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Cord Blood Stem Cell

Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency

Bone Marrow Transplantation volume 36, pages 295–299 (2005)Cite this article

Summary:

Primary immunodeficiencies (PID) are an important cause of childhood mortality. Haematopoietic stem cell transplantation (HSCT) is the best treatment for many PID. Umbilical cord stem cells are an alternative source of HSC. There is little data regarding outcome of umbilical cord stem cell transplantation (UCSCT) for PID. Our single centre experience is reported. A retrospective study of 14 of 148 patients transplanted for PID, who have received 15 UCSCT was performed, with specific regard to graft-versus-host disease (GvHD) and immune reconstitution. Eight patients with severe combined immunodeficiency (SCID), and six with other combined immunodeficiencies were treated. Of the patients, 12 received unrelated cords, and two had sibling transplants. Median age at transplant was 3.5 months, median nucleated cell dose was 0.8 × 108/kg. All engrafted. Median time to neutrophil engraftment was 22 days, median time to platelet engraftment was 51 days. One developed significant grade III GvHD post transplantation. In total, 11 patients had full donor T and six full donor B-cell chimerism, six of nine patients >1 year post-BMT had normal IgG levels and specific antibody responses to tetanus and Hib vaccines; two are being assessed. Two patients died of multi-organ failure related to pre-existing infection and inflammatory complications respectively. UCSCT should be considered for patients requiring stem cell therapy for PID.

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References

  1. Winkelstein JA, Marino MC, Johnston Jr RB et al. Chronic granulomatous disease. Report on a national registry of 368 patients. Medicine (Baltimore) 2000; 79: 155–169.

    Article  CAS  Google Scholar 

  2. Winkelstein JA, Marino MC, Ochs H et al. The X-linked hyper-IgM syndrome: clinical and immunologic features of 79 patients. Medicine (Baltimore) 2003; 82: 373–384.

    Article  CAS  Google Scholar 

  3. Sullivan KE, Mullen CA, Blaese RM, Winkelstein JA . A multi-institutional survey of the Wiskott-Aldrich syndrome. J Pediatr 1994; 125: 876–885.

    Article  CAS  PubMed  Google Scholar 

  4. Antoine C, Muller S, Cant A et al. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: a report of the European experience 1968–99. Lancet 2003; 361: 553–560.

    Article  PubMed  Google Scholar 

  5. Muller SM, Kohn T, Schulz AS et al. Similar pattern of thymic-dependent T-cell reconstitution in infants with severe combined immunodeficiency after human leukocyte antigen (HLA)-identical and HLA-nonidentical stem cell transplantation. Blood 2000; 96: 4344–4349.

    CAS  PubMed  Google Scholar 

  6. Cohen Y, Nagker A . Umbilical cord blood transplantation – how, when and for whom? Blood 2004; 18: 167–179.

    Article  Google Scholar 

  7. Gluckman E, Rocha V, Chevret S . Results of unrelated umbilical cord blood hematopoietic stem cell transplant. Transfus Clin Biol 2001; 8: 146–154.

    Article  CAS  PubMed  Google Scholar 

  8. Wagner JE, Barker JN, Defor ET et al. Treatment of unrelated donor umbilical cord blood in 102 patients with malignant and non-malignant diseases:influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival. Blood 2002; 100: 1611–1618.

    CAS  PubMed  Google Scholar 

  9. Knutsen AP, Wall DA . Umbilical cord blood transplantation in severe T-cell immunodeficiency disorders: two-year experience. J Clin Immunol 2000; 20: 466–476.

    Article  CAS  PubMed  Google Scholar 

  10. Benito A, Diaz MA, Alonso F et al. Successful unrelated umbilical cord blood transplantation in a child with Omenn's syndrome. Pediatr Hematol Oncol 1999; 16: 361–366.

    Article  CAS  PubMed  Google Scholar 

  11. Bonduel M, Pozo A, Zelazko M et al. Successful related umbilical cord blood transplantation for graft failure following T cell-depleted nonidentical bone marrow transplantation in a child with major histocompatibility complex class II deficiency. Bone Marrow Transplant 1999; 24: 437–440.

    Article  CAS  PubMed  Google Scholar 

  12. Fagioli F, Biasin E, Berger M et al. Successful unrelated cord blood transplantation in two children with severe combined immunodeficiency syndrome. Bone Marrow Transplant 2003; 312: 133–136.

    Article  Google Scholar 

  13. Bhattacharya A, Slatter M, Curtis A et al. Successful umbilical cord blood stem cell transplantation for chronic granulomatous disease. Bone Marrow Transplantation 2003; 31: 403–405.

    Article  CAS  PubMed  Google Scholar 

  14. Knutsen AP, Steffen M, Wassmer K, Wall DA . Umbilical cord blood transplantation in Wiskott-Aldrich syndrome. J Pediatr 2003; 142: 519–523.

    Article  PubMed  Google Scholar 

  15. 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.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  16. Filipovich AH . Unrelated cord blood transplantation for correction of genetic immunodeficiencies. J Pediatr 2001; 138: 459–461.

    Article  CAS  PubMed  Google Scholar 

  17. Slatter MA, Bhattacharya A, Barge D et al. Use of two unrelated umbilical cord stem cell units in stem cell transplantation for Wiskott-Aldrich Syndrome. Pediatr Blood Cancer (in press).

  18. Barker JN, Weisdorf DJ, DeFor TE et al. Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy. Blood 2005; 105: 1343–1347.

    Article  CAS  PubMed  Google Scholar 

  19. Dalal I, Reid B, Doyle J et al. Matched unrelated bone marrow transplantation for combined immunodeficiency. Bone Marrow Transplant 2000; 25: 613–621.

    Article  CAS  PubMed  Google Scholar 

  20. Gennery AR, Dickinson AM, Brigham K et al. CAMPATH-1M T cell depleted bone marrow transplantation for severe combined immunodeficiency: long-term follow up of 19 children treated in the period 1987–1998 in a single centre. Cytotherapy 2001; 3: 221–232.

    Article  CAS  PubMed  Google Scholar 

Download references

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Authors and Affiliations

  1. Paediatric Immunology Department, Newcastle General Hospital, Newcastle upon Tyne, UK

    A Bhattacharya, M A Slatter, T J Flood, M Abinun, A J Cant & A R Gennery

  2. Regional Blood Transfusion Centre, Holland Drive, Newcastle upon Tyne, UK

    C E Chapman

  3. Department of Immunology, Royal Victoria Infirmary, Newcastle upon Tyne Hospitals NHS Trust, UK

    D Barge

  4. Northern Molecular Genetics Service, Institute of Human Genetics. International Centre for Life, Newcastle upon Tyne, UK

    A Jackson

Authors
  1. A Bhattacharya
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  2. M A Slatter
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  3. C E Chapman
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  4. D Barge
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  5. A Jackson
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  6. T J Flood
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  7. M Abinun
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  8. A J Cant
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  9. A R Gennery
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Correspondence to A R Gennery.

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Bhattacharya, A., Slatter, M., Chapman, C. et al. Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency. Bone Marrow Transplant 36, 295–299 (2005). https://doi.org/10.1038/sj.bmt.1705054

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  • DOI: https://doi.org/10.1038/sj.bmt.1705054

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