Cord blood infusion-accelerated hematopoietic recovery after combined immunosuppressive therapy with fludarabine and rabbit ATG in severe aplastic anemia.

Immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG) and cyclosporine A (CsA) is currently the standard treatment for patients with severe aplastic anemia (SAA) who have no suitable donor or are ineligible for allogeneic stem cell transplantation. However, the delayed hematopoietic recovery, which accounts for most early deaths, remains a key problem. Thus, we designed an IST protocol with fludarabine, rabbit ATG, and CsA followed by unrelated cord blood (UCB) infusion to study whether hematopoiesis could be accelerated. Nineteen patients were enrolled in this study. The median neutrophil recovery time was 22 days and the treatment-related mortality within 3 months was 5.3%. The median platelet recovery time was 180 days. Six patients had transient or sustained UCB engraftment and the median platelet recovery time of these patients was significantly shorter than those who had no UCB engraftment (46 days vs 206 days, p = 0.006). The cumulative incidence of response rate at 12 months was up to 88.7% with CR rate of 72.2%. The overall survival at 2 years and 5 years was 94.7% and 78.9%, respectively. These results suggest that UCB infusion may play an important role in accelerating hematopoietic recovery in this protocol.