Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe XXXX (SAA): a report of the EBMT SAA working party.

@entity1 @entity548 @entity2150 @entity281 @entity2132 @entity3894
This is an analysis of 509 @entity1 with severe @entity2150 (SAA) treated in Europe between 1981 and 1986; 218 @entity1 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 @entity1 fulfilled the criteria of SAA; however, most @entity1 with a neutrophil count of less than 0.2 x 10(9)/l also had @entity281 and @entity548 . Therefore a further subclassification was defined by pretreatment peripheral blood neutrophil count: very severe @entity2150 (vSAA) (less than 0.2 x 10(9)/l neutrophils) and moderately severe @entity2150 (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 @entity1 with vSAA under 20 years of age (64% v. 38%; P = 0.01). IS was superior to BMT in @entity1 with mSAA aged 20 or more (82% v. 62%; P = 0.002). The two treatments gave comparable results in young @entity1 with mSAA (BMT = 58%, IS = 62%; P = 0.1), and in older @entity1 with vSAA (BMT = 44%, IS = 43%; P = 0.06). Overall 75/218 and 87/291 @entity1 , given BMT or IS respectively, died. The major cause of failure in BMT @entity1 was graft rejection (n = 22) or problems associated with @entity2132 . For ALG @entity1 the major problem was persistence of the @entity3894 with @entity548 (n = 32) or @entity281 (n = 46). This study indicates that over 60% of @entity1 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 @entity3894 and age.
