Tegenwoordig gunstige prognose na HLA-identieke beenmergtransplantatie bij kinderen met verworven ernstige aplastische anemie; evaluatie van 30 jaar beenmergtransplantaties in het Leids Universitair Medisch Centrum

To evaluate the results of 30 years of allogeneic HLA-identical bone marrow transplantation (BMT) as the treatment for children with acquired severe aplastic anaemia.

Design

Retrospective, descriptive.

Method

Of all patients who underwent an HLA-identical sibling-donor BMT for severe aplastic anaemia at the Department of Paediatrics, Leiden University Medical Center, in the period 1971-2000, and had a follow-up period of at least 1 year, the medical data were reviewed. The patients were split into 2 groups: patients transplanted before 1989 (n = 24), and patients who had their BMT from 1989 onwards (n = 20). This was due to a change in the treatment policy, namely a reduction in the period between diagnosis and BMT, resulting in fewer blood transfusions as well as changes in the prophylaxis against graft- versus-host disease (GvHD) from 1989 onwards (combination therapy using methotrexate and cyclosporin).

Results

There was an increase in the 1-year actuarial survival rate from 67 in the period before 1989 to 90 thereafter. The incidence of GvHD has significantly decreased since the introduction, in 1989, of the combination therapy using methotrexate and cyclosporin, with only 1/20 patients suffering from acute GvHD versus 13/24 prior to 1989 (p = 0.002). No patients acquired chronic GvHD after 1989, whereas before 1989, 10 patients had acquired this (p = 0.001).

Conclusion

The prognosis of allogeneic HLA-identical sibling transplantation for paediatric patients with severe aplastic anaemia has considerably improved over the last 30 years due to improved supportive care, a significant decrease in GvHD and a shorter period between diagnosis and BMT, with the result that less blood transfusions have been required and less sensitisation has occurred. The long-term survival chance has increased to 90.