Allogeneic bone marrow transplantation for severe aplastic anaemia

Allogeneic bone marrow transplantation (BMT) for aplastic anaemia was first introduced by E Donnall Thomas and Rainer Storb and colleagues in 1969. BMT or peripheral blood stem cell (PBSC) transfusion is the treatment of choice for young patients with SAA who have an HLA-identical sibling donor. The age at which transplantation is no longer the treatment of choice depends upon the severity of the disease, the general health of the patient and the nature of the presenting symptoms. The results of BMT collected by the European Blood and Marrow Transplant Registry (EBMTR) are shown in Figure 13.3. Overall survival is around 75-90%. There is a marked age effect, most notably with poor outcome for patient over the age of 40 years compared with younger patients. BMT has a better long-term survival than immunosuppressive treatment once the graft is firmly established and the patient is off immunosuppressive treatment. Patients are not at increased risk of later clonal disorders following a successful transplant. This has to be weighed against the increased risk of transplant-related mortality in the early post-transplant period and the poor quality of life if

Days after transplant

Figure 13.3 Survival following HLA-matched sibling donor stem cell transplantation. Data from EBMTR SAA working party, showing effect of age on outcome (with permission). The groups are not strictly similar, the oldest age group being transplanted significantly later than the youngest.

Days after transplant

Figure 13.3 Survival following HLA-matched sibling donor stem cell transplantation. Data from EBMTR SAA working party, showing effect of age on outcome (with permission). The groups are not strictly similar, the oldest age group being transplanted significantly later than the youngest.

the patient develops chronic graft-versus-host disease (GvHD) Early and late graft failure occur in 10-15% of patients. For patients over the age of 40 years, transplantation using a 'low-intensity' conditioning regimen may be reserved for those who fail to respond to ALG and have an HLA-identical sibling donor.

Bone marrow is the preferred source of stem cells rather than G-CSF-mobilized PBSCs due to a worse survival and higher incidence of chronic GvHD with PBSC. At least 3 X 108/kg of body weight of the recipient nucleated marrow cells (3 X 106 CD34+ cells) should be given; a lower stem cell dose increases the risk of graft failure.

The main causes of failure of BMT for aplastic anaemia are graft failure and post-graft infections. Patients with aplastic anaemia are immunocompetent apart from the neutropenia and indeed may be immunostimulated by infections and blood transfusions. Infection may be present before the transplant and become unmanageable afterwards. Chronic GvHD occurs in about 25% of successful transplants. Improvements in survival since 1969 may be attributed mainly to a reduction in graft failure and better support for the patient.

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