Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?

Journal of Allergy and Clinical Immunology

Volumn 126, Issue 3, 2010, Pages

  Gennery, Andrew R ^a,b   Slatter, Mary A ^a,b   Grandin, Laure ^c   Taupin, Pierre ^c   Cant, Andrew J ^b   Veys, Paul ^d   Amrolia, Persis J ^d   Gaspar, H Bobby ^d   Davies, E Graham ^d   Friedrich, Wilhelm ^e   Hoenig, Manfred ^e   Notarangelo, Luigi D ^f   Mazzolari, Evelina ^f   Porta, Fulvio ^f   Bredius, Robbert G M ^g   Lankester, Arjen C ^g   Wulffraat, Nico M ^h   Seger, Reinhard ⁱ   Güngör, Tayfun ⁱ   Fasth, Anders ^j   Sedlacek, Petr ^k   Neven, Benedicte ^c   Blanche, Stephane ^c   Fischer, Alain ^c   Cavazzana Calvo, Marina ^c   Landais, Paul ^c   more..

^a NEWCASTLE UNIVERSITY   (United Kingdom)

^b NEWCASTLE UPON TYNE HOSPITALS NHS FOUNDATION TRUST   (United Kingdom)

^c UNIVERSITÉ PARIS DESCARTES   (France)

^d GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^e UNIVERSITY OF ULM   (Germany)

^f UNIVERSITY OF BRESCIA   (Italy)

^g LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^h UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

ⁱ UNIVERSITY CHILDREN S HOSPITAL   (Switzerland)

^j UNIVERSITY OF GOTHENBURG   (Sweden)

^k UNIVERSITY HOSPITAL MOTOL   (Czech Republic)

more..

Author keywords

CD40 ligand deficiency; chronic granulomatous disease; hematopoietic stem cell transplantation; Primary immunodeficiency; severe combined immunodeficiency; Wiskott Aldrich syndrome

Indexed keywords

ADVERSE OUTCOME; AGE; ANTIBIOTIC THERAPY; ARTICLE; B LYMPHOCYTE; BIOCOMPATIBILITY; CHEMOTHERAPY; CHILD; CLINICAL EVALUATION; ENVIRONMENTAL FACTOR; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOCOMPATIBILITY; HLA MATCHING; HUMAN; INFANT; LIVER DYSFUNCTION; MAJOR CLINICAL STUDY; MALE; MALNUTRITION; MENINGITIS; OMENN SYNDROME; ORGAN DONOR; OVERALL SURVIVAL; PHENOTYPE; PRESCHOOL CHILD; PRIORITY JOURNAL; PROGNOSIS; PURINE NUCLEOSIDE PHOSPHORYLASE DEFICIENCY; RESPIRATORY FAILURE; SCHOOL CHILD; SEPTICEMIA; SEVERE COMBINED IMMUNODEFICIENCY; SIBLING RELATION; T LYMPHOCYTE; TREATMENT OUTCOME; VIRUS INFECTION; WISKOTT ALDRICH SYNDROME; EUROPE; FOLLOW UP; HISTORY; MULTIVARIATE ANALYSIS; STANDARDS; SURVIVAL RATE; TIME; TRENDS;

CHILD; CHILD, PRESCHOOL; EUROPE; FOLLOW-UP STUDIES; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTORY, 20TH CENTURY; HISTORY, 21ST CENTURY; HUMANS; MULTIVARIATE ANALYSIS; PROGNOSIS; SEVERE COMBINED IMMUNODEFICIENCY; SURVIVAL RATE; TIME FACTORS; TREATMENT OUTCOME;

EID: 77956395504     PISSN: 00916749     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jaci.2010.06.015     Document Type: Article

References (40)

