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Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.
BACKGROUND Effective ways to prevent arthropathy in severe hemophilia are unknown. METHODS We randomly assigned young boys with severe hemophilia A to regular infusions of recombinant factor VIIIExpand
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Low-Dose Tissue Plasminogen Activator Thrombolysis in Children
Purpose To compare results of low-dose tissue plasminogen activator (TPA) in children with arterial and venous thrombi relative to standard published dosing. Methods Subjects consisted of allExpand
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A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.
BACKGROUND The development of neutralizing anti-factor VIII alloantibodies (inhibitors) in patients with severe hemophilia A may depend on the concentrate used for replacement therapy. METHODS WeExpand
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Impact of Inherited Thrombophilia on Venous Thromboembolism in Children: A Systematic Review and Meta-Analysis of Observational Studies
Background— The aim of the present study was to estimate the impact of inherited thrombophilia (IT) on the risk of venous thromboembolism (VTE) onset and recurrence in children by a meta-analysis ofExpand
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Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B.
BACKGROUND Prophylactic factor replacement in patients with hemophilia B improves outcomes but requires frequent injections. A recombinant factor IX Fc fusion protein (rFIXFc) with a prolongedExpand
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Post-thrombotic syndrome in children: a systematic review of frequency of occurrence, validity of outcome measures, and prognostic factors
Background Post-thrombotic syndrome is a manifestation of chronic venous insufficiency following deep venous thrombosis. This systematic review was conducted to critically evaluate pediatric evidenceExpand
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How I treat venous thrombosis in children.
Evidence-based medicine for pediatric thrombosis is in its infancy. As recently as 10 years ago, antithrombotic therapy for infants and children was largely based on individual empiric experience,Expand
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von Willebrand disease (VWD): evidence‐based diagnosis and management guidelines, the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report (USA) 1
Summary.  von Willebrand disease (VWD) is a commonly encountered inherited bleeding disorder affecting both males and females, causing mucous membrane and skin bleeding symptoms, and bleeding withExpand
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Impact of Thrombophilia on Risk of Arterial Ischemic Stroke or Cerebral Sinovenous Thrombosis in Neonates and Children: A Systematic Review and Meta-Analysis of Observational Studies
Background— The aim of this study was to estimate the impact of thrombophilia on risk of first childhood stroke through a meta-analysis of published observational studies. Methods and Results— AExpand
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Assay of the von Willebrand factor (VWF) propeptide to identify patients with type 1 von Willebrand disease with decreased VWF survival.
Type 1 von Willebrand disease (VWD) is characterized by a partial quantitative deficiency of von Willebrand factor (VWF). Few VWF gene mutations have been identified that cause dominant type 1 VWD.Expand
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