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Immunosuppressive Therapy May Offer Good Response Rate, Survival in Severe Aplastic Anaemia
A DGReview of :"Outcome of adult severe or very severe aplastic anemia treated with immunosuppressive therapy compared with bone marrow transplantation: multicenter trial"
International Journal of Hematology
09/29/2003
By Jill Taylor
Use of immunosuppressive agents (ISA) in the management of adult aplastic anaemia (AA) can achieve a high response rate and long-term survival regardless of disease severity, say researchers.
To compare survival rates and long-term complications between treatment by bone marrow transplantation (BMT) and immunosuppressive agents (ISA), M. J. Ahn and colleagues of Hanyang University in Seoul, Korea, evaluated 229 adult AA patients treated with ISA and 64 patients treated with BMT.
Researchers found a complete or partial response of 46.8% in 156 patients with severe or very severe AA who were treated with ISA (antithymocyte globulin or antithymocyte globulin plus cyclosporine), with 7.1% patient relapses.
One case each of acute leukaemia, myelodysplastic syndrome, and paroxysmal nocturnal haemoglobinuria were observed at long-term follow up.
The 6-year overall survival rate for the ISA treatment group was 69%. Factors associated with poor survival included ISA treatment response, disease severity, and low absolute neutrophil count (< 200/mm3). The only pre-treatment factor associated with poor survival was low absolute neutrophil count.
In the BMT treatment group, sustained engraftment occurred in 82.8% of patients and graft failure occurred in 12.5%. Grade II to IV acute graft-versus-host disease developed in 31.3% of patients and chronic graft-versus-host disease developed 18.8%.
The 6-year survival rate for the BMT treatment group was 79%, not statistically significant from the 65% survival rate of patients in the ISA treatment group who were 14 to 40 years of age. However, treatment with BMT in adult AA achieved long-term engraftment and a lower relapse rate than did treatment with ISA.
Factors such as patient age, gender, initial platelet count, aetiology, or treatment regimen did not significantly affect survival for either treatment group.
Further studies with a larger patient population and long-term follow-up are needed, the investigators conclude.
Int J Hematol 2003;78:2:133-138.
"Outcome of adult severe or very severe aplastic anemia treated with immunosuppressive therapy compared with bone marrow transplantation: multicenter trial"
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