Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency

Journal of Allergy and Clinical Immunology

Volumn 134, Issue 4, 2014, Pages 935-943.e15

  Dvorak, Christopher C ^a   Hassan, Amel ^b   Slatter, Mary A ^c   Hönig, Manfred ^d   Lankester, Arjan C ^e   Buckley, Rebecca H ^f   Pulsipher, Michael A ^g   Davis, Jeffrey H ^h   Güngör, Tayfun ⁱ   Gabriel, Melissa ^j   Bleesing, Jacob H ^k   Bunin, Nancy ^l   Sedlacek, Petr ^m   Connelly, James A ⁿ   Crawford, David F ^o   Notarangelo, Luigi D ^p   Pai, Sung Yun ^q   Hassid, Jake ^a   Veys, Paul ^b   Gennery, Andrew R ^c   Cowan, Morton J ^a   more..

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c NEWCASTLE UNIVERSITY   (United Kingdom)

^d UNIVERSITY HOSPITAL ULM   (Germany)

^e LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^f DUKE UNIVERSITY MEDICAL CENTER   (United States)

^g UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^h BC CHILDREN S HOSPITAL   (Canada)

ⁱ UNIVERSITY CHILDREN S HOSPITAL   (Switzerland)

^j CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

^k CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^l CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^m UNIVERSITY HOSPITAL MOTOL   (Czech Republic)

ⁿ UNIVERSITY OF MICHIGAN   (United States)

^o UNIVERSITY OF OKLAHOMA COLLEGE OF MEDICINE   (United States)

^p HARVARD MEDICAL SCHOOL   (United States)

^q BOSTON CHILDREN S HOSPITAL   (United States)

more..

Author keywords

conditioning; hematopoietic cell transplantation; serotherapy; Severe combined immunodeficiency; sibling donors; umbilical cord blood; unrelated donors

Indexed keywords

CD3 ANTIGEN; CD4 ANTIGEN; CD8 ANTIGEN; IMMUNOGLOBULIN A; IMMUNOGLOBULIN G; IMMUNOGLOBULIN M; INTERLEUKIN 2 RECEPTOR GAMMA; JANUS KINASE 3;

ADENOSINE DEAMINASE DEFICIENCY; ARTICLE; B LYMPHOCYTE; CHEMOTHERAPY; CHILD; CHIMERA; DONOR; EVENT FREE SURVIVAL; FAMILY HISTORY; FEMALE; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA TYPING; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INFANT; MAJOR CLINICAL STUDY; MALE; MATCHED SIBLING DONOR; NATURAL KILLER CELL; NEWBORN; OVERALL SURVIVAL; PERIPHERAL BLOOD STEM CELL; RECIPIENT; RETROSPECTIVE STUDY; SEVERE COMBINED IMMUNODEFICIENCY; T LYMPHOCYTE; UMBILICAL CORD BLOOD; UNRELATED DONORS; ADULT; AUSTRALIA; CLINICAL TRIAL; COMPARATIVE STUDY; EUROPE; GRAFT VS HOST DISEASE; HISTOCOMPATIBILITY; HISTOCOMPATIBILITY TEST; IMMUNOLOGY; MORTALITY; MULTICENTER STUDY; NORTH AMERICA; SIBLING; SURVIVAL; TREATMENT OUTCOME; VOLUNTEER;

ADULT; AUSTRALIA; B-LYMPHOCYTES; CHILD; CHIMERISM; EUROPE; FEMALE; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOCOMPATIBILITY; HISTOCOMPATIBILITY TESTING; HUMANS; INFANT; INFANT, NEWBORN; MALE; NORTH AMERICA; RETROSPECTIVE STUDIES; SEVERE COMBINED IMMUNODEFICIENCY; SIBLINGS; SURVIVAL ANALYSIS; T-LYMPHOCYTES; TRANSPLANTATION CONDITIONING; TREATMENT OUTCOME; UNRELATED DONORS; VOLUNTEERS;

EID: 84908123060     PISSN: 00916749     EISSN: 10976825     Source Type: Journal    
DOI: 10.1016/j.jaci.2014.06.021     Document Type: Article

