Treatment strategies for patients with severe aplastic anemia.

Treatment strategies for patients with severe aplastic anemia (SAA), depend on the severity of the disease, the age of the patient and the availability of a family donor. Progress in the past has included the early use of combined immunosuppressive therapy (IST) and better matching strategies to select unrelated donors. Currently, the actuarial 10-year survival in 2479 patients registered within the European Group for Blood and Marrow Transplantation (EBMT), is 73 and 68% for patients receiving first-line BMT or IST. The outcome of BMT has significantly improved since 1996, and this is true for both matched sibling donor BMT as well as for alternative donor BMT. Survival is significantly better in children (<16 years) as compared with adults (79 vs 68%, P<0.0001). In contrast, there has been no significant improvement over time for patients receiving IST. Again, results were significantly better in children compared with adults (81 versus 70%, P=0.001), especially in very severe aplasia (83 versus 62%, P=0.0002). This report outlines some of these results as a basis for treatment strategies in SAA.