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 - Tập 126 - Trang 602-610.e11 - 2010
Andrew R. Gennery1,2, Mary A. Slatter1,2, Laure Grandin3, Pierre Taupin3, Andrew J. Cant2, Paul Veys4, Persis J. Amrolia4, H. Bobby Gaspar4, E. Graham Davies4, Wilhelm Friedrich5, Manfred Hoenig5, Luigi D. Notarangelo6, Evelina Mazzolari6, Fulvio Porta6, Robbert G.M. Bredius7, Arjen C. Lankester7, Nico M. Wulffraat8, Reinhard Seger9, Tayfun Güngör9, Anders Fasth10
1Institute of Cellular Medicine, Child Health, University of Newcastle upon Tyne, Newcastle Upon Tyne, United Kingdom
2Department of Paediatric Immunology, Newcastle Upon Tyne Hospitals Foundation Trust, Newcastle Upon Tyne, United Kingdom
3Service de Biostatistique, EA 4067, Faculty of Medicine, Assistance Publique—Hôpitaux de Paris, Hôpital Necker Enfants Malades, Paris Descartes University, Paris, France
4Department of Bone Marrow Transplantation, Great Ormond Street Children's Hospital, London, United Kingdom
5Universitaet Ulm, Ulm, Germany
6Universitá degli Studi di Brescia Spedali Civili, P.le Spedali Civili, Brescia, Italy
7Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands
8Department of Pediatrics, Section of Immunology, University Medical Center, Utrecht, The Netherlands
9Division of Immunology/Hematology/Oncology, University Children's Hospital, Zurich, Switzerland
10Pediatric Immunology and Infectious Diseases Unit, Department of Pediatrics, Göteborg University, Göteborg, Sweden

Tài liệu tham khảo

Fischer, 2007, Human primary immunodeficiency diseases, Immunity, 27, 835, 10.1016/j.immuni.2007.11.012 Fischer, 2005, Severe combined immunodeficiency: a model disease for molecular immunology and therapy, Immunol Rev, 203, 98, 10.1111/j.0105-2896.2005.00223.x Levy, 1997, Clinical spectrum of X-linked hyper-IgM syndrome, J Pediatr, 131, 47, 10.1016/S0022-3476(97)70123-9 Imai, 2004, Clinical course of patients with WASP gene mutations, Blood, 103, 456, 10.1182/blood-2003-05-1480 Jones, 2008, Special article: chronic granulomatous disease in the United Kingdom and Ireland: a comprehensive national patient-based registry, Clin Exp Immunol, 152, 211, 10.1111/j.1365-2249.2008.03644.x van den Berg, 2009, Chronic granulomatous disease: the European experience, PLoS One, 4, e5234, 10.1371/journal.pone.0005234 Antoine, 2003, 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, 553, 10.1016/S0140-6736(03)12513-5 Slatter, 2006, Umbilical cord stem cell transplantation for primary immunodeficiencies, Expert Opin Biol Ther, 6, 555, 10.1517/14712598.6.6.555 Slatter, 2008, 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, 361, 10.1016/j.jaci.2007.10.035 Rao, 2005, Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen, Blood, 105, 879, 10.1182/blood-2004-03-0960 Lion, 2003, Molecular monitoring of adenovirus in peripheral blood after allogeneic bone marrow transplantation permits early diagnosis of disseminated disease, Blood, 102, 1114, 10.1182/blood-2002-07-2152 Notarangelo, 2009, Primary immunodeficiencies: 2009 update, J Allergy Clin Immunol, 124, 1161, 10.1016/j.jaci.2009.10.013 Kane, 2001, Neonatal bone marrow transplantation for severe combined immunodeficiency, Arch Dis Child Fetal Neonatal Ed, 85, F110, 10.1136/fn.85.2.F110 Myers, 2002, Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival, Blood, 99, 872, 10.1182/blood.V99.3.872 Baker, 2009, Development of a routine newborn screening protocol for severe combined immunodeficiency, J Allergy Clin Immunol, 124, 522, 