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Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France.
Ther Adv Hematol
January 2025
Sobi, Basel, Switzerland.
Background: More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.
Objectives: We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.
Methods: Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE.
The Diagnostic Assessment of Inherited Platelet Function Defects.
Hamostaseologie
January 2025
Center for Clinical Transfusion Medicine Tuebingen, Tuebingen, Germany.
In this article, our goal is to offer an introduction and overview of the diagnostic approach to inherited platelet function defects (iPFDs) for clinicians and laboratory personnel who are beginning to engage in the field. We describe the most commonly used laboratory methods and propose a diagnostic four-step approach, wherein each stage requires a higher level of expertise and more specialized methods. It should be noted that our proposed approach differs from the ISTH Guidance on this topic in some points.
View Article and Find Full Text PDFComparison of Single Knee Arthroplasty and Bilateral Knee Arthroplasty in Haemophiliacs During a Single Operation: A Systematic Review and Meta-Analysis.
Haemophilia
January 2025
Department of Orthopedics, The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, China.
Background: Arthroplasty is the standard treatment for end-stage haemophilic knee arthritis; however, the choice between single knee arthroplasty (SKA) and bilateral knee arthroplasty (BKA) in a single operation remains controversial due to the risks specific to haemophiliacs.
Methods: Two independent researchers conducted searches across CNKI, CBM, Wanfang, PubMed, Cochrane Library, Embase, and Web of Science, with the last search performed on 15 October 2024. Study results include joint function, complication and various cost.
Operationalising a Haemophilia Gene Editing Lexicon for Practical Use.
Haemophilia
January 2025
Medicine and Pathology, Georgetown University, Washington, District of Columbia, USA.
Introduction: Gene editing therapies offer the possibility of substantial improvement in treatment and quality of life for people with haemophilia (PWH) in a landscape of dynamic therapeutic advancement. Developing a common and understandable language to discuss gene editing will be essential to ensure these treatments can be deployed in a safe and effective manner with fully informed and shared decision-making between healthcare professionals (HCPs) and PWH. A lexicon explaining and clarifying key concepts is one potential tool to address these aims.
View Article and Find Full Text PDFGene-ius at work: Hemophilia B treatment enters a new era.
Am J Health Syst Pharm
January 2025
Department of Pharmacy Practice, Auburn University Harrison College of Pharmacy, Auburn, AL, USA.
Disclaimer: In an effort to expedite the publication of articles, AJHP is posting manuscripts online as soon as possible after acceptance. Accepted manuscripts have been peer-reviewed and copyedited, but are posted online before technical formatting and author proofing. These manuscripts are not the final version of record and will be replaced with the final article (formatted per AJHP style and proofed by the authors) at a later time.
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