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Treatment Name: Antithymocyte Globulin (ATG, ATGAM®) + Cyclosporine (Gengraf®, Neoral®) + Eltrombopag (Promacta®)

Antithymocyte Globulin (ATG, ATGAM®) + Cyclosporine (Gengraf®, Neoral®) + Eltrombopag (Promacta®) is a Treatment Regimen for Severe Aplastic Anemia (SAA)

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References

1) Townsley DM, Scheinberg P, Winkler T, et al. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med. 2017;376:1540-1550.

2) Supplement to: Townsley DM, Scheinberg P, Winkler T, et al. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med. 2017;376:1540-1550.

Created: February 14, 2019 Updated: February 14, 2019

What is Severe Aplastic Anemia (SAA)?

Severe aplastic anemia (SAA) is a rare blood disorder that can be life-threatening if left untreated. SAA is thought to result from certain cells of the immune system inappropriately attacking healthy cells within the bone marrow. This leads to:

  • A low white blood cell count, which increases the risk of infection
  • A low red blood cell count, which decreases the ability to deliver oxygen and may cause fatigue, weakness, and shortness of breath
  • A low platelet count, which increases the risk of bruising and bleeding

The treatment of aplastic anemia may include immunosuppressant agents (shown below) or a stem cell transplant if a stem cell donor can be identified. A matched sibling donor is often thought to be be the best option, if one is available. The goals of these treatments are to restore the healthy bone marrow cells, prevent infection and bleeding, and to stop the need for blood or platelet transfusions.

Common cyclosporine + ATG + eltrombopag starting doses

  • ATG 40 mg/kg intravenous (I.V.) infusion over 4 to 24 hours once daily on Days 1, 2, 3, and 4
  • Usual cyclosporine starting dose: 5 mg/kg by mouth twice daily (Important note: Ideal Body Weight is recommended for dosing cyclosporine)
  • Usual Eltrombopag starting dose: 150 mg (three 50 mg tablets) by mouth once daily starting on Day 1