigimod PH20 subcutaneous in adult patients with primary immune thrombocytopenia | Phase 3 | Recruiting | Biological: efgartigimod PH20 SC | More Info |
| A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP) | The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab. | Phase 3 | Active, not recruiting | Drug: Rozanolixizumab | More Info |
Top Treatments in Research
| Agent | Class/Mechanism of Action | Development Status | Company | Clinical Studies | More Information |
|---|---|---|---|---|---|
| Rilzabrutinib | An oral Bruton’s tyrosine kinase inhibitor incorporating Sanofi’s TAILORED COVALENCY® technology being investigated for the treatment of immune-mediated diseases, including ITP. BTK is an intracellular signaling molecule involved in innate and adaptive immune responses related to certain immune-mediated diseases. By inhibiting BTK, rilzabrutinib has the potential to target the underlying disease pathogenesis. | Phase 3 | Principia Biopharma, a Sanofi Company|Sanofi | https://ClinicalTrials.gov/show/NCT04562766 | More Info |
| efgartigimod PH20 SC | a human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating immunoglobulin G (IgG) autoantibodies. It is the first and only approved FcRn blocker. | Phase 3 | Argenx | https://ClinicalTrials.gov/show/NCT04812925 | More Info |
| Rozanolixizumab | a subcutaneously administered, humanized monoclonal antibody that specifically binds, with high affinity, to human FcRn. It has been designed to block the interaction of FcRn and IgG, inhibiting IgG recycling and inducing the removal of pathogenic IgG autoantibodies.1,11 | Phase 3 | UCB Biopharma SRL|UCB Pharma | https://ClinicalTrials.gov/show/NCT04596995 | More Info |