Unrelated Cord Blood Transplantation for Severe Aplastic Anemia

Abstract In the present study we evaluated the feasibility of unrelated cord blood transplantation (UCBT) in patients with severe aplastic anemia (SAA). The outcome of 31 SAA patients (median age 28; range: 0.9-72.3 years old) who received UCBT was analyzed. The cumulative incidences of the neutrophil and platelet recovery after UCBT were 54.8 and 72.2%, respectively (95% confidence interval [CI] = 36.0%-70.3% and 51.3%-85.3%, respectively). The cumulative incidences of grade ≥II acute and chronic graft-versus-host disease (aGVHD, cGVHD) were 17.1% (95% CI=6.2%-32.8%) and 19.7% (95% CI=6.2%-38.8%), respectively. Currently, 13 patients are alive, having survived for 33.7 months (median; range: 6-77 months) after UCBT. The probability of overall survival (OS) at 2 years was 41.1% (95% CI=23.8%-57.7%). A conditioning regimen that included low-dose total body irradiation (TBI) (2-5 Gy), fludarabine, and cyclophosphamide resulted in a favorable OS (80%; 95% CI=20.4%-96.9%). This result suggests that UCBT using the optimal conditioning regimen can be a salvage treatment for patients without a suitable bone marrow donor and warrants evaluation in further prospective studies.