References (37)

1. C. Dvorak, and M. Cowan Hematopoietic stem cell transplantation for primary immunodeficiency disease Bone Marrow Transplant 41 2008 119 126
2. 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
3. C. Dvorak, G. Hung, B. Horn, E. Dunn, C. 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 haplocompatible nonmyeloablative transplantation Biol Blood Marrow Transplant 14 2008 1125 1133
4. W.J. Murphy, V. Kumar, and M. Bennett Rejection of bone marrow allografts by mice with severe combined immune deficiency (SCID). Evidence that natural killer cells can mediate the specificity of marrow graft rejection J Exp Med 165 1987 1212 1217
5. Z. Xiao, E. Dunn, K. Singh, I. Khan, S. Yannone, and M. Cowan A non-leaky Artemis-deficient mouse that accurately models the human severe combined immune deficiency phenotype, including resistance to hematopoietic stem cell transplantation Biol Blood Marrow Transplant 15 2009 1 11
6. R.H. Buckley, S.E. Schiff, R.I. Schiff, L. Markert, L.W. Williams, and J.L. Roberts Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency N Engl J Med 340 1999 508 516
7. E. Grunebaum, E. Mazzolari, F. Porta, D. Dallera, A. Atkinson, and B. Reid Bone marrow transplantation for severe combined immune deficiency JAMA 295 2006 508 518
8. E. Haddad, S. Leroy, and R. Buckley B-cell reconstitution for SCID: should a conditioning regimen be used in SCID treatment? J Allergy Clin Immunol 131 2013 994 1000
9. J. Fernandes, V. Rocha, M. Labopin, B. Neven, D. Moshous, and A. Gennery Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood? Blood 119 2012 2949 2955
10. 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 e1-11
11. E. Haddad, P. Landais, W. Friedrich, B. Gerritsen, M. Cavazzana-Calvo, and G. Morgan 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 91 1998 3646 3653
12. M.A. Slatter, K. Brigham, A.M. Dickinson, H.L. Harvey, D. Barge, and A. Jackson Long-term immune reconstitution after anti-CD52-treated or anti-CD34-treated hematopoietic stem cell transplantation for severe T-lymphocyte immunodeficiency J Allergy Clin Immunol 121 2008 361 367
13. A. Bhattacharya, M. Slatter, C. Chapman, D. Barge, A. Jackson, and T. Flood Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency Bone marrow Transplant 36 2005 295 299
14. T. Taga, E. Itoh, Y. Noda, H. Kato, Y. Maruo, and T. Takano Successful unrelated umbilical cord blood cell transplantation without conditioning for a neonate with severe combined immunodeficiency Pediatr Transplant 15 2011 E152 E155
15. 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
16. A. Janda, P. Sedlacek, M. Hönig, W. Friedrich, M. Champagne, and T. Matsumoto Multicenter survey on the outcome of transplantation of hematopoietic cells in patients with the complete form of DiGeorge anomaly Blood 116 2010 2229 2236
17. A.S. O'Marcaigh, K. DeSantes, D. Hu, H. Pabst, B. Horn, and L. Li Bone marrow transplantation for T-B- severe combined immunodeficiency disease in Athabascan-speaking native Americans Bone Marrow Transplant 27 2001 703 709
18. J. Heimall, M. Keller, R. Saltzman, N. Bunin, D. McDonald-McGinn, and E. Zakai Diagnosis of 22q11.2 deletion syndrome and Artemis deficiency in two children with T-B-NK+ immunodeficiency J Clin Immunol 32 2012 1141 1144
19. L. Griffith, M. Cowan, L. Notarangelo, J. Puck, R. Buckley, and F. Candotti Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management J Allergy Clin Immunol 124 2009 1152 1160
20. W. Shearer, E. Dunn, L. Notarangelo, C. Dvorak, J. Puck, and B. Logan Diagnosing SCID, leaky SCID, and Omenn syndrome: the primary immune deficiency treatment consortium experience J Allergy Clin Immunol 133 2014 1092 1098
