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

Acquired aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (AA) is an immune-mediated disorder leading to bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… characterized by pancytopenia pancytopenia: A shortage of all types of blood cells - red blood cells, white blood cells, and platelets. , with infectious and bleeding complications. The disease course may be complicated by paroxysmal nocturnal hemoglobinuria paroxysmal nocturnal hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH), necessitating screening with flow cytometry flow cytometry: (sy-TOM-uh-tree) A laboratory test that gives information about cells, such as size, shape, and percentage of live cells. Flow cytometry is the test doctors use to see if there are any proteins missing from the surface of blood cells. It is the standard test for confirming a diagnosis of paroxysmal… (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 antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. , calcineurin inhibitor (CNI) and eltrombopag eltrombopag: What are the possible side effects of eltrombopag (Promacta)? Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat. Stop using eltrombopag and call your doctor at once if you have: … (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.