From the FDA Drug Label
Severe Aplastic Anemia: Refractory Severe Aplastic Anemia In the single-arm, open-label trial, 43 patients with refractory severe aplastic anemia received eltrombopag. The most common adverse reactions (greater than or equal to 20%) were nausea, fatigue, cough, diarrhea, and headache.
The diagnosis of Aplastic Anemia is not directly stated in the label, but it is mentioned that 43 patients with refractory severe aplastic anemia received eltrombopag in a single-arm, open-label trial. The treatment for Aplastic Anemia with eltrombopag is mentioned, but the label does not provide a comprehensive treatment plan for aplastic anemia. Eltrombopag may be used in the treatment of refractory severe aplastic anemia, but the label does not provide enough information to determine the diagnosis or a complete treatment plan for aplastic anemia 1.
From the Research
Aplastic anemia should be treated with immunosuppressive therapy, including anti-thymocyte globulin (ATG) and cyclosporine, or hematopoietic stem cell transplantation (HSCT) for younger patients with matched donors, as these approaches have been shown to improve morbidity, mortality, and quality of life outcomes. The diagnosis of aplastic anemia is often difficult due to the required exclusion of numerous inherited or acquired diseases with similar phenotypes 2. Treatment depends on severity, patient age, and availability of a stem cell donor. For severe aplastic anemia, the first-line treatment is typically immunosuppressive therapy with ATG and cyclosporine, or HSCT for younger patients with matched donors. ATG is usually administered at 40 mg/kg/day for 4 days, followed by cyclosporine at 5-10 mg/kg/day in divided doses to maintain blood levels of 200-400 ng/mL for at least 6 months 3.
Some key points to consider in the treatment of aplastic anemia include:
- The use of eltrombopag, a thrombopoietin receptor agonist, may be added at 150 mg daily if response to immunosuppression is inadequate 4.
- Supportive care includes transfusions of red blood cells and platelets as needed, along with prophylactic antibiotics for neutropenic patients.
- The condition is believed to be primarily autoimmune in nature, with T-cell mediated destruction of hematopoietic stem cells.
- Prompt diagnosis and treatment are essential as severe aplastic anemia has high mortality if left untreated, but with appropriate therapy, 70-80% of patients can achieve long-term survival 5.
- For older patients (>60), dose-attenuated immunosuppressive therapy may be considered due to concerns about tolerance of full-dose therapy 4.
Overall, the treatment of aplastic anemia requires a comprehensive approach that takes into account the severity of the disease, patient age, and availability of a stem cell donor, with the goal of improving morbidity, mortality, and quality of life outcomes.