תאריך: 31/12/2013
KEY POINTS
- Acquired aplastic anemia (AA) is an acquired condition of bone marrow failure characterized by peripheral pancytopenia and a hypoplastic bone marrow.
- There is increasing evidence that acquired AA is immune mediated.
- Bone marrow transplantation (BMT) is the recommended first-line therapy for patients with a human leukocyte antigen (HLA)-matched sibling donor, with 5-year survival rates exceeding 90%.
- Immunosuppressive therapy (IST) with horse antithymocyte globulin and cyclosporine is the recommended first-line therapy for patients without an HLA-matched sibling donor.
Survival rates are similar to those for BMT with a matched sibling donor, but relapse, clonal hematopoiesis, leukemia, autoimmunity, and cancer remain concerns that require long-term follow-up.
Pediatr Clin N Am 60 (2013) 1311–1336
https://dx.doi.org/10.1016/j.pcl.2013.08.011