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Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation
Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation
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Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation

Maria Teresa de la Morena David Leonard; Troy R Torgerson; Otavio Cabral Marques; Mary Anne Slatter; Asghar Aghamohammadi; Sharat Chandra; Luis Murguia Favela; Francisco A Bonilla; Maria Kanariou; Rongras Damrongwatanasuk; Caroline Y Kuo; Christopher C Dvorak; Isabelle Meyts; Karin Chen; Lisa Kobrynski; Neena Kapoor; Darko Richter; Daniela DiGiovanni; Fatima Dhalla; Evangelia Farmaki; Carsten Speckmann; Teresa Español; Anna YU Shcherbina; Imelda Celine Guerra Hanson; Jiri Litzman; John M Routes; Melanie Wong; Ramsay Fuleihan; Suranjith L Seneviratne; Trudy N Small; Ales Janda; Liliana Bezrodnik; Reinhard Seger; Andrea Gomez Raccio; John David M Edgar; Janet Chou; Jordan Keith Abbott; Joris van Montfrans; Luis Ignacio González Granado; Nancy Bunin; Necil Kutukculer; Paul Gray; Gisela Seminario; Srdjan Pasic; Victor Aquino; Christian Wysocki; Hassan Abolhassani; Morna Dorsey; Charlotte Cunningham Rundles; Alan Paul Knutsen; John William Sleasman; Beatriz Tavares Costa Carvalho; Antonio Condino-Neto; Eyal Grunebaum; Helen Chapel; Hans Dieter Ochs; Alexandra Hult Filipovich; Mort Cowan; Andrew Gennery; Andrew James Cant; Luigi D Notarangelo; Chaim M Roifman

Journal of Allergy and Clinical Immunology v. 139, n. 4, p. 1282-1292, 2017

Philadelphia 2017

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2
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study
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Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study

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

Journal of allergy and clinical immunology, 2018-03, Vol.141 (3), p.1036-1049.e5 [Peer Reviewed Journal]

United States: Elsevier Inc

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3
Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study
Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study
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Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study

Ferrua, Francesca ; Galimberti, Stefania ; Courteille, Virginie ; Slatter, Mary Anne ; Booth, Claire ; Moshous, Despina ; Neven, Benedicte ; Blanche, Stephane ; Cavazzana, Marina ; Laberko, Alexandra ; Shcherbina, Anna ; Balashov, Dmitry ; Soncini, Elena ; Porta, Fulvio ; Al-Mousa, Hamoud ; Al-Saud, Bandar ; Al-Dhekri, Hasan ; Arnaout, Rand ; Formankova, Renata ; Bertrand, Yves ; Lange, Andrzej ; Smart, Joanne ; Wolska-Kusnierz, Beata ; Aquino, Victor M ; Dvorak, Christopher C ; Fasth, Anders ; Fouyssac, Fanny ; Heilmann, Carsten ; Hoenig, Manfred ; Schuetz, Catharina ; Kelecic, Jadranka ; Bredius, Robbert G.M ; Lankester, Arjan C ; Lindemans, Caroline A ; Suarez, Felipe ; Sullivan, Kathleen E ; Albert, Michael H ; Kalwak, Krzysztof ; Barlogis, Vincent ; Bhatia, Monica ; Bordon, Victoria ; Czogala, Wojciech ; Alonso, Laura ; Dogu, Figen ; Gozdzik, Jolanta ; Ikinciogullari, Aydan ; Krivan, Gergely ; Ljungman, Per ; Meyts, Isabelle ; Mustillo, Peter ; Smith, Angela R ; Speckmann, Carsten ; Sundin, Mikael ; Keogh, Steven John ; Shaw, Peter John ; Boelens, Jaap Jan ; Schulz, Ansgar S ; Sedlacek, Petr ; Veys, Paul ; Mahlaoui, Nizar ; Janda, Ales ; Davies, E. Graham ; Fischer, Alain ; Cowan, Morton J ; Gennery, Andrew Richard

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2019-06, Vol.143 (6), p.2238-2253 [Peer Reviewed Journal]

