Summary:
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 15.1×107/kg and 17×107/kg, respectively. Neutrophil engraftment was achieved on days +15 and +23, and platelet count >50×109/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.
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References
Rosen FS, Cooper MD, Wedgwood RJ . The primary immunodeficiencies. N Engl J Med 1995; 333: 431–440.
Gatti RA, Meuwissen HJ, Allen HD et al. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 1968; 2: 1366–1369.
Haddad E, Landais P, Friedrich W et al. Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: a European retrospective study of 116 patients. Blood 1998; 91: 3646–3653.
Buckley RH, Schiff SE, Schiff RI et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999; 340: 508–516.
Amrolia P, Gaspar HB, Hassan A et al. Nonmyeloablative stem cell transplantation for congenital immunodeficiencies. Blood 2000; 96: 1239–1246.
Toren A, Nagler A, Amariglio N et al. Successful human umbilical cord blood stem cell transplantation without conditioning in severe combined immune deficiency. Bone Marrow Transplant 1999; 23: 405–408.
Knutsen AP, Wall DA . Umbilical cord blood transplantation in severe T-cell immunodeficiency disorders: two-year experience. J Clin Immunol 2000; 20: 466–476.
Niehues T, Rocha V, Filipovich AH et al. Factors affecting lymphocyte subset reconstitution after either related or unrelated cord blood transplantation in children – a Eurocord analysis. Br J Haematol 2001; 114: 42–48.
Moretta A, Maccario R, Fagioli F et al. Analysis of immune reconstitution in children undergoing cord blood transplantation. Exp Hematol 2001; 29: 371–379.
Elder ME, Skoda-Smith S, Kadlecek TA et al. Distinct T cell developmental consequences in humans and mice expressing identical mutations in the DLAARN motif of ZAP-70. J Immunol 2001; 166: 656–661.
Chilosi M, Facchetti F, Notarangelo LD et al. CD30 cell expression and abnormal soluble CD30 serum accumulation in Omenn's syndrome: evidence for a T helper 2-mediated condition. Eur J Immunol 1996; 26: 329–334.
Aleman K, Noordzij JG, de Groot R et al. Reviewing Omenn syndrome. Eur J Pediatr 2001; 160: 718–725.
Rubinstein P, Dobrila L, Rosenfield RE et al. Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution. Proc Natl Acad Sci USA 1995; 92: 10119–10122.
Appleton AL, Curtis A, Wilkes J et al. Differentiation of materno-fetal GVHD from Omenn's syndrome in pre-BMT patients with severe combined immunodeficiency. Bone Marrow Transplant 1994; 14: 157–159.
Barata LT, Henriques R, Hivroz C et al. Primary immunodeficiency Secondary to ZAP-70 deficiency. Acta Med Port 2001; 14: 413–417.
Wagner JE, Kernan NA, Steinbuch M et al. Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease. Lancet 1995; 346: 214–219.
Comans-Bitter WM, de Groot R, van den Beemd R et al. Immunophenotyping of blood lymphocytes in childhood. Reference values for lymphocyte subpopulations. J Pediatr 1997; 130: 388–393.
Acknowledgements
This work was supported by grants from the Associazione Italiana per la Ricerca sul Cancro (AIRC, Milano), from the Ministero dell'Università e della Ricerca Scientifica e Tecnologica (MURST, Roma) to E.M. and from the Associazione Donatrici Italiane di Sangue di Cordone Ombelicale (ADISCO, Sezione Piemonte).
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Fagioli, F., Biasin, E., Berger, M. et al. Successful unrelated cord blood transplantation in two children with severe combined immunodeficiency syndrome. Bone Marrow Transplant 31, 133–136 (2003). https://doi.org/10.1038/sj.bmt.1703800
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DOI: https://doi.org/10.1038/sj.bmt.1703800
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