Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome

• Published in: Blood Vol. 121; no. 9; pp. 1510 - 1516
• Main Authors: Mahlaoui, Nizar, Pellier, Isabelle, Mignot, Cécile, Jais, Jean-Philippe, Bilhou-Nabéra, Chrystèle, Moshous, Despina, Neven, Bénédicte, Picard, Capucine, de Saint-Basile, Geneviève, Cavazzana-Calvo, Marina, Blanche, Stéphane, Fischer, Alain
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 28.02.2013
• Subjects: Adolescent; Adult; Age of Onset; Child; Child, Preschool; Cohort Studies; Humans; Infant; Infant, Newborn; Prognosis; Research Design; Severity of Illness Index; Wiskott-Aldrich Syndrome - diagnosis; Wiskott-Aldrich Syndrome - epidemiology; Wiskott-Aldrich Syndrome - pathology; Young Adult

On the basis of a nationwide database of 160 patients with Wiskott-Aldrich syndrome (WAS), we identified a subset of infants who were significantly more likely to be attributed with an Ochs score of 5 before the age of 2 (n = 26 of 47 [55%], P = 2.8 × 10−7). A retrospective analysis revealed that th...