Rapid and complete reconstitution of autologous haemopoiesis after cord blood infusion in treatment‐naive patients with severe aplastic anemia receiving high‐dose cyclophosphamide/ATG therapy

Abstract Although high‐dose cyclophosphamide seems to achieve durable complete remission, there are still concerns about its too much early toxicity. So, we designed a clinical study to investigate the effects of high‐dose cyclophosphamide/ ATG combined with cord blood infusion as first‐line therapy for patients with severe aplastic anemia. Patients and Method: Between January 2003 and September 2007, we treated 16 treatment‐naive patients with severe aplastic anemia with cord blood infusion after high‐dose cyclophosphamide (50 mg/kg/d × 2) and rabbit antithymocyte globulin (3 mg/kg/d × 5) therapy. Results: Although only one patient had durable full donor engraftment, 14 of the enrolled 16 patients had rapid autologous hematopoietic recovery. The median recovery time for neutrophils and platelets was only 23 and 37 d after infusion of cord blood. Of the 15 responding patients, all patients achieved treatment‐free remission: nine patients met the criteria for a complete remission; six patients achieved a partial remission. Conclusion: Infusion of cord blood after high‐dose cyclophosphamide/ ATG resulted in a rapid autologous hematologic recovery and a high response rate in patients with treatment‐naive patients with severe aplastic anemia. These promising results merit further investigation and confirmation on a larger number of patients.