Unrelated cord blood transplantation for newly diagnosed patients with severe acquired aplastic anemia using a reduced-intensity conditioning: high graft rejection, but good survival

Bone Marrow Transplant. 2012 Sep;47(9):1186-90. doi: 10.1038/bmt.2011.251. Epub 2012 Jan 16.

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Aplastic / drug therapy
  • Anemia, Aplastic / immunology
  • Anemia, Aplastic / surgery*
  • Antilymphocyte Serum / administration & dosage
  • Child
  • Child, Preschool
  • Cord Blood Stem Cell Transplantation / adverse effects
  • Cord Blood Stem Cell Transplantation / methods*
  • Cyclophosphamide / administration & dosage
  • Cyclosporine / administration & dosage
  • Female
  • Graft Rejection / etiology*
  • Graft Rejection / immunology
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Male
  • Middle Aged
  • Mycophenolic Acid / administration & dosage
  • Mycophenolic Acid / analogs & derivatives
  • Survival Analysis
  • Transplantation Conditioning / adverse effects
  • Transplantation Conditioning / methods*
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives
  • Young Adult

Substances

  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • Cyclosporine
  • Cyclophosphamide
  • Vidarabine
  • Mycophenolic Acid
  • fludarabine