Idiopathic Aplastic Anemia in Children and Adults: Diagnosis, Treatments, and Management - A Review

Abstract

Aplastic Anemia (AA) is also known as idiopathic aplastic anemia (IAA) and the production of new blood cells ceases in AA, which leads to an abnormal hematological syndrome such as pancytopenia and suppression of hypo-cellular bone marrow. The pathophysiology of AA in most cases is immune-mediated and is stimulated by type 1 cytotoxic T cells. Reliable early diagnostic tests of IAA are not yet available, therefore most of the cases are identified in advanced stages. Recently, abnormal immune response and hematopoietic cell deficiencies are defined genetically, such as in target cells of telomere repair gene mutations and by the dysregulation of T-cell activation pathways. Importantly, anti-thymocyte globulins and cyclosporine-associated immunosuppression are successful treatments for restoring blood cell production in most of the cases, however, clonal hematologic diseases remain challenging. In the current review, we have discussed the common practices in the treatment, pathophysiology, diagnosis, and management of AA.

Keywords: Aplastic anemia; T-cell activation; hematologic diseases; immunosuppression; leukemia; pathophysiology.