Long-term outcome in Wiskott-Aldrich syndrome and X-linked thrombocytopenia patients: an observational -prospective multi-center study of the Italian Primary Immune Deficiency Network (IPINET)Research in context

Long-term outcome in Wiskott-Aldrich syndrome and X-linked thrombocytopenia patients: an observational -prospective multi-center study of the Italian Primary Immune Deficiency Network (IPINET)Research in context

Summary: Background: Wiskott-Aldrich Syndrome (WAS) is characterized by eczema, infections, and severe bleeding, but may also include autoimmunity and malignancy. Subjects with X-linked thrombocytopenia (XLT) can display a mild phenotype, although severe complications may occur at any age. WAS and...

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Main Authors: Annarosa Soresina, Roberto Rondelli, Lucia Dora Notarangelo, Franco Locatelli, Alessandro Aiuti, Alessandra Biffi, Marco Rabusin, Claudio Pignata, Giuseppe Menna, Arcangelo Prete, Maura Faraci, Attilio Rovelli, Francesca Conti, Patrizia Bertolini, Chiara Azzari, Caterina Cancrini, Marco Zecca, Francesca Ferrua, Maria Pia Cicalese, Francesco Cecere, Laura Dotta, Baldassarre Martire, Silvia Giliani, Daniele Moratto, Cinzia Mazza, Alessandro Plebani, Luigi D. Notarangelo, Andrea Pession, Raffaele Badolato, Fulvio Fulvio
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:EClinicalMedicine
Subjects:
Wiskott-Aldrich syndrome
X-linked thrombocytopenia
Overall survival
Disease-related events
Management
Online Access:http://www.sciencedirect.com/science/article/pii/S2589537025002032
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