21. A. Bacigalupo Antilymphocyte/thymocyte globulin for graft versus host disease prophylaxis: efficacy and side effects Bone Marrow Transplant 35 2005 225 231
22. M. Cavazzana-Calvo, F. Carlier, F. Le Deist, 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
23. C. Dvorak, M. Cowan, B. Logan, L. Notarangelo, L. Griffith, and J. Puck The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the Primary Immune Deficiency Treatment Consortium prospective study 6901 J Clin Immunol 33 2013 1156 1164
24. C. Dvorak, A. Sandford, A. Fong, M. Cowan, T. George, and D. Lewis Maternal T-cell engraftment associated with severe hemophagocytosis of the bone marrow in untreated X-linked severe combined immunodeficiency J Pediatr Hematol Oncol 30 2008 396 400
25. H. Meuwissen, R. Gatti, P. Terasaki, R. Hong, and R. Good Treatment of lymphopenic hypogammaglobulinemia and bone-marrow aplasia by transplantation of allogeneic marrow. Crucial role of histocompatibility matching N Engl J Med 281 1969 691 697
26. P. Kottaridis, D. Milligan, R. Chopra, R. Chakraverty, S. Chakrabarti, and S. Robinson In vivo CAMPATH-1H prevents graft-versus-host disease following nonmyeloablative stem cell transplantation Blood 96 2000 2419 2425
27. R. Chiesa, K. Gilmour, W. Qasim, S. Adams, A. Worth, and H. Zhan Omission of in vivo T-cell depletion promotes rapid expansion of naïve CD4+ cord blood lymphocytes and restores adaptive immunity within 2 months after unrelated cord blood transplant Br J Haematol 156 2012 656 666
28. F. Baran-Marszak, J. Feuillard, I. Najjar, C. Le Clorennec, J. Béchet, and I. Dusanter-Fourt Differential roles of STAT1alpha and STAT1beta in fludarabine-induced cell cycle arrest and apoptosis in human B cells Blood 104 2004 2475 2483
29. K. Schultz, J. Paquet, S. Bader, and K. HayGlass Requirement for B cells in T cell priming to minor histocompatibility antigens and development of graft-versus-host disease Bone Marrow Transplant 16 1995 289 295
30. K. Larsson, J. Aschan, M. Remberger, O. Ringdén, J. Winiarski, and P. Ljungman Reduced risk for extensive chronic graft-versus-host disease in patients receiving transplants with human leukocyte antigen-identical sibling donors given polymerase chain reaction-based preemptive therapy against cytomegalovirus Transplantation 77 2004 526 531
31. C. Pichereau, K. Desseaux, A. Janin, C. Scieux, R. Peffault de Latour, and A. Xhaard The complex relationship between human herpesvirus 6 and acute graft-versus-host disease Biol Blood Marrow Transplant 18 2012 141 144
32. R. Buckley, C. Win, B. Moser, R. Parrott, E. Sajaroff, and M. Sarzotti-Kelsoe Post-transplantation B cell function in different molecular types of SCID J Clin Immunol 33 2013 96 110
33. A. Kwan, J. Church, M. Cowan, R. Agarwal, N. Kapoor, and D. Kohn Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years J Allergy Clin Immunol 132 2013 140 150
34. V.H.-D. Kim, L. Murguia, T. Schechter, E. Grunebaum, and C.M. Roifman Emergency treatment for ζ chain-associated protein of 70 kDa (ZAP70) deficiency J Allergy Clin Immunol 131 2013 1233 1235
35. K. Straathof, K. Rao, M. Eyrich, G. Hale, P. Bird, and E. Berrie Haemopoietic stem-cell transplantation with antibody-based minimal-intensity conditioning: a phase 1/2 study Lancet 374 2009 912 920
36. A. Czechowicz, D. Kraft, I. Weissman, and D. Bhattacharya Efficient transplantation via antibody-based clearance of hematopoietic stem cell niches Science 318 2007 1296 1299
37. R. Savic, M. Cowan, C. Dvorak, S.-Y. Pai, L. Pereira, and I. Bartelink Effect of weight and maturation on busulfan clearance in infants and small children undergoing hematopoietic cell transplantation Biol Blood Marrow Transplant 19 2013 1608 1614