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  • Title: Successful unrelated umbilical cord blood transplantation in a child with Omenn's syndrome.
    Author: Benito A, Diaz MA, Alonso F, Fontán G, Madero L.
    Journal: Pediatr Hematol Oncol; 1999; 16(4):361-6. PubMed ID: 10407874.
    Abstract:
    Omenn's syndrome is a variant of combined immunodeficiency disease (CID). Like other CID forms, it causes death unless the patient receives a bone marrow transplant (BMT). Previous reports have shown that BMTs from unrelated donors in Omenn's syndrome have very poor results, with a high rate of infections during transplantation and graft rejection, when compared with transplants from related donors or patients with other CID. This study discusses the case of a 19-month-old child with Omenn's syndrome, who received an unrelated cord blood stem cell transplant (CBT). Donor and recipient had 1 HLA-Ag mismatched on HLA-B. Symptomatology improved early after CBT. The child achieved leukocytes and platelet engraftment and was discharged on day +34. His follow-up has been uneventful and at this time, 27 months after CBT, immune functions have been recovered.
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