The choice of primary treatment should be based on the availability of an HLA identical sibling, the age of the patient and the severity of the disease. Patients without a donor should be given immunosuppressive therapy (IS) as first line therapy. In the presence of a matched donor and young patients with severe or very severe aplastic anaemia, bone marrow transplantation (BMT) is probably the treatment of choice.

Bone Marrow Transplantation:

For patients not entered in a clinical trial, a preparative regimen with cyclophosphamide and antithymocyte globulin (ATG) and a post-transplant immunosuppression with MTX and ciclosporine A (CSA) is recommended (see schema in Figure 1).

Immunosuppressive Treatment:

Standard protocols for immunosuppression should contain antithymocyte globulin (ATG) and ciclosporine A (CSA) (see schema in Figure 2). The role of haematopoietic growth factors as adjunct to immunosuppressive treatment is evaluated in ongoing prospective randomised clinical trials (see Trials and Studies).