MOSBY-ELSEVIER

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4
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies : an international multicenter retrospective study
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies : an international multicenter retrospective study
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Long-term follow-up of IPEX syndrome patients after different therapeutic strategies : an international multicenter retrospective study

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

2018

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5
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study
Material Type:
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Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study

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

The Journal of allergy and clinical immunology, 2018-03, Vol.141 (3), p.1036-1049.e5

eScholarship, University of California

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Citations Cited by
6
Hematopoietic Cell Transplant for CD40 Ligand Deficiency—Comparing Busulfan Versus Treosulfan
Hematopoietic Cell Transplant for CD40 Ligand Deficiency—Comparing Busulfan Versus Treosulfan
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Hematopoietic Cell Transplant for CD40 Ligand Deficiency—Comparing Busulfan Versus Treosulfan

Carruthers, Vicky-Anne ; Lum, Su Han ; Flood, Terence ; Slatter, Mary A. ; Gennery, Andrew R.

Journal of clinical immunology, 2022-04, Vol.42 (3), p.703-705 [Peer Reviewed Journal]

New York: Springer US

Full text available

Citations Cited by
7
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
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
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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

Ozsahin, Hulya ; Cavazzana-Calvo, Marina ; Notarangelo, Luigi D. ; Schulz, Ansgar ; Thrasher, Adrian J. ; Mazzolari, Evelina ; Slatter, Mary A. ; Le Deist, Francoise ; Blanche, Stephane ; Veys, Paul ; Fasth, Anders ; Bredius, Robbert ; Sedlacek, Petr ; Wulffraat, Nico ; Ortega, Juan ; Heilmann, Carsten ; O’Meara, Anne ; Wachowiak, Jacek ; Kalwak, Krzysztof ; Matthes-Martin, Susanne ; Gungor, Tayfun ; Ikinciogullari, Aydan ; Landais, Paul ; Cant, Andrew J. ; Friedrich, Wilhelm ; Fischer, Alain

Blood, 2008-01, Vol.111 (1), p.439-445 [Peer Reviewed Journal]

Washington, DC: Elsevier Inc

Full text available

Citations Cited by
8
Monogenic early-onset lymphoproliferation and autoimmunity: Natural history of STAT3 gain-of-function syndrome
Monogenic early-onset lymphoproliferation and autoimmunity: Natural history of STAT3 gain-of-function syndrome
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Monogenic early-onset lymphoproliferation and autoimmunity: Natural history of STAT3 gain-of-function syndrome

Leiding, Jennifer W. ; Vogel, Tiphanie P. ; Smith, Madison R. ; Carisey, Alexandre ; Vargas-Hernández, Alexander ; Silva-Carmona, Manuel ; Neven, Bénédicte ; Ronan Leahy, Timothy ; Taskinen, Mervi ; Kostyuchenko, Larysa ; Scherbina, Anna ; Bauer, Cindy S. ; Giovannini-Chami, Lisa ; Romberg, Neil ; Prince, Benjamin T. ; Bohnsack, John ; Pachlopnik Schmid, Jana ; Bahna, Sami ; Brothers, Shannon ; Chan, Alice Y. ; Chandrakasan, Shanmuganathan ; Christiansen, Mette ; Cole, Theresa ; Desai, Mukesh M. ; Gottrand, Frédéric ; Grimbacher, Bodo ; Hoffman, Hal M. ; Guerrerio, Anthony L. ; Kainulainen, Leena ; Lawrence, Monica G. ; Martelius, Timi ; Neth, Olaf ; Lugo Reyes, Saúl Oswaldo ; Russell, Mark A. ; Schejter, Yael ; Skevofilax, Effie ; Suan, Daniel ; Velasco, Helena ; Walkovich, Kelly ; Aleshkevich, Svetlana ; Allende, Luis M. ; Atschekzei, Faranaz ; Aygunes, Utku ; Barlogis, Vincent ; Belko, John ; Bezrodnik, Liliana ; Biebl, Ariane ; Caldirola, Maria Soledad ; Castelle, Martin ; Cokugras, Haluk ; Cox, Fionnuala ; Dalgic, Buket ; ASH Dalm, Virgil ; Dujovny, Tal ; Eken, Ahmet ; Fabre, Alexandre ; Fischer, Alain ; Hannibal, Mark ; Huppert, Laura ; Karakoc-Aydiner, Elif ; Pac Kisaarslan, Ayşenur ; Kiykim, Ayca ; Klocperk, Adam ; Kuzmenko, Natalia ; Lianou, Dimitra ; Lorenz, Myriam Ricarda ; Manson, Ania ; Marega, Lia Furlaneto ; Nolan, David ; Norlin, Anna-Carin ; Oleastro, Matias ; Ozcan, Alper ; Picard, Capucine ; Polychronopoulou, Sophia ; Quesada, Juan Francisco ; Randall, Katrina L. ; Resin, Geraldine ; Rodina, Yulia ; Rohrlich, Pierre ; Sachs, Johnathan ; Sakovich, Inga ; Santarlas, Christopher ; Scheffler Mendoza, Selma C. ; Schwarz, Klaus ; Sediva, Anna ; Slatter, Mary ; Sleasman, John ; Suratannon, Narissara ; Tanita, Kay ; Travis, Stephen ; Tsinti, Maria ; Unal, Ekrem ; Urdinez, Luciano ; Vazquez-Gomez, Felisa ; Weiss, Mitchell J. ; Zhang, Yu ; Seppänen, Mikko R.J. ; Sogkas, Georgios ; Milner, Joshua D. ; Forbes Satter, Lisa R.

