Literature DB >> 24270390

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

Z Nademi1, M Slatter1, E Gambineri2, S C Mannurita2, D Barge3, S Hodges4, S Bunn4, J Thomas4, B Haugk5, S Hambleton1, T Flood6, A Cant1, M Abinun1, A Gennery1.   

Abstract

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Year:  2013        PMID: 24270390     DOI: 10.1038/bmt.2013.181

Source DB:  PubMed          Journal:  Bone Marrow Transplant        ISSN: 0268-3369            Impact factor:   5.483


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  10 in total

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

Authors:  Lauri M Burroughs; Troy R Torgerson; Rainer Storb; Paul A Carpenter; David J Rawlings; Jean Sanders; Andrew M Scharenberg; Suzanne Skoda-Smith; Janet Englund; Hans D Ochs; Ann E Woolfrey
Journal:  J Allergy Clin Immunol       Date:  2010-11       Impact factor: 10.793

2.  Submyeloablative cord blood transplantation corrects clinical defects seen in IPEX syndrome.

Authors:  K G Lucas; D Ungar; M Comito; M Bayerl; B Groh
Journal:  Bone Marrow Transplant       Date:  2006-11-20       Impact factor: 5.483

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

Authors:  Markus G Seidel; Gerhard Fritsch; Thomas Lion; Birgit Jürgens; Andreas Heitger; Rosa Bacchetta; Anita Lawitschka; Christina Peters; Helmut Gadner; Susanne Matthes-Martin
Journal:  Blood       Date:  2009-05-28       Impact factor: 22.113

4.  Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning.

Authors:  Aarati Rao; Naynesh Kamani; Alexandra Filipovich; Susan Molleran Lee; Stella M Davies; Jignesh Dalal; Shalini Shenoy
Journal:  Blood       Date:  2006-09-21       Impact factor: 22.113

5.  The spectrum of autoantibodies in IPEX syndrome is broad and includes anti-mitochondrial autoantibodies.

Authors:  Masanobu Tsuda; Troy R Torgerson; Carlo Selmi; Eleonora Gambineri; Magda Carneiro-Sampaio; Sara Ciullini Mannurita; Patrick S C Leung; Gary L Norman; M Eric Gershwin
Journal:  J Autoimmun       Date:  2010-07-22       Impact factor: 7.094

Review 6.  Clinical and molecular features of the immunodysregulation, polyendocrinopathy, enteropathy, X linked (IPEX) syndrome.

Authors:  R S Wildin; S Smyk-Pearson; A H Filipovich
Journal:  J Med Genet       Date:  2002-08       Impact factor: 6.318

7.  Clinical and molecular profile of a new series of patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome: inconsistent correlation between forkhead box protein 3 expression and disease severity.

Authors:  Eleonora Gambineri; Lucia Perroni; Laura Passerini; Lucia Bianchi; Claudio Doglioni; Franco Meschi; Riccardo Bonfanti; Yves Sznajer; Alberto Tommasini; Anita Lawitschka; Anne Junker; Desiree Dunstheimer; Peter H Heidemann; Giantonio Cazzola; Marco Cipolli; Wilhelm Friedrich; Dragana Janic; Nadira Azzi; Erick Richmond; Silvia Vignola; Arrigo Barabino; Giuseppe Chiumello; Chiara Azzari; Maria-Grazia Roncarolo; Rosa Bacchetta
Journal:  J Allergy Clin Immunol       Date:  2008-10-25       Impact factor: 10.793

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

Authors:  Hong Zhan; Jo Sinclair; Stuart Adams; Catherine M Cale; Simon Murch; Lucia Perroni; Graham Davies; Persis Amrolia; Waseem Qasim
Journal:  Pediatrics       Date:  2008-03-03       Impact factor: 7.124

9.  Defective regulatory and effector T cell functions in patients with FOXP3 mutations.

Authors:  Rosa Bacchetta; Laura Passerini; Eleonora Gambineri; Minyue Dai; Sarah E Allan; Lucia Perroni; Franca Dagna-Bricarelli; Claudia Sartirana; Susanne Matthes-Martin; Anita Lawitschka; Chiara Azzari; Steven F Ziegler; Megan K Levings; Maria Grazia Roncarolo
Journal:  J Clin Invest       Date:  2006-06       Impact factor: 14.808

10.  Immune dysregulation, polyendocrinopathy, enteropathy, x-linked syndrome: a paradigm of immunodeficiency with autoimmunity.

Authors:  Federica Barzaghi; Laura Passerini; Rosa Bacchetta
Journal:  Front Immunol       Date:  2012-07-31       Impact factor: 7.561

  10 in total
  12 in total

Review 1.  Autoimmune Enteropathy: An Updated Review with Special Focus on Stem Cell Transplant Therapy.

Authors:  Zunirah Ahmed; Aamer Imdad; James A Connelly; Sari Acra
Journal:  Dig Dis Sci       Date:  2018-11-10       Impact factor: 3.199

2.  Recessively Inherited LRBA Mutations Cause Autoimmunity Presenting as Neonatal Diabetes.

Authors:  Matthew B Johnson; Elisa De Franco; Hana Lango Allen; Aisha Al Senani; Nancy Elbarbary; Zeynep Siklar; Merih Berberoglu; Zineb Imane; Alireza Haghighi; Zahra Razavi; Irfan Ullah; Saif Alyaarubi; Daphne Gardner; Sian Ellard; Andrew T Hattersley; Sarah E Flanagan
Journal:  Diabetes       Date:  2017-05-04       Impact factor: 9.461

Review 3.  Hematopoietic stem cell transplantation for primary immunodeficiencies.

