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Learn about certain blood disorders: Immune Thrombocytopenia (ITP) and Severe Aplastic Anemia (SAA)

What is Immune Thrombocytopenia (ITP)?

ITP is a blood disorder characterized by low blood platelet counts, purpura, and bleeding episodes caused by antiplatelet autoantibodies. ITP may result in severe and unpredictable bleeding.01 ITP may be caused by autoimmune disorders, infections, pregnancy, certain medication use, or an unknown cause.02  

ITP can be acute, persistent or chronic.01

  • Acute – Most common in young children, often following a viral infection, and resolves within a few months. In most cases, treatment is not needed. 
  • Persistent – Lasting 3-12 months after diagnosis. 
  • Chronic – Lasting more than 12 months after diagnosis. 

Persistent or chronic ITP typically affects adults (fewer than 10% of children develop chronic ITP). Treatment is generally recommended to bring platelet counts to normal and stop active bleeding.01 Estimated prevalence of ITP is 17 per 100,000 people, with higher prevalence among adults over 65.03

What are Symptoms of ITP?

The symptoms of ITP can vary, and some people with ITP may experience no symptoms at all. Typically, symptoms increase as platelet count gets lower. Common symptoms are:04 

  • Easy or excessive bruising 
  • Tiny red/purple spots on the skin (also known as petechiae) 
  • Easy or excessive bleeding 
    • Bleeding from the gums or nose 
    • Blood in urine or stools 
    • Extremely heavy menstrual flow 
    • Internal bleeding 
  • Feeling tired or fatigued 

How is ITP Treated?

Treatment depends on severity of symptoms, platelet count, age, response to therapy and its side effects, and many other factors. While there is no well-established treatment that cures patients with ITP, most patients find that their platelet count will improve following treatment.04

Treatment Overview04

First Line Second Line
(corticosteroid-dependent or have no response to corticosteroids)
Corticosteroids such as:
• Prednisone
• Dexamethasone
• Methylprednisolone		 Thrombopoietin Receptor Agonists (TPO-RAs) such as:
• Eltrombopag
• Romiplostim
• Avatrombopag
Intravenous immunoglobulin (IVIG) Rituximab
Platelet transfusions Fostamatinib disodium hexahydrate
Anti-D immunoglobulin Splenectomy

What is Severe Aplastic Anemia?

Aplastic Anemia (AA) is a rare form of bone marrow failure, whereby the bone marrow does not produce new cells, leaving the body susceptible to bleeding and infection.05 About 2 out of every 1 million people in the United States are diagnosed with aplastic anemia each year.05

Aplastic anemia is classified as severe (SAA) if at least two of the following are present:06

  • Absolute reticulocyte count (ARC) <60,000/mcL; some centers use a threshold of <50,000/mcL
  • Platelet count <20,000/mcL
  • Absolute neutrophil count (ANC) <500/mcL

Treatment Overview 0607

Treatment Algorithm, Severe or very severe AA, Age less than 40 years with a rapidly-available donor, Age more or equal 40 or without rapidly available donor, Hematopoietic Cell Transplant, Immunosuppressive therapy (IST), hATG (horse antithymocyte globulin) + cyclosporine A, Triple Therapy: hATG + Cyclosporine A + Eltrombopag

Learn about a new treatment option that may be right for your patients with these conditions.

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IMPORTANT SAFETY INFORMATION

WARNINGS and PRECAUTIONS

Hepatic Decompensation in Patients with Chronic Hepatitis C. In patients with chronic hepatitis C, ALVAIZ in combination with interferon and ribavirin may increase the risk of hepatic decompensation. 

Hepatotoxicity. ALVAIZ may increase the risk of severe and potentially life-threatening hepatotoxicity. 

Increased Risk of Death and Progression of Myelodysplastic Syndromes to Acute Myeloid Leukemia. In a clinical trial of patients with intermediate to high risk MDS with thrombocytopenia, an increased number of progressions from MDS to AML was observed compared to placebo. 

Thrombotic/Thromboembolic Complications may result from increases in platelet counts with ALVAIZ. Reported thrombotic/thromboembolic complications included both venous and arterial events and were observed at low and at normal platelet counts.  