Journal of allergy and clinical immunology, 2023-04, Vol.151 (4), p.1081-1095 [Peer Reviewed Journal]

United States: Elsevier Inc

Full text available

Citations Cited by
9
Long-term immune reconstitution after anti-CD52–treated or anti-CD34–treated hematopoietic stem cell transplantation for severe T-lymphocyte immunodeficiency
Long-term immune reconstitution after anti-CD52–treated or anti-CD34–treated hematopoietic stem cell transplantation for severe T-lymphocyte immunodeficiency
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Long-term immune reconstitution after anti-CD52–treated or anti-CD34–treated hematopoietic stem cell transplantation for severe T-lymphocyte immunodeficiency

Slatter, Mary A., MD ; Brigham, Kenneth, MPhil ; Dickinson, Anne M., PhD ; Harvey, Helen L., BSc ; Barge, Dawn, PhD ; Jackson, Antony, BSc ; Bown, Nicholas, PhD ; Flood, Terence J., MD ; Cant, Andrew J., MD ; Abinun, Mario, MD ; Gennery, Andrew R., MD

Journal of allergy and clinical immunology, 2008-02, Vol.121 (2), p.361-367 [Peer Reviewed Journal]

New York, NY: Mosby, Inc

Full text available

Citations Cited by
10
Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity
Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity
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Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity

Bognàr, Tim ; Garcia-Rosa, Moises ; Lalmohamed, Arief ; Güngör, Tayfun ; Hauri-Hohl, Mathias ; Prockop, Susan ; Oram, Layne ; Pai, Sung-Yun ; Brooks, Jordan ; Savic, Rada M. ; Dvorak, Christopher C. ; Long-Boyle, Janel R. ; Krajinovic, Maja ; Bittencourt, Henrique ; Teyssier, Anne-Charlotte ; Théorêt, Yves ; Martinez, Cary ; Egberts, Toine C. G. ; Morales, Erin ; Slatter, Mary ; Cuvelier, Geoffrey D. E. ; Chiesa, Robert ; Wynn, Robert F. ; Coussons, Mary ; Cicalese, Maria P. ; Ansari, Marc ; Long, Susan E. ; Ebens, Christen L. ; Lust, Hannah ; Chaudhury, Sonali ; Nath, Christa E. ; Shaw, Peter J. ; Keogh, Steven J. ; van der Stoep, M. Y. Eileen C. ; Bredius, Robbert ; Lindemans, Caroline A. ; Boelens, Jaap-Jan ; Bartelink, Imke H.

Blood advances, 2024-07 [Peer Reviewed Journal]

Elsevier Inc

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