Aplastic anaemia

Marrow failure (aplastic anaemia) may be primary, of which 75% are idiopathic acquired, and 25% secondary to a variety of agents, including chemicals (e.g. benzene), drugs and infections. Treatment is chosen according to the severity of the cytopenia, the age of the patient, the availability of a suitable bone marrow donor and, less commonly, the cause (if known). Good supportive treatment is important. The major therapeutic choice is between allogeneic bone marrow transplantation and immunosuppression, e.g. with antilymphocytic globulin and Ciclosporin; and perhaps haemopoietic growth factors (see above). Survival rates after allogeneic transplantation are in the region of 75-80% for data collected from transplant centres by the International Bone Marrow Transplant Registry, though chronic graft-versus-host disease causes continued morbidity.

Immunosuppression is used in patients who are not candidates for bone marrow transplantation due to age or to the lack of a donor (up to 70%). Horse antithymocyte globulin (ATG) or rabbit antilymphocyte globulin (ALG) induce haematological responses (transfusion-independence and freedom from infection) in 40-50%. The addition of Ciclosporin to ATG or ALG improves response rates to 70-80% and survival rates in responders to 90%. Adverse effects of ATG and ALG include anaphylaxis, exacerbation of cytopenias and serum sickness. Ciclosporin is nephrotoxic. In refractory patients G-CSF and erythropoetin can improve blood counts, as can androgens in some patients.

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