Role of Eltrombopag and Cyclosporine in Aplastic Anemia

First-Line Treatment Approach

For severe aplastic anemia, the National Comprehensive Cancer Network recommends immunosuppressive therapy with horse ATG plus cyclosporine, with response rates of 60-70% 1

For patients with aplastic anemia refractory to initial immunosuppressive therapy, the American Society of Hematology suggests adding eltrombopag to supportive care 1

The National Comprehensive Cancer Network indicates that immunosuppressive therapy with ATG and cyclosporine is particularly effective in younger patients with hypoplastic bone marrow and trisomy 8 2
HLA-DR15 positivity strengthens the indication for immunosuppressive therapy, especially in patients over 50 years with long-duration transfusion dependency, according to the National Comprehensive Cancer Network 2
Hypocellular bone marrow remains a significant predictor of achieving red blood cell transfusion independence with immunosuppressive therapy, as stated by the National Comprehensive Cancer Network 2

Patients should be monitored with complete blood counts and bone marrow evaluations to assess response, as recommended by the American College of Physicians 1

All blood products for aplastic anemia patients should be irradiated and filtered to prevent transfusion-associated complications, according to the American Association of Blood Banks 1

HLA typing and evaluation for bone marrow transplantation should be considered for eligible patients who fail to respond to immunosuppressive therapy, as suggested by the American Society of Hematology 1

The American Society of Hematology suggests that liver function abnormalities, including increased liver transaminases, are a critical safety concern in patients treated with eltrombopag, with a reported incidence of 13% of patients 3
Thrombosis is a treatment-related serious adverse event, and patients should be monitored for signs of thromboembolism, as reported in the Journal of Internal Medicine 4

Liver function tests should be monitored regularly to assess hepatotoxicity, with increased liver transaminases being the only dose-limiting toxicity, as reported in Blood 3
Patients should be monitored for rebound thrombocytopenia, which may occur upon discontinuation of eltrombopag, with worsening thrombocytopenia reported in some cases 5
Combination therapy with azacitidine in myelodysplastic syndrome has shown increased progression to AML, highlighting the need for careful consideration of combination therapy concerns, as reported in the Journal of Internal Medicine 4 and 5

The Blood Reviews guideline suggests that unlike ITP where gradual tapering is recommended, the aplastic anemia literature describes abrupt discontinuation in robust responders 6, 7

The American Society of Hematology suggests adding eltrombopag to supportive care for patients refractory to initial immunosuppressive therapy, with response rates of 40-48% 6, 7
For treatment-naïve severe aplastic anemia, combine eltrombopag with horse ATG plus cyclosporine starting from day 1 and continuing for 6 months, yielding complete response rates of 58% and overall response rates of 94% at 6 months 6, 7