Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anaemia (SAA): a report of the EBMT* SAA Working Party

This is an analysis of 509 patients with severe aplastic anaemia (SAA) treated in Europe between 1981 and 1986; 218 patients were treated by allogeneic bone marrow transplantation (BMT) from HLA identical sibling donors and 291 with immunosuppressive therapy (IS) with antilymphocyte globulin (ALG). The overall actuarial survival was 63% after BMT and 61% after IS therapy at 6 years. All patients fulfilled the criteria of SAA; however, most patients with a neutrophil count of less than 0.2 x 10(9)/l also had infections and haemorrhages. Therefore a further subclassification was defined by pretreatment peripheral blood neutrophil count: very severe aplastic anaemia (vSAA) (less than 0.2 x 10(9)/l neutrophils) and moderately severe aplastic anaemia (mSAA) (0.2-0.5 x 10(9)/l neutrophils). A Cox regression analysis showed that the only significant pre-treatment variables were a low neutrophil count (P = 0.001) and increasing age (P = 0.05). Thus it seemed reasonable to analyse survival data after combined stratification for neutrophils (vSAA versus mSAA) and age (cut off at 20 years). BMT was superior to IS in patients with vSAA under 20 years of age (64% v. 38%; P = 0.01). IS was superior to BMT in patients with mSAA aged 20 or more (82% v. 62%; P = 0.002). The two treatments gave comparable results in young patients with mSAA (BMT = 58%, IS = 62%; P = 0.1), and in older patients with vSAA (BMT = 44%, IS = 43%; P = 0.06). Overall 75/218 and 87/291 patients, given BMT or IS respectively, died. The major cause of failure in BMT patients was graft rejection (n = 22) or problems associated with graft-versus-host disease. For ALG patients the major problem was persistence of the aplasia with haemorrhage (n = 32) or infections (n = 46). This study indicates that over 60% of patients with SAA can be successfully treated with either BMT or IS. Overall survival does not differ in the two groups, though significant differences emerge after stratification for severity of the aplasia and age.