1. A. Fischer Human primary immunodeficiency diseases Immunity 27 2007 835 845
2. A. Fischer, F. Le Deist, S. Hacein-Bey-Abina, I. André-Schmutz, S. Basile Gde, and J.P. de Villartay Severe combined immunodeficiency: a model disease for molecular immunology and therapy Immunol Rev 203 2005 98 109
3. J. Levy, T. Espanol-Boren, C. Thomas, A. Fischer, P. Tovo, and P. Bordigoni Clinical spectrum of X-linked hyper-IgM syndrome J Pediatr 131 1997 47 54
4. K. Imai, T. Morio, Y. Zhu, Y. Jin, S. Itoh, and M. Kajiwara Clinical course of patients with WASP gene mutations Blood 103 2004 456 464
5. L.B. Jones, P. McGrogan, T.J. Flood, A.R. Gennery, L. Morton, and A. Thrasher Special article: chronic granulomatous disease in the United Kingdom and Ireland: a comprehensive national patient-based registry Clin Exp Immunol 152 2008 211 218
6. J.M. van den Berg, E. van Koppen, A. Ahlin, B.H. Belohradsky, E. Bernatowska, and L. Corbeel Chronic granulomatous disease: the European experience PLoS One 4 2009 e5234
7. C. Antoine, S. Müller, A. Cant, M. Cavazzana-Calvo, P. Veys, and J. Vossen European Group for Blood and Marrow Transplantation, European Society for Immunodeficiency. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99 Lancet 361 2003 553 560
8. M.A. Slatter, and A.R. Gennery Umbilical cord stem cell transplantation for primary immunodeficiencies Expert Opin Biol Ther 6 2006 555 565
9. 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
10. K. Rao, P.J. Amrolia, A. Jones, C.M. Cale, P. Naik, and D. King Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen Blood 105 2005 879 885
11. T. Lion, R. Baumgartinger, F. Watzinger, S. Matthes-Martin, M. Suda, and S. Preuner Molecular monitoring of adenovirus in peripheral blood after allogeneic bone marrow transplantation permits early diagnosis of disseminated disease Blood 102 2003 1114 1120
12. L.D. Notarangelo, A. Fischer, R.S. Geha, J.L. Casanova, H. Chapel, M.E. Conley International Union of Immunological Societies Expert Committee on Primary Immunodeficiencies Primary immunodeficiencies: 2009 update J Allergy Clin Immunol 124 2009 1161 1178
13. L. Kane, A.R. Gennery, B.N. Crooks, T.J. Flood, M. Abinun, and A.J. Cant Neonatal bone marrow transplantation for severe combined immunodeficiency Arch Dis Child Fetal Neonatal Ed 85 2001 F110 F113
14. L.A. Myers, D.D. Patel, J.M. Puck, and R.H. Buckley Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival Blood 99 2002 872 878
15. M.W. Baker, W.J. Grossman, R.H. Laessig, G.L. Hoffman, C.D. Brokopp, and D.F. Kurtycz Development of a routine newborn screening protocol for severe combined immunodeficiency J Allergy Clin Immunol 124 2009 522 527
16. J.A. Winkelstein, M.C. Marino, H. Ochs, R. Fuleihan, P.R. Scholl, and R. Geha The X-linked hyper-IgM syndrome: clinical and immunologic features of 79 patients Medicine (Baltimore) 82 2003 373 384
17. A.R. Gennery, K. Khawaja, P. Veys, R.G. Bredius, L.D. Notarangelo, and E. Mazzolari Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002 Blood 103 2004 1152 1157
18. J.F. Fernandes, V. Rocha, P. Landais, B. Neven, A. Gennery, P. Veys for Eurocord and Inborn Errors Working Party of EBMT Comparison of outcomes of mismatched related stem cell and unrelated cord blood transplants in children with severe T-cell deficiencies Bone Marrow Transplant 43 suppl 1 2009 S76
19. 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
20. 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
21. S.E. Prockop, and H.T. Petrie Regulation of thymus size by competition for stromal niches among early T cell progenitors J Immunol 173 2004 1604 1611
