Experience of a new high‐purity factor X concentrate in subjects with hereditary factor X deficiency undergoing surgery
Open Access
- 24 May 2016
- journal article
- research article
- Published by Wiley in Haemophilia
- Vol. 22 (5), 713-720
- https://doi.org/10.1111/hae.12954
Abstract
Introduction 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 (pdFX) has been developed for patients with hereditary FX deficiency. Aim Our objective was to assess the safety and efficacy of pdFX in subjects with FX deficiency undergoing surgery. Methods Subjects with hereditary mild‐to‐severe FX deficiency (basal plasma FX activity [FX:C] 50 IU dL−1 until the subject was no longer at risk of bleeding due to surgery. Efficacy of pdFX 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 (AEs), 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, pdFX treatment was assessed as “excellent” in preventing bleeding and achieving haemostasis. No blood transfusions were required, no AEs related to pdFX were observed, and no clinically significant trends were found in any laboratory parameters. Conclusion These data demonstrate that pdFX is safe and effective as replacement therapy in five subjects with mild‐to‐severe FX deficiency undergoing surgery on seven occasions.Keywords
Funding Information
- Baxter International
- Biogen
- CSL Behring
- Pfizer
This publication has 15 references indexed in Scilit:
- Efficacy, safety and pharmacokinetics of a new high‐purity factor X concentrate in subjects with hereditary factor X deficiencyHaemophilia, 2016
- Guideline for the diagnosis and management of the rare coagulation disordersBritish Journal of Haematology, 2014
- Kreuth III: European consensus proposals for treatment of haemophilia with coagulation factor concentratesHaemophilia, 2014
- Successful use of BPL Factor X concentrate in a child with severe factor X deficiencyThe Journal of Haemophilia Practice, 2013
- Efficacy of prophylaxis and genotype‐phenotype correlation in patients with severe Factor X deficiency in IranHaemophilia, 2011
- Factor X DeficiencySeminars in Thrombosis and Hemostasis, 2009
- Diagnosis and treatment of inherited factor X deficiencyHaemophilia, 2008
- Factor X deficiency: clinical manifestation of 102 subjects from Europe and Latin America with mutations in the factor 10 geneHaemophilia, 2006
- Recessively inherited coagulation disordersBlood, 2004
- Congenital factor X deficiency: spectrum of bleeding symptoms in 32 Iranian patientsBritish Journal of Haematology, 1998