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Limperger, Verena; Kenet, Gili; Goldenberg, Neil A.; Heller, Christine; Holzhauer, Susanne; Junker, Ralf; Klostermeier, Ulrich C.; Knoefler, Ralf; Kurnik, Karin; Krümpel, Anne; Mesters, Rolf; Stach, Michael; Young, Guy; Nowak-Göttl, Ulrike (2016): Impact of high-risk thrombophilia status on recurrence among children with a first non-central-venous-catheter-associated VTE: an observational multicentre cohort study. In: British Journal of Haematology, Vol. 175, No. 1: pp. 133-140
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Abstract

Deficiency of antithrombin (AT), protein C (PC) or protein S (PS) constitutes a major risk factor for venous thromboembolism (VTE). Individuals at high risk for recurrence who benefit from screening need to be identified. The primary study objective was to determine the individual recurrence risk among children with a first non-central-venous-catheter-associated VTE with respect to their thrombophilia status and to evaluate if the clinical presentation at first VTE onset differs between children with AT, PC or PS deficiency versus no thrombophilia. We calculated the absolute risk of VTE recurrence and event-free-survival adjusted for thrombophilia, age, sex and positive family VTE history in 161 consecutively enrolled paediatric VTE patients. The presence of a deficiency relative to no thrombophilia was evaluated as a potential predictor of recurrence. Predictors for recurrence were AT deficiency (hazard ratio/95% CI: 65/246-172) and female gender (26/11-635). The annual recurrence rates (95% CIs) were 54% (26-10) in AT-deficient children, 13% (03-38) in patients with PC deficiency, 07% (008-24) in the PS-deficient cohort and 09% (04-18) in patients with no thrombophilia. Positive family VTE history or combined thrombophilias did not predict recurrence. Given the overall annual incidence rate of recurrence of 15% we suggest screening for AT deficiency in children with VTE.