Unresolved issues in hematopoietic stem cell transplantation for severe combined immunodeficiency: Need for safer conditioning and reduced late effects

Journal of Allergy and Clinical Immunology

Volumn 131, Issue 5, 2013, Pages 1306-1311

  Horn, Biljana ^a   Cowan, Morton J ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

alternative donors; immune reconstitution; late effects of transplantation; Severe combined immunodeficiency; T cell depleted stem cell transplantation

Indexed keywords

DNA; FLUDARABINE;

ARTICLE; CELLULAR IMMUNITY; CONSENSUS; DNA REPAIR; DONOR SELECTION; GENOTYPE; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; LYMPHOCYTE FUNCTION; MYELOABLATIVE CONDITIONING; PHENOTYPE; PRIORITY JOURNAL; SEVERE COMBINED IMMUNODEFICIENCY; VDJ RECOMBINATION;

DONOR SELECTION; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; SEVERE COMBINED IMMUNODEFICIENCY; TIME FACTORS; TRANSPLANTATION CONDITIONING;

EID: 84876932180     PISSN: 00916749     EISSN: 10976825     Source Type: Journal    
DOI: 10.1016/j.jaci.2013.03.014     Document Type: Article

References (22)

1. A.S. O'Marcaigh, K. DeSantes, D. Hu, H. Pabst, B. Horn, and L. Li Bone marrow transplant for T-B- severe combined immunodeficiency disease in Athabascan-speaking native Americans Bone Marrow Transplant 27 2001 703 709
2. B. Neven, S. Leroy, H. Decaluwe, F. Le Deist, C. Picard, and D. Moshous Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency Blood 113 2009 4114 4124
3. M.D. Railey, Y. Lokhnygina, and R. Buckley Long-term clinical outcome of patients with severe combined immunodeficiency who received related donor bone marrow transplants without pretransplant chemotherapy or post-transplant GVHD prophylaxis J Pediatr 155 2009 834 840
4. C. Dvorak, G.-Y. Hung, B. Horn, E. Dunn, C.-Y. Oon, and M. Cowan Megadose CD34+ cell grafts improve recovery of T cell engraftment but not B cell immunity in patients with severe combined immunodeficiency disease undergoing haploidentical nonmyeloablative transplantation Biol Blood Marrow Transplant 14 2008 1125 1133
5. P. Titman, E. Pink, E. Skucek, K. O'Hanlon, T. Cole, and J. Gaspar Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies Blood 112 2008 3907 3913
6. M. Honig, M. Albert, A. Schulz, M. Sparber-Sauer, C. Schutz, and B. Belohradsky Patients with adenosine deaminase deficiency surviving after hematopoietic stem cell transplantation are at high risk of CNS complications Blood 109 2007 3595 3602
7. A. Hassan, C. Booth, A. Brightwell, Z. Allwood, P. Veys, and K. Rao outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency Blood 120 2012 3615 3624
8. S. Hacein-Bay-Abina, J. Hauer, A. Lim, C. Picard, G. Wang, and C. Berry Efficacy of gene therapy for X-linked severe combined immunodeficiency N Engl J Med 363 2010 355 364
9. M. Honig, C. Schuetz, K. Schwarz, M. Rojewski, E. Jacobsen, and G. Lahr Immunological reconstitution in a patient with ZAP-70 deficiency following transfusion of blood lymphocytes from previously transplanted sibling without conditioning Bone Marrow Transplant 47 2012 305 307
10. Y. Bertrand, S.M. Muller, J.L. Casanova, G. Morgan, A. Fischer, and W. Friedrich Reticular dysgenesis: HLA non-identical bone marrow transplants in a series of 10 patients Bone Marrow Transplant 29 2002 759 762
11. W. Al-Herz, A. Bousfiha, J.L. Casanova, H. Chapel, M.E. Conley, and C. Cunningham-Rundles Primary immunodeficiency disease: an update on the classification from the International Union of Immunological Societies Expert Committee for Primary Immunofeficiency Front Immunol 2 2011 54
12. A. Gennery, M. Slatter, L. Grandin, P. Taupin, A. Cant, and P. Veys Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? J Allergy Clin Immunol 126 2010 602 610
13. R. Buckley, S. Schiff, R. Schiff, L. Markert, L. Williams, and J. Roberts Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency N Engl J Med 340 1999 508 516
14. N. Patel, J. Chinen, H. Rosenblatt, I. Hanson, B. Brown, and M. Paul Long-term outcomes of nonconditioned patients with severe combined immunodeficiency transplanted with HLA-identical or haploidentical bone marrow depleted of T-cells with anti-CD6 mAb J Allergy Clin Immunol 122 2008 1185 1193
15. J. Fernandes, V. Rocha, M. Labopin, B. Neven, D. Moshous, and R. Gennery Transplantation in patients with SCID: mismatched related stem cell or unrelated cord blood? Blood 119 2012 2949 2955
16. D. Patel, M. Gooding, R. Parrott, K. Curtis, B. Haynes, and R. Buckeley Thymic function after hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency N Engl J Med 342 2000 1325 1332
17. M. Sarzotti, D.D. Patel, X. Li, D.A. Ozaki, S. Cao, and S. Langdon T cell repertoire development in humans with SCID after nonablative allogeneic marrow transplantation J Immunol 342 2003 1325 1332
18. M. Sarzotti-Kelsoe, C. Win, R. Parrott, M. Cooney, B. Moser, and J. Roberts Thymic output, T-cell diversity, and T-cell function in long-term human SCID chimeras Blood 114 2009 1445 1453
19. M. Cavazzana-Calvo, F. Carlier, F. LeDeist, E. Morillon, P. Taupin, and D. Gautier Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell-immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype Blood 109 2007 4575 4581
20. R. Buckley B-cell function in severe combined immunodeficiency after stem cell or gene therapy J Allergy Clin Immunol 125 2010 790 797
21. M. Eapen, K. Woo Ahn, P. Orchard, M. Cowan, S. Davies, and A. Fasth Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism Biol Blood Marrow Transplant 18 2012 1438 1445
22. N. Kamani, S. Kumar, A. Hassebroek, M. Eapen, J. LeRademacher, and J. Casper Malignancies after hematopoietic cell transplantation for primary immune deficiencies: a report from the Center for International Blood and Marrow Transplant Research Biol Blood Marrow Transplant 17 2011 1783 1789