Single centre experience of haematopoietic SCT for patients with immunodysregulation, polyendocrinopathy, enteropathy, X-linked syndrome

被引：27

作者：

Nademi, Z. ^{[1
,2
]}

Slatter, M. ^{[1
,2
]}

Gambineri, E. ^{[3
]}

Mannurita, S. C. ^{[3
]}

Barge, D. ^{[4
]}

Hodges, S. ^{[5
]}

Bunn, S. ^{[5
]}

Thomas, J. ^{[5
]}

Haugk, B. ^{[6
]}

Hambleton, S. ^{[1
,2
]}

Flood, T. ^{[1
]}

Cant, A. ^{[1
,2
]}

Abinun, M. ^{[1
,2
]}

Gennery, A. ^{[1
,2
]}

机构：

[1] Newcastle Upon Tyne Hosp Trust, Dept Paediat Immunol, Florence, Italy

[2] Newcastle Univ, Inst Cellular Med, Newcastle Upon Tyne NE1 7RU, Tyne & Wear, England

[3] Paediatr Bone Marrow Transplant Unit, Florence, Italy

[4] Newcastle Upon Tyne Hosp Trust, Immunol Lab, Newcastle Upon Tyne, Tyne & Wear, England

[5] Newcastle Upon Tyne Hosp Trust, Dept Paediat Gastroenterol, Newcastle Upon Tyne, Tyne & Wear, England

[6] Newcastle Upon Tyne Hosp Trust, Dept Cellular Pathol, Newcastle Upon Tyne, Tyne & Wear, England

来源：

BONE MARROW TRANSPLANTATION | 2014年 / 49卷 / 02期

关键词：

IMMUNE DYSREGULATION; IPEX SYNDROME; T-CELLS; TRANSPLANTATION; ENGRAFTMENT;

D O I：

10.1038/bmt.2013.181

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

引用

页码：310 / 312

页数：3

共 10 条

[1] Defective regulatory and effector T cell functions in patients with FOXP3 mutations [J].

Bacchetta, Rosa ;

Passerini, Laura ;

Gambineri, Eleonora ;

Dai, Minyue ;

Allan, Sarah E. ;

Perroni, Lucia ;

Dagna-Bricarelli, Franca ;

Sartirana, Claudia ;

Matthes-Martins, Susanne ;

Lawitschka, Anita ;

Azzari, Chiara ;

Ziegler, Steven F. ;

Levings, Megan K. ;

Roncarolo, Maria Grazia .

JOURNAL OF CLINICAL INVESTIGATION, 2006, 116 (06) :1713-1722

[2] Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome: a paradigm of immunodeficiency with autoimmunity [J].

Barzaghi, Federica ;

Passerini, Laura ;

Bacchetta, Rosa .

FRONTIERS IN IMMUNOLOGY, 2012, 3

[3] Stable hematopoietic cell engraftment after low-intensity nonmyeloablative conditioning in patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome [J].

Burroughs, Lauri M. ;

Torgerson, Troy R. ;

Storb, Rainer ;

Carpenter, Paul A. ;

Rawlings, David J. ;

Sanders, Jean ;

Scharenberg, Andrew M. ;

Skoda-Smith, Suzanne ;

Englund, Janet ;

Ochs, Hans D. ;

Woolfrey, Ann E. .

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2010, 126 (05) :1000-1005

[4]

Gambineri Eleonora, 2008, J Allergy Clin Immunol, V122, P1105, DOI 10.1016/j.jaci.2008.09.027

[5] Submyeloablative cord blood transplantation corrects clinical defects seen in IPEX syndrome [J].

Lucas, K. G. ;

Ungar, D. ;

Comito, M. ;

Bayerl, M. ;

Groh, B. .

BONE MARROW TRANSPLANTATION, 2007, 39 (01) :55-56

[6] Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning [J].

Rao, Aarati ;

Kamani, Naynesh ;

Filipovich, Alexandra ;

Lee, Susan Molleran ;

Davies, Stella M. ;

Dalal, Jignesh ;

Shenoy, Shalini .

BLOOD, 2007, 109 (01) :383-385

[7] Selective engraftment of donor CD4+25high FOXP3-positive T cells in IPEX syndrome after nonmyeloablative hematopoietic stem cell transplantation [J].

Seidel, Markus G. ;

Fritsch, Gerhard ;

Lion, Thomas ;

Juergens, Birgit ;

Heitger, Andreas ;

Bacchetta, Rosa ;

Lawitschka, Anita ;

Peters, Christina ;

Gadner, Helmut ;

Matthes-Martin, Susanne .

BLOOD, 2009, 113 (22) :5689-5691

[8] The spectrum of autoantibodies in IPEX syndrome is broad and includes anti-mitochondrial autoantibodies [J].

Tsuda, Masanobu ;

Torgerson, Troy R. ;

Selmi, Carlo ;

Gambineri, Eleonora ;

Carneiro-Sampaio, Magda ;

Mannurita, Sara Ciullini ;

Leung, Patrick S. C. ;

Norman, Gary L. ;

Gershwin, M. Eric .

JOURNAL OF AUTOIMMUNITY, 2010, 35 (03) :265-268

[9] Clinical and molecular features of the immunodysregulation, polyendocrinopathy, enteropathy, X linked (IPEX) syndrome [J].

Wildin, RS ;

Smyk-Pearson, S ;

Filipovich, AH .

JOURNAL OF MEDICAL GENETICS, 2002, 39 (08) :537-545

[10] Immune reconstitution and recovery of FOXP3 (forkhead box P3)-expressing T cells after transplantation for IPEX (immune dysregulation, polyendocrinopathy, enteropathy, X-linked) syndrome [J].

Zhan, Hong ;

Sinclair, Jo ;

Adams, Stuart ;

Cale, Catherine M. ;

Murch, Simon ;

Perroni, Lucia ;

Davies, Graham ;

Amrolia, Persis ;

Qasim, Waseem .

PEDIATRICS, 2008, 121 (04) :E998-E1002

← 1 →