From the Guidelines
The FDA-approved medications for aplastic anemia include immunosuppressive therapy, such as anti-thymocyte globulin (ATG) and cyclosporine, as well as hematopoietic growth factors like eltrombopag (Promacta). The primary treatment approach for severe aplastic anemia (SAA) involves immunosuppressive therapy, which is typically the first-line treatment for patients who are not candidates for immediate bone marrow transplantation.
- The standard immunosuppressive regimen consists of ATG combined with cyclosporine 1.
- This regimen is usually considered for patients who are < 50 years of age with SAA or very severe aplastic anemia (vSAA) without HLA-matched sibling donors.
- For hematopoietic growth factors, eltrombopag (Promacta) is FDA-approved for severe aplastic anemia, typically given at a starting dose of 50 mg daily, which can be adjusted based on platelet response.
- Eltrombopag can be used alone in refractory cases or in combination with immunosuppressive therapy. The choice of treatment depends on the severity of the disease, the patient's age, and their eligibility for bone marrow transplantation, as outlined in guidelines for SAA treatment 1.
- Key considerations include the patient's response to initial therapy and their overall health status, with options for transplantation, including HLA-matched sibling donors, matched unrelated donors, or cord blood transplantation, considered based on age and disease severity.
- The treatment approach for aplastic anemia is multifaceted, requiring careful consideration of the patient's individual circumstances to optimize outcomes in terms of morbidity, mortality, and quality of life.
From the FDA Drug Label
1.3 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.
The FDA approved medication for the treatment of severe aplastic anemia is eltrombopag (ALVAIZ), specifically for adult patients who have had an insufficient response to immunosuppressive therapy 2.
From the Research
FDA Approved Medications for Aplastic Anemia
The following medications are used in the treatment of aplastic anemia:
- Antithymocyte globulin (ATG) 3, 4, 5, 6
- Cyclosporine A (CsA) 3, 4, 5, 6
- Thrombopoietin receptor agonist (TPO-RA), such as eltrombopag 3, 7 Other treatments may include:
- Allogeneic hematopoietic stem cell transplantation 7
- Anabolic hormone therapy 7
- Granulocyte colony-stimulating factor 7
- Iron chelation therapy 7
Combination Therapies
Combination therapies, such as the use of ATG and CsA, have shown satisfactory results in patients with aplastic anemia 3, 4, 5, 6. The addition of other immunosuppressive agents or growth factors to the standard IST regimen may also be effective 3.
Dosage and Efficacy
The dosage of ATG may vary, with some studies using a lower dose of 25 mg/kg/day × 4 days 6, while others use a standard dose of 40 mg/kg/day × 4 days 4, 6. The efficacy of these treatments may depend on various factors, including the interval between the onset of symptoms and the initiation of therapy 6.