Authors:  Elizabeth Kang; Andrew Gennery
Journal:  Hematol Oncol Clin North Am       Date:  2014-09-16       Impact factor: 3.722

4.  Successful Hematopoietic Stem Cell Transplantation in a Patient with LPS-Responsive Beige-Like Anchor (LRBA) Gene Mutation.

Authors:  Bianca Tesi; Peter Priftakis; Fredrik Lindgren; Samuel C C Chiang; Nikolaos Kartalis; Alexandra Löfstedt; Esther Lörinc; Jan-Inge Henter; Jacek Winiarski; Yenan T Bryceson; Marie Meeths
Journal:  J Clin Immunol       Date:  2016-05-04       Impact factor: 8.317

5.  Gut immune reconstitution in immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome after hematopoietic stem cell transplantation.

Authors:  Eleonora Gambineri; Sara Ciullini Mannurita; Helen Robertson; Marina Vignoli; Beate Haugk; Paolo Lionetti; Sophie Hambleton; Dawn Barge; Andrew R Gennery; Mary Slatter; Zohreh Nademi; Terence J Flood; Anthony Jackson; Mario Abinun; Andrew J Cant
Journal:  J Allergy Clin Immunol       Date:  2014-10-25       Impact factor: 10.793

6.  Treatment of Infantile Inflammatory Bowel Disease and Autoimmunity by Allogeneic Stem Cell Transplantation in LPS-Responsive Beige-Like Anchor Deficiency.

Authors:  Shahrzad Bakhtiar; Laura Gámez-Díaz; Andrea Jarisch; Jan Soerensen; Bodo Grimbacher; Bernd Belohradsky; Klaus-Michael Keller; Christoph Rietschel; Thomas Klingebiel; Sibylle Koletzko; Michael H Albert; Peter Bader
Journal:  Front Immunol       Date:  2017-01-31       Impact factor: 7.561

7.  Resetting microbiota by Lactobacillus reuteri inhibits T reg deficiency-induced autoimmunity via adenosine A2A receptors.

Authors:  Baokun He; Thomas K Hoang; Ting Wang; Michael Ferris; Christopher M Taylor; Xiangjun Tian; Meng Luo; Dat Q Tran; Jain Zhou; Nina Tatevian; Fayong Luo; Jose G Molina; Michael R Blackburn; Thomas H Gomez; Stefan Roos; J Marc Rhoads; Yuying Liu
Journal:  J Exp Med       Date:  2016-12-19       Impact factor: 14.307

Review 8.  Precision diabetes: learning from monogenic diabetes.

Authors:  Andrew T Hattersley; Kashyap A Patel
Journal:  Diabetologia       Date:  2017-03-17       Impact factor: 10.122

9.  Clinical Heterogeneity of Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome: A French Multicenter Retrospective Study.

Authors:  R Duclaux-Loras; F Charbit-Henrion; B Neven; J Nowak; S Collardeau-Frachon; C Malcus; P F Ray; D Moshous; J Beltrand; O Goulet; N Cerf-Bensussan; A Lachaux; F Rieux-Laucat; F M Ruemmele
Journal:  Clin Transl Gastroenterol       Date:  2018-11-02       Impact factor: 4.488

10.  Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

Authors:  Federica Barzaghi; Laura Cristina Amaya Hernandez; Benedicte Neven; Silvia Ricci; Zeynep Yesim Kucuk; Jack J Bleesing; Zohreh Nademi; Mary Anne Slatter; Erlinda Rose Ulloa; Anna Shcherbina; Anna Roppelt; Austen Worth; Juliana Silva; Alessandro Aiuti; Luis Murguia-Favela; Carsten Speckmann; Magda Carneiro-Sampaio; Juliana Folloni Fernandes; Safa Baris; Ahmet Ozen; Elif Karakoc-Aydiner; Ayca Kiykim; Ansgar Schulz; Sandra Steinmann; Lucia Dora Notarangelo; Eleonora Gambineri; Paolo Lionetti; William Thomas Shearer; Lisa R Forbes; Caridad Martinez; Despina Moshous; Stephane Blanche; Alain Fisher; Frank M Ruemmele; Come Tissandier; Marie Ouachee-Chardin; Frédéric Rieux-Laucat; Marina Cavazzana; Waseem Qasim; Barbarella Lucarelli; Michael H Albert; Ichiro Kobayashi; Laura Alonso; Cristina Diaz De Heredia; Hirokazu Kanegane; Anita Lawitschka; Jong Jin Seo; Marta Gonzalez-Vicent; Miguel Angel Diaz; Rakesh Kumar Goyal; Martin G Sauer; Akif Yesilipek; Minsoo Kim; Yesim Yilmaz-Demirdag; Monica Bhatia; Julie Khlevner; Erick J Richmond Padilla; Silvana Martino; Davide Montin; Olaf Neth; Agueda Molinos-Quintana; Justo Valverde-Fernandez; Arnon Broides; Vered Pinsk; Antje Ballauf; Filomeen Haerynck; Victoria Bordon; Catharina Dhooge; Maria Laura Garcia-Lloret; Robbert G Bredius; Krzysztof Kałwak; Elie Haddad; Markus Gerhard Seidel; Gregor Duckers; Sung-Yun Pai; Christopher C Dvorak; Stephan Ehl; Franco Locatelli; Frederick Goldman; Andrew Richard Gennery; Mort J Cowan; Maria-Grazia Roncarolo; Rosa Bacchetta
Journal:  J Allergy Clin Immunol       Date:  2017-12-11       Impact factor: 10.793

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