Reduced Intensity Conditioning and Hematopoietic Stem Cell Transplantation in Pediatric Nonmalignant Disease: A New Therapeutic Paradigm

被引：4
作者：
Abikoff, Cori M. [1 ]
Cairo, Mitchell S. [1 ,2 ,3 ,4 ,5 ]
机构：
[1] New York Med Coll, Dept Pediat, Valhalla, NY 10595 USA
[2] New York Med Coll, Dept Med, Valhalla, NY 10595 USA
[3] New York Med Coll, Dept Pathol, Valhalla, NY 10595 USA
[4] New York Med Coll, Dept Microbiol & Immunol, Valhalla, NY 10595 USA
[5] New York Med Coll, Dept Cell Biol & Anat, Valhalla, NY 10595 USA
来源：
JOURNAL OF PEDIATRICS | 2014年 / 164卷 / 05期
关键词：
BONE-MARROW-TRANSPLANTATION; SEVERE COMBINED IMMUNODEFICIENCY; 1ST INTERNATIONAL-CONFERENCE; GRAFT FAILURE; GENE-THERAPY; HOST-DISEASE; CHILDREN; TOXICITY; OUTCOMES; BLOOD;
D O I：
10.1016/j.jpeds.2014.01.061
中图分类号：
R72 [儿科学];
学科分类号：
100202 ;
摘要：
引用
收藏
页码：952 / +
页数：4
相关论文
共 28 条
[1]   Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome [J].
Aiuti, Alessandro ;
Biasco, Luca ;
Scaramuzza, Samantha ;
Ferrua, Francesca ;
Cicalese, Maria Pia ;
Baricordi, Cristina ;
Dionisio, Francesca ;
Calabria, Andrea ;
Giannelli, Stefania ;
Castiello, Maria Carmina ;
Bosticardo, Marita ;
Evangelio, Costanza ;
Assanelli, Andrea ;
Casiraghi, Miriam ;
Di Nunzio, Sara ;
Callegaro, Luciano ;
Benati, Claudia ;
Rizzardi, Paolo ;
Pellin, Danilo ;
Di Serio, Clelia ;
Schmidt, Manfred ;
Von Kalle, Christof ;
Gardner, Jason ;
Mehta, Nalini ;
Neduva, Victor ;
Dow, David J. ;
Galy, Anne ;
Miniero, Roberto ;
Finocchi, Andrea ;
Metin, Ayse ;
Banerjee, Pinaki P. ;
Orange, Jordan S. ;
Galimberti, Stefania ;
Valsecchi, Maria Grazia ;
Biffi, Alessandra ;
Montini, Eugenio ;
Villa, Anna ;
Ciceri, Fabio ;
Roncarolo, Maria Grazia ;
Naldini, Luigi .
SCIENCE, 2013, 341 (6148) :865-U71
[2]   Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency. [J].
Aiuti, Alessandro ;
Cattaneo, Federica ;
Galimberti, Stefania ;
Benninghoff, Ulrike ;
Cassani, Barbara ;
Callegaro, Luciano ;
Scaramuzza, Samantha ;
Andolfi, Grazia ;
Mirolo, Massimiliano ;
Brigida, Immacolata ;
Tabucchi, Antonella ;
Carlucci, Filippo ;
Eibl, Martha ;
Aker, Memet ;
Slavin, Shimon ;
Al-Mousa, Hamoud ;
Al Ghonaium, Abdulaziz ;
Ferster, Alina ;
Duppenthaler, Andrea ;
Notarangelo, Luigi ;
Wintergerst, Uwe ;
Buckley, Rebecca H. ;
Bregni, Marco ;
Marktel, Sarah ;
Valsecchi, Maria Grazia ;
Rossi, Paolo ;
Ciceri, Fabio ;
Miniero, Roberto ;
Bordignon, Claudio ;
Roncarolo, Maria-Grazia .
NEW ENGLAND JOURNAL OF MEDICINE, 2009, 360 (05) :447-458
[3]   The Burden of Cure: Long-term Side Effects Following Hematopoietic Stem Cell Transplantation (HSCT) in Children [J].
Baker, K. Scott ;
Bresters, Dorine ;
Sande, Jane E. .
PEDIATRIC CLINICS OF NORTH AMERICA, 2010, 57 (01) :323-+
[4]   Gene therapy for leukodystrophies [J].
Biffi, Alessandra ;
Aubourg, Patrick ;
Cartier, Nathalie .
HUMAN MOLECULAR GENETICS, 2011, 20 :R42-R53
[5]   Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure [J].
Boelens, J. J. ;
Wynn, R. F. ;
O'Meara, A. ;
Veys, P. ;
Bertrand, Y. ;
Souillet, G. ;
Wraith, J. E. ;
Fischer, A. ;
Cavazzana-Calvo, M. ;
Sykora, K. W. ;
Sedlacek, P. ;
Rovelli, A. ;
Uiterwaal, C. S. P. M. ;
Wulffraat, N. .
BONE MARROW TRANSPLANTATION, 2007, 40 (03) :225-233
[6]   Stem cell transplantation for pediatric lymphoma: past, present and future [J].
Bradley, M. B. ;
Cairo, M. S. .
BONE MARROW TRANSPLANTATION, 2008, 41 (02) :149-158
[7]   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
[8]   NCI, NHLBI/PBMTC First International Conference on Late Effects After Pediatric Hematopoietic Cell Transplantation: Persistent Immune Deficiency in Pediatric Transplant Survivors [J].
Bunin, Nancy ;
Small, Trudy ;
Szabolcs, Paul ;
Baker, K. Scott ;
Pulsipher, Michael A. ;
Torgerson, Troy .
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2012, 18 (01) :6-15
[9]   Pediatric blood and marrow transplantation: State of the science [J].
Cairo, M. S. ;
Heslop, H. .
BONE MARROW TRANSPLANTATION, 2008, 41 (02) :97-97
[10]   NCI, NHLBI/PBMTC First International Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: Endocrine Challenges-Thyroid Dysfunction, Growth Impairment, Bone Health, & Reproductive Risks [J].
Dvorak, Christopher C. ;
Gracia, Clarisa R. ;
Sanders, Jean E. ;
Cheng, Edward Y. ;
Baker, K. Scott ;
Pulsipher, Michael A. ;
Petryk, Anna .
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2011, 17 (12) :1725-1738
123