Outcomes of Unrelated Cord Blood Transplantation for 18 Patients with Severe Aplastic Anemia

Objective To evaluate the outcomes of unrelated cord blood transplantation (UCBT) for severe aplastic anemia (SAA).

Methods The clinical data of 18 SAA patients, including 9 very severe aplastic anemia (vSAA), received UCBT from Sep 2009 to Feb 2017 were analyzed retrospectively. Three patients received a single unrelated cord blood unit, fifteen patients received double unrelated cord blood units. One patient was 4/6 HLA match, nine patients were 5/6 HLA match, eight patients were 6/6 HLA match. The conditioning regimens were as follow: 1 patient received modified BU/CY- based regimen, 1 patient received fludarabine (Flu) + antithymocyte globulin (ATG) + cyclophosphamide (CY) + total body irradiation (TBI) regimen, 16 patients received fludarabine (Flu) + antithymocyte globulin (ATG) + cyclophosphamide (CY) regimens. Prophylaxis for graft-versus host disease (GVHD): cyclosporine (CsA) plus short-term methotrexate (MTX).

Results There was1 patient early death on day +2, 6 patients experienced primary graft rejection, but 5 of them acquired autologous myeloid recovery, 11 patients were engrafted successfully. The median days of neutrophils(ANC)above 0.5 × 10⁹/L and platelets (PLT) more than 20 × 10⁹/L were 18.5(9~29)days and 26.5(20~48)days, respectively. Six patients (54.5%) developed acute GVHD (aGVHD), 1 for grade I aGVHD, 3 for grade Ⅱ aGVHD, 2 for grade Ⅳ aGVHD. Of 9 patients, 2 occurred chronic GVHD (cGVHD), 1 for mild cGVHD, 1 for severe cGVHD. After a median follow-up of 56(4 ∼ 72)months, 9 patients (50.0%) died, out of which one died of severe aGVHD, the reasons of death were GVHD, severe infection, posttransplant lymphoproliferative disorders (PTLD) and transplant-associated thrombotic microangiopathy (TMA). The 5-year estimated overall survival was 46.9% ± 12.3%. No patient relapsed.

Conclusion UCBT is an effective therapy for SAA patients who lack an appropriate matched related donor.