Long-term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation

A total of 100 children under the age of 17 years with acquired aplastic an aemia (AA) were initially treated with immunosuppressive therapy (IST) (n = 63) or bone marrow transplantation (BMT) (n = 37) from an HLA-matched fami ly donor. The projected 10-year survival rates were 55 +/- 8% and 97 +/- 3% respectively (P = 0.004). Because the IST group included 11 non-responders who were salvaged by BMT from an HLA-matched unrelated donor, we compared failure-free survival (FFS) between the groups. The probability of FFS at 1 0 years was 97 +/- 3% for the BMT group, compared with 40 +/- 8% for the IS T group (P = 0.0001). Seven patients evolved to myelodysplastic syndrome (M DS) with monosomy 7 and the estimated cumulative incidence of MDS 10 years after diagnosis was 20 +/- 7% in the IST group. We compared the outcome of children treated with IST during the two consecutive periods of 1983-91 (gr oup A, n = 40) and 1991-8 (group B, n = 23) to assess the impact of combine d therapy with antithymocyte globulin and cyclosporin. The probability of F FS at 7 years follow-up was the same in the two groups (50 +/- 8% vs. 40 +/ - 15%, P = 0.40). We recommend BMT as first-line therapy in paediatric seve re AA patients with an HLA-matched family donor. Alternative donor BMT is r ecommended as salvage therapy in patients who relapse or do not respond to initial IST.