22. K.R. Garrod, F.C. Liu, L.E. Forrest, I. Parker, S.M. Kang, and M.D. Cahalan NK cell patrolling and elimination of donor-derived dendritic cells favor indirect alloreactivity J Immunol 184 2010 2329 2336
23. A. Fischer, F. Le Deist, S. Hacein-Bey-Abina, I. Andre-Schmutz, S. Basile Gde, and J.P. de Villartay Severe combined immunodeficiency: a model disease for molecular immunology and therapy Immunol Rev 203 2005 98 109
24. 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
25. H. White, A. Thrasher, P. Veys, C. Kinnon, and H.B. Gaspar Intrinsic defects of B cell function in X-linked severe combined immunodeficiency Eur J Immunol 30 2000 732 737
26. M. Sarzotti-Kelsoe, C.M. Win, R.E. Parrott, M. Cooney, B.K. Moser, and J.L. Roberts Thymic output, T cell diversity and T cell function in long-term human SCID chimeras Blood 114 2009 1445 1453
27. J.A. Borghans, R.G. Bredius, M.D. Hazenberg, H. Roelofs, E.C. Jol-van der Zijde, and J. Heidt Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency Blood 108 2006 763 769
28. 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
29. E. Mazzolari, C. Forino, S. Guerci, L. Imberti, A. Lanfranchi, and F. Porta Long-term immune reconstitution and clinical outcome after stem cell transplantation for severe T-cell immunodeficiency J Allergy Clin Immunol 120 2007 892 899
30. S.Y. Pai, D. DeMartiis, C. Forino, S. Cavagnini, A. Lanfranchi, and S. Giliani Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation Bone Marrow Transplant 38 2006 671 679
31. A.H. Filipovich, J.V. Stone, S.C. Tomany, M. Ireland, C. Kollman, and C.J. Pelz Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program Blood 97 2001 1598 1603
32. R.A. Seger, T. Gungor, B.H. Belohradsky, S. Blanche, P. Bordigoni, and P. Di Bartolomeo Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000 Blood 100 2002 4344 4350
33. L. Gomez, F. Le Deist, S. Blanche, M. Cavazzana-Calvo, C. Griscelli, and A. Fischer Treatment of Omenn syndrome by bone marrow transplantation J Pediatr 127 1995 76 81
34. R. Renella, C. Picard, B. Neven, M. Ouachée-Chardin, J.L. Casanova, and F. Le Deist Human leucocyte antigen-identical hematopoietic stem cell transplantation in major histocompatibility complex class II immunodeficiency: reduced survival correlates with an increased incidence of acute graft-versus-host disease and pre-existing viral infections Br J Haematol 134 2006 510 516
35. H. Ozsahin, M. Cavazzana-Calvo, L.D. Notarangelo, A. Schulz, A.J. Thrasher, and E. Mazzolari Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation Blood 111 2008 439 445
36. C. Laffort, F. Le Deist, M. Favre, S. Caillat-Zucman, I. Radford-Weiss, and M. Debré Severe cutaneous papillomavirus disease after haemopoietic stem-cell transplantation in patients with severe combined immune deficiency caused by common gammac cytokine receptor subunit or JAK-3 deficiency Lancet 363 2004 2051 2054
37. M.H. Rogers, R. Lwin, L. Fairbanks, B. Gerritsen, and H.B. Gaspar Cognitive and behavioral abnormalities in adenosine deaminase deficient severe combined immunodeficiency J Pediatr 139 2001 44 50
38. M. Hönig, M.H. Albert, A. Schulz, M. Sparber-Sauer, C. Schütz, 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
39. L.M. Griffith, M.J. Cowan, D.B. Kohn, L.D. Notarangelo, J.M. Puck, and K.R. Schultz Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs J Allergy Clin Immunol 122 2008 1087 1096
40. L.M. Griffith, M.J. Cowan, L.D. Notarangelo, J.M. Puck, R.H. Buckley, and F. Candotti Workshop Participants. Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management J Allergy Clin Immunol 124 2009 1152 1160 e12