Immunosuppressive therapy in severe aplastic anemia

Severe aplastic anemia, a disease characterized by pancytopenia and a hypocellular marrow, is treatable by either immunosuppressive therapy (IST) or hematopoietic stem cell transplant. Much is understood about the immune-mediated pathophysiology of AA now, but the inciting factor remains elusive. Many groups around the globe contributed to understanding the disease pathophysiology and optimizing the IST regimen. Horse antithymocyte globulin and cyclosporine, the initial IST regimen, achieved a hema-tologic response rate in about 60% to 65% of treated patients, with less than 10% achieving a complete count recovery. However, adding a thrombopoietin receptor agonist, eltrombopag (EPAG), to IST improved these response rates to nearly 80% and an unprecedented increase in complete response to almost 40%. The latest report indicates that a high-risk clonal evolution to myeloid malignancies is not increased with hematopoietic stem cell stimulation by adding EPAG in the front line setting. Despite the great success of IST and EPAG in improving early outcomes, relapse remains a problem. Further optimization of up -front therapy and treatment protocol is needed to prevent relapses and decrease clonal evolution rates for even better long-term results. (c) 2022 Published by Elsevier Inc.

Automated summary

Severe aplastic anemia, a disease characterized by pancytopenia and a hypocellular marrow, can be treated with immunosuppressive therapy (IST) or hematopoietic stem cell transplant. Adding eltrombopag (EPAG) to the IST regimen improves treatment response rates without increasing the risk of myeloid malignancies. However, relapse remains a challenge, and further optimization of treatment protocols is needed.