Successful unrelated cord blood transplantation in two children with severe combined immunodeficiency syndrome

Bone Marrow Transplant. 2003 Jan;31(2):133-6. doi: 10.1038/sj.bmt.1703800.

Abstract

We describe the successful unrelated cord blood transplantation in two patients affected by a Zap-70 deficiency and an Omenn-like syndrome, respectively. The patients were hospitalised for recurrent infections at the age of 13 and 2 months, respectively. An unrelated cord blood unit was found for each. The conditioning regimen was cyclophosphamide, busulfan and antithymocyte globulin. The total number of infused cells was 15.1 x 10(7)/kg and 17 x 10(7)/kg, respectively. Neutrophil engraftment was achieved on days +15 and +23, and platelet count >50 x 10(9)/l was achieved on days +21 and +52, respectively. One patient presented acute Graft-versus-host disease (GVHD) grade I and the other grade III. Chimerism was mixed and full donor. Normal lymphoproliferative response to mitogens and alloantigens was detectable at 6 months for both. No chronic GVHD was observed in either. The patients are alive and well at 53 and 15 months after transplantation. In conclusion, umbilical cord blood represents a valid alternative source of haemopoietic stem cells.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antilymphocyte Serum / therapeutic use
  • Busulfan / therapeutic use
  • Cyclosporine / therapeutic use
  • Fetal Blood*
  • Gastroenteritis / complications
  • Gastroenteritis / microbiology
  • Graft vs Host Disease / prevention & control
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Infant
  • Infant, Newborn
  • Salmonella Infections / complications
  • Severe Combined Immunodeficiency / etiology
  • Severe Combined Immunodeficiency / immunology
  • Severe Combined Immunodeficiency / therapy*
  • Stem Cell Transplantation / methods*
  • T-Lymphocytes / immunology
  • Transplantation Chimera
  • Transplantation Conditioning / methods
  • Transplantation, Homologous
  • Treatment Outcome

Substances

  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • Cyclosporine
  • Busulfan