Front-line immunosuppressive treatment of acquired aplastic anemia

In this article, front-line innmunosuppressive therapy (IST) for acquired plastic anemia (AA) is illustrated and discussed. Also second-line and salvage options are briefly illustrated. First-line IST should consist of horse anti-thymocyte globulin (ATG) and CsA that has been shown to result in response rates between 60 and 80%. CsA should be given for 12 months until transfusion independence is achieved and then tapered very slowly in the presence of a CR. Patients with a partial response are usually continued on CsA. Tight monitoring of the blood count during CsA tapering is necessary to identify early loss of response. G-CSF 5 mu g/kg/day s.c. in the first 30 days has been shown to reduce infections and hospitalization and to identify early responders, as those who achieve neutrophils count >= 0.5 x 10(9)/L by day + 30. This schedule is recommended in the first month of therapy. Afterward, G-CSF can be considered in neutropenic febrile episodes. Patients not achieving transfusion independence after a first course of IST may be considered for second-line IST, or for an allogeneic hematopoietic SCT depending on patient age, ongoing infection, neutrophil count and transfusion requirements. Third-line IST is rarely given, but some options are discussed. Bone Marrow Transplantation (2013) 48, 174-177; doi:10.1038/bmt.2012.222; published online 19 November 2012