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Experience of a new high‐purity factor X concentrate in subjects with hereditary factor X deficiency undergoing surgery
Author(s) -
Escobar M. A.,
Auerswald G.,
Austin S.,
Huang J. N.,
Norton M.,
Millar C. M.
Publication year - 2016
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.12954
Subject(s) - medicine , surgery , adverse effect , coagulation
Maintaining haemostasis in surgery is challenging for hereditary rare bleeding disorders in which multi‐coagulation‐factor concentrates are the only therapeutic option. Hereditary factor X ( FX ) deficiency affects 1:500 000 to 1:1 000 000 individuals, and no specific replacement FX concentrate has been available. A high‐purity, plasma‐derived FX concentrate (pd FX ) has been developed for patients with hereditary FX deficiency. Aim Our objective was to assess the safety and efficacy of pd FX in subjects with FX deficiency undergoing surgery. Methods Subjects with hereditary mild‐to‐severe FX deficiency (basal plasma FX activity [ FX :C] <20 IU dL −1 ) undergoing surgery received pd FX preoperatively to raise FX :C to 70–90 IU dL −1 and postoperatively to maintain levels >50 IU dL −1 until the subject was no longer at risk of bleeding due to surgery. Efficacy of pd FX was assessed by blood loss during surgery, requirement for blood transfusion, postoperative bleeding from the surgical or other sites, and changes in haemoglobin levels. Safety was assessed by adverse events ( AE s), development of inhibitors, and clinically significant changes in laboratory parameters. Results Five subjects (aged 14–59 years) underwent seven surgical procedures (four major and three minor). Treatment duration was 1–15 days. For each procedure, pd FX treatment was assessed as “excellent” in preventing bleeding and achieving haemostasis. No blood transfusions were required, no AE s related to pd FX were observed, and no clinically significant trends were found in any laboratory parameters. Conclusion These data demonstrate that pd FX is safe and effective as replacement therapy in five subjects with mild‐to‐severe FX deficiency undergoing surgery on seven occasions.