Clinical Features, Treatments, and Outcomes of an International Cohort of 96 Children with Takayasu Arteritis.
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OBJECTIVE: To characterize the clinical features, treatment, and outcomes of a large, international cohort of childhood-onset Takayasu arteritis (cTAK). METHODS: Patients with a diagnosis of cTAK were identified in A Registry of Childhood Vasculitis and divided into subcohorts by era-of diagnosis: pre-biologic (PB; 1986-2006), early biologic (EB; 2007-2017), and recently diagnosed (RD; 2018-present). Longitudinal patient data were collected at time of diagnosis (TOD), postinduction (PI; 3-6 months after TOD), and 12 months after TOD (12m). Meaningful clinical improvement was defined as at least a 50% reduction in the Pediatric Vasculitis Activity Score. Damage was assessed by the Pediatric Vasculitis Damage Index. RESULTS: Among 96 patients (69% female, median age 12.8 years [interquartile range 9.8-15.3]) from nine countries, 86% showed clinical improvement at PI and 90% maintained improvement at 12m. Glucocorticoids (GC; n = 84, 88%) and methotrexate (n = 47, 49%) were the two most common medications prescribed at TOD, often together and in combination with another medication. GC use continued at 12m in 79%. Patients more recently diagnosed were more likely to receive treatment with tumor necrosis factor inhibitors (0% PB, 26% EB, and 35% RD; P = 0.007) and reach 70% improvement (59% PB, 81% EB, and 100% RD; P = 0.007) at PI. However, neither improvement nor damage at 12m differed among the subcohorts. CONCLUSION: Although treatment approaches for cTAK have evolved and most patients demonstrate sustained clinical improvement at 12m, these advances have not translated into substantial changes in outcomes, highlighting the need for optimized therapeutic strategies.