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Non Malignant Diseases

Stem cell transplantation for primary immunodeficiencies

Bone Marrow Transplantation volume 41, pages S83–S86 (2008)Cite this article

Abstract

BMT is curative in almost 75% of children affected by severe primary immunodeficiencies (PIDs). Recently, the chance of cure has increased thanks to the availability of matched unrelated donors (MUDs). Nevertheless, besides the conventional indications to BMT (profound or absent T-cell function, profound or absent natural killer function, known syndromes with T-cell deficiencies), indications to BMT for PIDs affecting the quality of life or having an expectation of life that does not exceed the third-fourth decade remain unclear. Infact, if it is evident that the survival rate in an infant grafted for a PID with a MUD is expected to be more than 80%, alternative treatments such as gene therapy are now available.

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References

  1. Porta F, Friedrich W . Bone marrow transplantation in congenital immunodeficiency diseases. Bone Marrow Transplant 1998; 21 (Suppl 2): S21–S23. Review.

    PubMed  Google Scholar 

  2. Bertand Y, Landais P, Friedrich W, Gerritsen B, Morgan G, Fasth A et al. Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for blood and marrow transplantation and the European society for immunodeficiency. J Pediatr 1999; 134: 740–748.

    Article  Google Scholar 

  3. Lanfranchi A, Verardi R, Tettoni K, Neva A, Mazzolari E, Pennacchi M et al. Haploidentical peripheral blood and marrow stem cell transplantation in nine cases of primary immunodeficiency. Haematologica 2000; 85: 41–46.

    CAS  Google Scholar 

  4. Seger RA, Gungor T, Belohradsky BH, Blanche S, Bordigoni P, Di Bartolomeo P et al. Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985–2000. Blood 2002; 100: 4344–4350. Review.

    Article  CAS  Google Scholar 

  5. Driesse GJ, Gerritsen EJ, Fischer A, Fasth A, Hop WC, Veys P et al. Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report. Bone Marrow Transplant 2003; 32: 657–663.

    Article  Google Scholar 

  6. Bartolome J, Porta F, Lanfranchi A, Rodriguez-Molina JJ, Cela E, Cantalejo A et al. B cell function after haploidentical in utero bone marrow transplantation in a patient with severe combined immunodeficiency. Bone Marrow Transplant 2002; 29: 625–628.

    Article  CAS  Google Scholar 

  7. Pirovano S, Notarangelo LD, Malacarne F, Mazzolari E, Porta F, Lanfranchi A et al. Reconstitution of T-cell compartment after in utero stem cell transplantation: analysis of T-cell repertoire and thymic output. Haematologica 2004; 89: 450–461.

    CAS  PubMed  Google Scholar 

  8. Mazzolari E, Moshous D, Forino C, De Martiis D, Offer C, Lanfranchi A et al. Hematopoietic stem cell transplantation in Omenn syndrome: a single-center experience. Bone Marrow Transplant 2005; 36: 107–114.

    Article  CAS  Google Scholar 

  9. Grunebaum E, Mazzolari E, Porta F, Dallera D, Atkinson A, Reid B et al. Bone marrow transplantation for severe combined immune deficiency. JAMA 2006; 295: 508–518.

    Article  CAS  Google Scholar 

  10. Pai SY, DeMartiis D, Forino C, Cavagnini S, Lanfranchi A, Giliani S et al. Stem cell transplantation for the Wiskott–Aldrich Syndrome: a single-centre experience confirms efficacy of matched unrelated transplantation. Bone Marrow Transplant 2006; 38: 671–679.

    Article  Google Scholar 

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Authors and Affiliations

  1. Oncology-Haematology and BMT Unit, Ospedale dei Bambini, Spedali Civili, Brescia

    F Porta, C Forino, D De Martiis, E Soncini, L Notarangelo, K Tettoni, C D'Ippolito, R Soresina, K Shiha, S Berta, R Baffelli, F Bolda, A Bosi, F R Schumacher, A Lanfranchi & E Mazzolari

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  1. F Porta
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  2. C Forino
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  3. D De Martiis
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  4. E Soncini
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  5. L Notarangelo
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  14. F R Schumacher
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  15. A Lanfranchi
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  16. E Mazzolari
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Correspondence to F Porta.

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Porta, F., Forino, C., De Martiis, D. et al. Stem cell transplantation for primary immunodeficiencies. Bone Marrow Transplant 41 (Suppl 2), S83–S86 (2008). https://doi.org/10.1038/bmt.2008.61

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