Management of acquired aplastic anaemia

Outcome of patients with aplastic anaemia (AA), whether treated with allogeneic BMT or immunosuppressive therapy has steadily increased over the last three decades. However, there is a difference in quality of outcome between these two therapeutic modalities. There is no plateau for survival after ATG as patients are at later risk of transformation to myelodysplasia (MDS) or acute myeloid leukaemia (AML), paroxysmal nocturnal haemoglobinuria and relapse of their aplasia. In contrast, AA patients are not at risk of these later complications if they have undergone successful bone marrow transplantation. Long term survival after HLA identical sibling BMT is 80–90%, but GVHD and graft rejection remain to be addressed. The results of unrelated donor BMT for AA have shown considerable improvement over the last five years. Difficulties remain for those patients who fail immunosuppressive therapy and in whom BMT is not possible, since alternative immunosuppressive agents have so far proven to be somewhat disappointing.