title: Unrelated cord blood transplantation for newly diagnosed patients with severe acquired aplastic anemia using a reduced-intensity conditioning: high graft rejection, but good survival.
abstract: We report a single-center experience in treating 18 consecutive patients with severe aplastic anemia (SAA) who received unrelated cord blood transplantation (CBT). The median age was 17 years (range 5-61 years). Sixteen cases received a reduced-intensity regimen composed of CY (total dose 1200 mg/m(2)), rabbit antithymocyte globulin (ATG, total dose 30 mg/kg) and fludarabine (FLU, total dose 120 mg/m(2)). CYA and mycophenolate mofetil were used as GVHD prophylaxis. Two patients were not evaluable for engraftment because of early death on day +21 and +22. Only one of the sixteen cases achieved engraftment, but experienced secondary graft failure 3 months post transplantation. Fifteen patients experienced primary graft rejection, but all of them acquired autologous recovery. The 3-month and 6-month cumulative incidence of response was 56% and 81%, respectively. So far, 16 patients have survived for 330-1913 days (median, 750 days) after transplantation. The probability of OS at 2 years was 88.9%. Our data indicate that CBT for newly diagnosed SAA using no irradiation but FLU and ATG-based conditioning still seems to inevitably lead to the high risk of rejection, but may facilitate autologous recovery and improve survival with low risk of transplant-related mortality.
authors.affiliation: Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei, China.
authors.forename: H-L
authors.initials: HL
authors.lastname: Liu
authors.forename: Z-M
authors.initials: ZM
authors.lastname: Sun
authors.forename: L-Q
authors.initials: LQ
authors.lastname: Geng
authors.forename: X-B
authors.initials: XB
authors.lastname: Wang
authors.forename: K-Y
authors.initials: KY
authors.lastname: Ding
authors.forename: B-I
authors.initials: BI
authors.lastname: Tang
authors.forename: J
authors.initials: J
authors.lastname: Tong
authors.forename: Z-Y
authors.initials: ZY
authors.lastname: Wang
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000188:drug therapy / Q000276:immunology / Q000601:surgery*; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D003520:Cyclophosphamide / Q000008:administration & dosage; D016572:Cyclosporine / Q000008:administration & dosage; D005260:Female; D006084:Graft Rejection / Q000209:etiology* / Q000276:immunology; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000031:analogs & derivatives; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods*; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D003520:Cyclophosphamide; D014740:Vidarabine; D009173:Mycophenolic Acid; C024352:fludarabine