Initial management of patients with acquired aplastic anemia in the United States: results from a large national claims database.

Abstract

Acquired aplastic anemia (AA) is an immune-mediated disorder leading to bone marrow failure characterized by pancytopenia, with infectious and bleeding complications. The disease course may be complicated by paroxysmal nocturnal hemoglobinuria (PNH), necessitating screening with flow cytometry (FC) at the time of AA diagnosis. Management strategies vary based on disease severity. Severe AA patients are usually heavily transfusion-dependent (HT-AA) and typically treated with antithymocyte globulin, calcineurin inhibitor (CNI) and eltrombopag (EPAG) as triple therapy, while allogeneic hematopoietic stem cell transplant (HSCT) is often reserved for younger patients with matched sibling donors. Moderate AA patients are less transfusion-dependent (LT-AA) and may be observed or treated with CNI or EPAG. We conducted a retrospective cohort study using Blue Cross Blue Shield Axis database, examining adult patients diagnosed with AA between 07/01/2016 and 06/30/2022. We evaluated their management within the first 6 months following the diagnosis. Of 793 identified individuals (542 LT-AA, 251 HT-AA), with a median age of 49 years, only 42.6% received AA-directed therapy. Triple therapy and HSCT were infrequently used for patients with HT-AA (4.4% and 18.7%, respectively), while the most common treatment was the combination of a CNI and EPAG (LT-AA 37.8%, HT-AA 51.7%). The median time from diagnosis to treatment was 22 days, with older patients (age ≥ 40 years) experiencing treatment initiation delays (p = 0.03). FC testing was underutilized with only 55.5% of patients undergoing evaluation. These findings highlight the need for better access to diagnostic evaluation and appropriate AA-directed therapy for patients with AA in real-world settings.