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

Barzaghi, Federica; Cristina Amaya Hernandez, Laura; Neven, Bénédicte; Ricci, Silvia; Yesim Kucuk, Zeynep; Bleesing, Jack; Nademi, Zohreh; Slatter, Mary; R. Ulloa, Erlinda; Shcherbina, Anna; Roppelt, Anna; Worth, Austen; Silva, Juliana; Aiuti, Alessandro; Murguía-Favela, Luis; Speckmann, Carsten; Carneiro‐Sampaio, Magda; Folloni Fernandes, Juliana; Barış, Safa; Özen, Ahmet; Karakoç-Aydıner, Elif; Kıykım, Ayça; Schulz, Ansgar; Steinmann, Sandra; Dora Notarangelo, Lucia; Gambineri, Eleonora; Lionetti, Paolo; T. Shearer, William; R. Forbes, Lisa; Martinez, Caridad; Moshous, Despina; Blanche, Stéphane; Fisher, Alain; M. Ruemmele, Frank; Tissandier, Côme; Marie, O; Rieux‐Laucat, Frédéric; Cavazzana, Marina; Qasim, Waseem; Lucarelli, Barbarella; H. Albert, Michael; Kobayashi, Ichiro; Alonso, Laura; Díaz de Heredia, Cristina; Kanegane, Hirokazu; Lawitschka, Ánita; Jin Seo, Jong; González‐Vicent, Marta; Ángel Díaz, Miguel; K. Goyal, Rakesh; G. Sauer, Martin; Yeşilipek, Akif; Kim, Min‐Soo; Yılmaz Demirdağ, Yeşim; Bhatia, Monica; Khlevner, Julie; J. Richmond Padilla, Erick; Martino, Silvana; Montin, Davide; Neth, Olaf; Molinos-Quintana, Águeda; Valverde-Fernández, Justo; Broides, Arnon; Pinsk, Vered; Ballauf, Antje; Haerynck, Filomeen; Bordon, Victoria; Dhooge, Catharina; Garcia-Lloret, Maria; G. M. Bredius, Robbert; Kałwak, Krzysztof; Haddad, Élie; G. Seidel, Markus; Dückers, Gregor; Pai, Sung‐Yun; C. Dvorak, Christopher; Ehl, Stephan; Locatelli, Franco; D. Goldman, Frederick; R. Gennery, Andrew; J. Cowan, Mort; Roncarolo, Maria-Grazia; Bacchetta, Rosa

doi:10.1016/j.jaci.2017.10.041

Public

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

Published in Journal of Allergy and Clinical Immunology • Dec 11, 2017

Authors:

Federica Barzaghi

Laura Cristina Amaya Hernandez

Bénédicte Neven

Abstract

BACKGROUND: Immunodysregulation polyendocrinopathy enteropathy x-linked (IPEX) syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) and i...

Subject

Medicine

Retrospective cohort study

Multicenter study

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