Successful long-term hematological and immunological reconstitution by autologous cord blood transplantation combined with post-transplant immunosuppression in two children with severe aplastic anemia

Pediatr Transplant. 2019 Feb;23(1):e13320. doi: 10.1111/petr.13320. Epub 2018 Nov 8.

Abstract

aUCBT is a valuable curative option in pediatric patients with refractory idiopathic SAA and no available matched sibling or unrelated donors. Experience in the use of autologous cord blood units in patients with SAA is limited and private for-profit cord blood-banking programs are controversial. We report the successful treatment of two patients with SAA, aged 15 and 24 months, with autologous cord blood combined with immunosuppression. After conditioning with 200 mg/kg cyclophosphamide and ATG, 7.5 mg/kg, 32.2 × 107 /kg, and 3.8 × 107 /kg autologous cord blood nucleated cells were infused, respectively. One of our patients underwent transplantation after failure of IST. Both patients received post-transplant immunosuppression with cyclosporine for 12 months. They remain disease-free 6 years post-transplantation.

Keywords: autologous umbilical cord blood transplantation; immunosuppression; severe aplastic anemia.

Publication types

  • Case Reports

MeSH terms

  • Anemia, Aplastic / blood
  • Anemia, Aplastic / immunology
  • Anemia, Aplastic / therapy*
  • Child, Preschool
  • Combined Modality Therapy
  • Cord Blood Stem Cell Transplantation / methods*
  • Cyclosporine / therapeutic use*
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Male
  • Transplantation, Autologous

Substances

  • Immunosuppressive Agents
  • Cyclosporine