New hope for patients with severe aplastic anemia

摘要

Severe aplastic anemia (SAA) is a rare and serious blood disorder in which a patient's bone marrow fails to generate enough erythrocytes, leukocytes and platelets 1. As a result, people living with SAA may experience debilitating symptoms and complications, such as fatigue, trouble breathing, recurring infections and abnormal bruising or bleeding, which can limit their daily activities 1. When untreated, almost all of the patients die within one year after diagnosis 2. The current standard of care includes immunosuppressive therapy (1ST) or hematopoietic stem cell transplantation. However, one quarter to one third of patients will not respond to 1ST, and 30-40 of responders will relapse, with symptoms returning 3. Hematologic response is strongly correlated with long-term survival 4.