Outcome of Patients with Aplastic Anemia Given First Line Transplantion or Immunosuppression in the Last Decade: A Report from European Group for Blood and Marrow Transplantation (EBMT).

Background Changes have occurred in the treatment of acquired severe aplastic anemia (SAA) over the past decade,and the optimal treatment for children and adults is often debated.

Aim of the study. Aim of this study was to assess whether the outcome of patients with SAA has improved in two sequential time periods, 1990–1995 and 1996–2002.

Patients and methods., We analyzed a total of 2479 consecutive patients with SAA classified according to first line treatment: allogeneic bone marrow transplantation (BMT) (n=1567) or immunosuppressive therapy (IS) (n=912), and stratified for age (</=> 16 years).

Transplantation. The overall actuarial 5 year survival was 74% before 1995 and has not change overall (it is currently 75%). Survival was improved in the two time periods, for patients given first line BMT (69% vs 79%, p=0.001) but not in patients given first line IS (76% vs 72%, p=0.3). In the latter patients there was a confounding effect of disease severity: patients with very severe AA (neutrophils <200) improved their autcome after IS therapy in the two time periods though not significantly (72% vs 82%, p=0.1); patients with SAA (neutrophils 200–500) had superimposable outcome (76%) and patients with non severe AA actually had worse outcome in the most recent period (77% vs 55%, p=0.01). Overall improvement was significant in children (p=0.02) but not in adults (p=0.4). In multivariate COX analysis negative predictors of survival were a long interval between diagnosis and first treatment (>50 days, RR 1.5, p=0.00001) and age > 16 (RR1.5, p=0.0001).

Interpretation and conclusions. This study shows that outcome has improved in the past decade in subsets of patients with SAA: BMT patients and IS patients if they have very severe aplasia. Early intervention should be reccommended in all patients, whathever the first line therapy, due the very strong effect of interval between diagnosis and treatment on survival.