From the Guidelines
The standard treatment for severe aplastic anemia typically involves immunosuppressive therapy or hematopoietic stem cell transplantation (HSCT), with HSCT being the first-line treatment for patients under 50 years old with a matched sibling donor. For these patients, HSCT is generally recommended as the initial treatment, as it offers a potential cure for the disease 1. The conditioning regimen usually includes cyclophosphamide and anti-thymocyte globulin (ATG), followed by the transplantation.
For patients without a matched donor or those over 50, immunosuppressive therapy is recommended, consisting of horse ATG combined with cyclosporine 1. Cyclosporine is typically continued for at least 6-12 months, with slow tapering if blood counts improve. Supportive care includes transfusions of red blood cells and platelets as needed, and prophylactic antibiotics for neutropenic patients. Eltrombopag, a thrombopoietin receptor agonist, may be added if response to initial therapy is inadequate.
Some key points to consider in the treatment of severe aplastic anemia include:
- The use of haploidentical HSCT (HID-HSCT) as a viable option for patients without a matched sibling donor, with similar outcomes to matched sibling donor HSCT (MSD-HSCT) 1
- The importance of systematic, standardized pre-transplant risk stratification, using tools such as the European Group for Blood and Marrow Transplantation (EBMT) risk score and hematopoietic cell transplantation-specific comorbidity index (HCT-CI) 1
- The potential for reduced-intensity conditioning regimens, such as fludarabine and cyclophosphamide, for older patients or those with comorbidities 1
Overall, the goal of treatment for severe aplastic anemia is to suppress the autoimmune destruction of bone marrow stem cells, allowing recovery of normal blood cell production and improving quality of life. HSCT is generally the preferred treatment for patients under 50 with a matched sibling donor, while immunosuppressive therapy is recommended for those without a matched donor or over 50.
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 normal treatment for severe aplastic anemia is immunosuppressive therapy. If the patient has had an insufficient response to immunosuppressive therapy, eltrombopag (ALVAIZ) may be used 2.
- Key points:
- Eltrombopag is used for adult patients with severe aplastic anemia.
- It is used when the patient has not responded sufficiently to immunosuppressive therapy.
- The treatment should be used under the guidance of a healthcare professional.
From the Research
Normal Treatment for Severe Aplastic Anemia
The normal treatment for severe aplastic anemia includes:
- Immunosuppressive therapy with antithymocyte globulin (ATG) and cyclosporine (CsA) 3, 4, 5
- Allogeneic hematopoietic stem cell transplantation 6
- Supportive therapy, including transfusion, granulocyte colony-stimulating factor, and iron chelation therapy 6
- Thrombopoietin receptor agonist (TPO-RA) treatment 6
- Anabolic hormone therapy 6
Immunosuppressive Therapy
Immunosuppressive therapy is the treatment modality for the majority of patients with aplastic anemia, most of whom are not candidates for allogeneic stem-cell transplantation 5. The combination of ATG and CsA has become the standard of care for appropriate patients 3, 4, 5.
Alternative Immunosuppressive Regimens
Alternative immunosuppressive regimens, such as tacrolimus plus rabbit ATG-based immunosuppressive therapy, have also been studied 7.
Treatment Choice
The treatment choice is made based on the disease severity and patient age 6.