Article
Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.
The Journal of allergy and clinical immunology - 1 Mar 2018
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
Abstract excerpt
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 immunosuppression (IS), have not been thoroughly examined. OBJECTIVE: This analysis sought to evaluate disease onset,...
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