Advances in hematopoietic stem cell transplantation for primary immunodeficiency

被引:13
作者
Gennery, Andrew R. [1 ,2 ]
Cant, Andrew J. [1 ,2 ]
机构
[1] Newcastle Gen Hosp, Childrens Bone Marrow Transplant Unit, Newcastle Upon Tyne NE4 6BE, Tyne & Wear, England
[2] Univ Newcastle Upon Tyne, Inst Cellular Med, Newcastle Upon Tyne NE1 7RU, Tyne & Wear, England
关键词
D O I
10.1016/j.iac.2008.01.006
中图分类号
R392 [医学免疫学];
学科分类号
100102 ;
摘要
The molecular bases of most primary immunodeficiencies (PID) have been discovered. Long-term follow-up of patient cohorts treated with antimicrobial prophylaxis has demonstrated good short-term prognosis but with increasing morbidity and mortality over time. The results of hematopoietic stem cell transplantation (HSCT) for PID have improved incrementally over time, with survival and cure of 90% for some defined diseases. This article examines the advances in HSCT for PID and argues that HSCT should be considered earlier for most patients.
引用
收藏
页码:439 / +
页数:20
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共 74 条
[1]   Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy [J].
Aiuti, Alessandro ;
Cassani, Barbara ;
Andolfi, Grazia ;
Mirolo, Massimiliano ;
Biasco, Luca ;
Recchia, Alessandra ;
Urbinati, Fabrizia ;
Valacca, Cristina ;
Scaramuzza, Samantha ;
Aker, Memet ;
Slavin, Shimon ;
Cazzola, Matteo ;
Sartori, Daniela ;
Ambrosi, Alessandro ;
Di Serio, Clelia ;
Roncarolo, Maria Grazia ;
Mavilio, Fulvio ;
Bordignon, Claudio .
JOURNAL OF CLINICAL INVESTIGATION, 2007, 117 (08) :2233-2240
[2]   Adoptive immunotherapy with allodepleted donor T-cells improves immune reconstitution after haploidentical stem cell transplantation [J].
Amrolia, Persis J. ;
Muccioli-Casadei, Giada ;
Huls, Helen ;
Adams, Stuart ;
Durett, April ;
Gee, Adrian ;
Yvon, Eric ;
Weiss, Heidi ;
Cobbold, Mark ;
Gaspar, H. Bobby ;
Rooney, Cliona ;
Kuehnle, Ingrid ;
Ghetie, Victor ;
Schindler, John ;
Krance, Robert ;
Heslop, Helen E. ;
Veys, Paul ;
Vitetta, Ellen ;
Brenner, Malcolm K. .
BLOOD, 2006, 108 (06) :1797-1808
[3]   Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies:: report of the European experience 1968-99 [J].
Antoine, C ;
Müller, S ;
Cant, A ;
Cavazzana-Calvo, M ;
Veys, P ;
Vossen, J ;
Fasth, A ;
Heilmann, C ;
Wulffraat, N ;
Seger, R ;
Blanche, S ;
Friedrich, W ;
Abinun, M ;
Davies, G ;
Bredius, R ;
Schulz, A ;
Landais, P ;
Fischer, A .
LANCET, 2003, 361 (9357) :553-560
[4]   Cotransplantation of ex vivo-expanded mesenchymal stem cells accelerates lymphocyte recovery and may reduce the risk of graft failure in haploidentical hematopoietic stem-cell transplantation [J].
Ball, Lynne M. ;
Bernardo, Maria Ester ;
Roelofs, Helene ;
Lankester, Arjan ;
Cometa, Angela ;
Egeler, R. Maarten ;
Locatelli, Franco ;
Fibbe, Willem E. .
BLOOD, 2007, 110 (07) :2764-2767
[5]   Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency [J].
Bhattacharya, A ;
Slatter, MA ;
Chapman, CE ;
Barge, D ;
Jackson, A ;
Flood, TJ ;
Abinun, M ;
Cant, AJ ;
Gennery, AR .
BONE MARROW TRANSPLANTATION, 2005, 36 (04) :295-299
[6]   Extracorporeal photochemotherapy is accompanied by increasing levels of circulating CD4+CD25+GITR+Foxp3+CD62L+functional regulatory T-cells in patients with graft-versus-host disease [J].
Biagi, Ettore ;
Di Biaso, Iolanda ;
Leoni, Veronica ;
Gaipa, Giuseppe ;
Rossi, Vincenzo ;
Bugarin, Cristina ;
Renoldi, Giuliano ;
Parma, Matteo ;
Balduzzi, Adriana ;
Perseghin, Paolo ;
Biondi, Andrea .
TRANSPLANTATION, 2007, 84 (01) :31-39
[7]   CD34+ stem cell top-ups without conditioning after initial haematopoietic stem cell transplantation for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies [J].
Booth, Claire ;
Ribeil, Jean-Antoine ;
Audat, Francoise ;
Dal-Cortivo, Liliane ;
Veys, Paul A. ;
Thrasher, Adrian J. ;
Davies, E. Graham ;
Lefrere, Francois ;
Fischer, Alain ;
Cavazzana-Calvo, Marina ;
Gaspar, H. Bobby .
BRITISH JOURNAL OF HAEMATOLOGY, 2006, 135 (04) :533-537
[8]   Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency [J].
Borghans, Jose A. ;
Bredius, Robbert G. ;
Hazenberg, Mette D. ;
Roelofs, Helene ;
Zijde, Els C. Jol-van der ;
Heidt, Jeroen ;
Otto, Sigrid A. ;
Kuijpers, Taco W. ;
Fibbe, Willem E. ;
Vossen, Jaak M. ;
Miedema, Frank ;
van Tol, Maarten J. .
BLOOD, 2006, 108 (02) :763-769
[9]   Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency [J].
Buckley, RH ;
Schiff, SE ;
Schiff, RI ;
Markert, ML ;
Williams, LW ;
Roberts, JL ;
Myers, LA ;
Ward, FE .
NEW ENGLAND JOURNAL OF MEDICINE, 1999, 340 (07) :508-516
[10]   Follow up of patients with chronic granulomatous disease diagnosed since 1990 [J].
Cale, CM ;
Jones, AM ;
Goldblatt, D .
CLINICAL AND EXPERIMENTAL IMMUNOLOGY, 2000, 120 (02) :351-355