Indira Warrier

TL;DR: This report begins with a brief summary of the panel’s recommendations, followed by a more detailed analysis of its methodology, the findings of the comprehensive literature review, and a full presentation of the recommendations.

TL;DR: In this article, the authors evaluated the pharmacokinetics, efficacy and safety of a plasma-free recombinant FVIII concentrate, ADVATE [Antihemophilic Factor (Recombinant), Plasma/Albumin-Free Method, rAHF-PFM], in children < 6 years of age with severe hemophilia.

TL;DR: To the Editor: Inhibitors to factor IX (fIX) develop in approximately 1.5% to 3% of persons with severe hemophilia B and are commonly associated with the complete absence of fIX antigen due to large gene deletions or nonsense mutations.

TL;DR: By preventing new target joints, prophylaxis can lead to reduction in long‐term morbidity and a better quality of life despite increased central lines and higher factor usage.

TL;DR: The very high risk of anaphylaxis associated with a complete gene deletion suggests that the lack of expression of a partial protein product may predispose to anphylaxis and/or that the absence of a closely linked, codeleted gene enhances the anaphyactic immune response.