Cataracts. In clinical trials, development or worsening of cataracts was reported in patients treated with eltrombopag. 

ADVERSE REACTIONS  

The following clinically significant adverse reactions associated with ALVAIZ are described above.  

  • Hepatic Decompensation in Patients with Chronic Hepatitis C 
  • Hepatotoxicity 
  • Increased Risk of Death and Progression of Myelodysplastic Syndromes to Acute Myeloid Leukemia 
  • Thrombotic/Thromboembolic Complications 
  • Cataracts 

Common adverse reactions associated with the use of ALVAIZ observed in placebo-controlled clinical trials in adults and pediatric patients include nausea, diarrhea, upper respiratory tract infection, nasopharyngitis, cough, anemia, pyrexia, and fatigue. 

USE IN SPECIFIC POPULATIONS  

Pregnancy and Lactation 

There is insufficient data in pregnant women to assess any drug-associated risks for major birth defects, miscarriage, or adverse maternal or fetal outcomes. Due to the potential for serious adverse reactions in a breastfed child from eltrombopag, breastfeeding is not recommended during treatment. 

Pediatric Use 

The safety and effectiveness of ALVAIZ have been established in pediatric patients 6 years and older with persistent or chronic ITP. The safety and effectiveness of ALVAIZ have not been established in pediatric patients less than 6 years of age with persistent or chronic ITP. The safety and effectiveness in pediatric patients with thrombocytopenia associated with chronic hepatitis C and refractory severe aplastic anemia have not been established. 

INDICATIONS AND USAGE 

Treatment of Thrombocytopenia in Patients with Persistent or Chronic Immune Thrombocytopenia 

ALVAIZ™ (eltrombopag tablets) are indicated for the treatment of thrombocytopenia in adult and pediatric patients 6 years and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. ALVAIZ should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding. 

Treatment of Thrombocytopenia in Patients with Hepatitis C Infection 

ALVAIZ is indicated for the treatment of thrombocytopenia in adult patients with chronic hepatitis C to allow the initiation and maintenance of interferon-based therapy. ALVAIZ should be used only in patients with chronic hepatitis C whose degree of thrombocytopenia prevents the initiation of interferon-based therapy or limits the ability to maintain interferon-based therapy. 

Treatment of Severe Aplastic Anemia 

ALVAIZ is indicated for the treatment of adult patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy. 

Limitations of Use 
  • ALVAIZ is not indicated for the treatment of patients with myelodysplastic syndromes (MDS). 
  • Safety and efficacy have not been established in combination with direct-acting antiviral agents used without interferon for treatment of chronic hepatitis C infection. 

Please see the full Prescribing Information, including Medication Guide and Boxed Warning. 

References

  1. Back to contents.

    Justiz Vaillant AA, Gupta N. ITP – Immune Thrombocytopenic Purpura. StatPearls. 2023 Jan. Updated May 6, 2023. Available at: https://www.ncbi.nlm.nih.gov/books/NBK537240/ 

  2. Back to contents.

    Idiopathic Thrombocytopenic Purpura. Johns Hopkins Medicine. Accessed July 5, 2023. Available at: https://www.hopkinsmedicine.org/health/conditions-and-diseases/idiopathic-thrombocytopenic-purpura.

  3. Back to contents.

    Liang Y, et al. Prevalence of primary immune thrombocytopenia and related healthcare resource utilization among Texas Medicaid beneficiaries. Curr Med Res Opin. 2021 Jun;37(8):1315-1322. 

  4. Back to contents.

    https://rarediseases.org/rare-diseases/immune-thrombocytopenia/. Accessed June 16, 2023. 

  5. Back to contents.

    Definition & Facts for Aplastic Anemia & Myelodysplastic Syndromes. National Institute of Diabetes and Digestive and Kidney Diseases. Available at: https://www.niddk.nih.gov/health-information/blood-diseases/aplastic-anemia-myelodysplastic-syndromes/definition-facts. Accessed July 4, 2023.

  6. Back to contents.

    Olson TS, Dunbar CE. Treatment of aplastic anemia in adults. Wolters Kluwer UpToDate. Accessed July 4, 2023.

  7. Back to contents.

    Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017; 129(11):1428-1436. https://doi.org/10.1182/blood-2016-08-693481.