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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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Could targeted gene insertion of factor 9 be a potential durable treatment for Hemophilia B?

Expert Rev Hematol

January 2025

Department of Life Sciences and Biotechnology and Laboratorio per le Tecnologie delle Terapie Avanzate (LTTA), University of Ferrara, Ferrara, Italy.

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Stromal vascular fraction (SVF) is a heterogeneous collection of cells obtained from adipose tissue through lipoaspiration and is an alter-native intraarticular treatment option, especially in osteoarthritis (OA). The anti-inflammatory and extracellular tissue repair-stimulating properties of SVF increase its effectiveness in regeneration and repair mechanisms. One of the most common symptoms of hemophilia A and B is hemophilic arthropathy (HA).

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Avascular necrosis (AVN) of the femoral head accounts for up to 10% of all total hip arthroplasties performed annually. Typically associated with intravascular coagulation, AVN is extremely rare in patients with bleeding disorders such as hemophilia B. In this report, we describe the therapeutic management of a 46-year-old male with hemophilia B, presenting with chronic left hip pain and AVN of the femoral head.

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Background: The impact of nonneutralizing antibodies (NNAs) in moderate hemophilia is elusive.

Objectives: To explore the presence of NNAs in Nordic persons with moderate hemophilia A (MHA) and B (MHB) in relation to treatment modality, clinical outcome, history of inhibitor, and the corresponding factor VIII (FVIII)/factor IX (FIX) gene mutation.

Methods: A cross-sectional multicenter study covering persons with MHA and MHB in Sweden, Finland, and Norway.

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