10.1016/j.jaci.2009.04.007 Winkelstein, 2003, The X-linked hyper-IgM syndrome: clinical and immunologic features of 79 patients, Medicine (Baltimore), 82, 373, 10.1097/01.md.0000100046.06009.b0 Gennery, 2004, Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002, Blood, 103, 1152, 10.1182/blood-2003-06-2014 Fernandes, 2009, Comparison of outcomes of mismatched related stem cell and unrelated cord blood transplants in children with severe T-cell deficiencies, Bone Marrow Transplant, 43, S76 Grunebaum, 2006, Bone marrow transplantation for severe combined immune deficiency, JAMA, 295, 508, 10.1001/jama.295.5.508 Neven, 2009, Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency, Blood, 113, 4114, 10.1182/blood-2008-09-177923 Prockop, 2004, Regulation of thymus size by competition for stromal niches among early T cell progenitors, J Immunol, 173, 1604, 10.4049/jimmunol.173.3.1604 Garrod, 2010, NK cell patrolling and elimination of donor-derived dendritic cells favor indirect alloreactivity, J Immunol, 184, 2329, 10.4049/jimmunol.0902748 Fischer, 2005, Severe combined immunodeficiency: a model disease for molecular immunology and therapy, Immunol Rev, 203, 98, 10.1111/j.0105-2896.2005.00223.x Buckley, 1999, Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency, N Engl J Med, 340, 508, 10.1056/NEJM199902183400703 White, 2000, Intrinsic defects of B cell function in X-linked severe combined immunodeficiency, Eur J Immunol, 30, 732, 10.1002/1521-4141(200003)30:3<732::AID-IMMU732>3.0.CO;2-L Sarzotti-Kelsoe, 2009, Thymic output, T cell diversity and T cell function in long-term human SCID chimeras, Blood, 114, 1445, 10.1182/blood-2009-01-199323 Borghans, 2006, Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency, Blood, 108, 763, 10.1182/blood-2006-01-009241 Cavazzana-Calvo, 2007, 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, 4575, 10.1182/blood-2006-07-029090 Mazzolari, 2007, Long-term immune reconstitution and clinical outcome after stem cell transplantation for severe T-cell immunodeficiency, J Allergy Clin Immunol, 120, 892, 10.1016/j.jaci.2007.08.007 Pai, 2006, Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation, Bone Marrow Transplant, 38, 671, 10.1038/sj.bmt.1705512 Filipovich, 2001, 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, 1598, 10.1182/blood.V97.6.1598 Seger, 2002, Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985-2000, Blood, 100, 4344, 10.1182/blood-2002-02-0583 Gomez, 1995, Treatment of Omenn syndrome by bone marrow transplantation, J Pediatr, 127, 76, 10.1016/S0022-3476(95)70260-1 Renella, 2006, Br J Haematol, 134, 510, 10.1111/j.1365-2141.2006.06213.x Ozsahin, 2008, 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, 439, 10.1182/blood-2007-03-076679 Laffort, 2004, 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, 2051, 10.1016/S0140-6736(04)16457-X Rogers, 2001, Cognitive and behavioral abnormalities in adenosine deaminase deficient severe combined immunodeficiency, J Pediatr, 139, 44, 10.1067/mpd.2001.115023 Hönig, 2007, Patients with adenosine deaminase deficiency surviving after hematopoietic stem cell transplantation are at high risk of CNS complications, Blood, 109, 3595, 10.1182/blood-2006-07-034678 Griffith, 2008, Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs, J Allergy Clin Immunol, 122, 1087, 10.1016/j.jaci.2008.09.045 Griffith, 2009, Workshop Participants. Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management, J Allergy Clin Immunol, 124, 1152, 10.1016/j.jaci.2009.10.022