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

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.

• Authors: F Fagioli, E Biasin, M Berger, F Nesi, E H Saroglia, R Miniero, S Martino, P A Tovo
• Journal: Bone marrow transplantation (Bone Marrow Transplant) Vol. 31 Issue 2 Pg. 133-6 (Jan 2003) ISSN: 0268-3369 [Print] England
• PMID: 12621496 (Publication Type: Case Reports, Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Antilymphocyte Serum
• Immunosuppressive Agents
• Cyclosporine
• Busulfan

Topics

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