Pharmacokinetics of a high‐purity plasma‐derived factor X concentrate in subjects with moderate or severe hereditary factor X deficiency

Hereditary factor X (FX) deficiency affects 1:500 000 to 1:1 000 000 of individuals. There are few published data on the pharmacokinetics (PK) of FX for existing treatments for FX deficiency, and no specific replacement factor concentrate exists. A high‐purity plasma‐derived FX concentrate (pdFX) has been developed for use as replacement therapy in subjects with hereditary FX deficiency.

[1]  K. Khair,et al.  Successful use of BPL Factor X concentrate in a child with severe factor X deficiency , 2013 .

[2]  O. Christophe,et al.  Coagulation Factor X Interaction with Macrophages through Its N-Glycans Protects It from a Rapid Clearance , 2012, PloS one.

[3]  F. Peyvandi,et al.  Efficacy of prophylaxis and genotype‐phenotype correlation in patients with severe Factor X deficiency in Iran , 2012, Haemophilia : the official journal of the World Federation of Hemophilia.

[4]  P. Kouides,et al.  Diagnosis and treatment of inherited factor X deficiency , 2008, Haemophilia : the official journal of the World Federation of Hemophilia.

[5]  S. Knaub,et al.  Pharmacokinetics of Beriplex P/N prothrombin complex concentrate in healthy volunteers , 2007, Thrombosis and Haemostasis.

[6]  M. Morfini.  Pharmacokinetic studies: international guidelines for the conduct and interpretation of such studies , 2006 .

[7]  J. Korth-Bradley,et al.  Use of pharmacokinetics in the coagulation factor treatment of patients with haemophilia , 2005, Haemophilia : the official journal of the World Federation of Hemophilia.

[8]  D. Perry,et al.  The rare coagulation disorders – review with guidelines for management from the United Kingdom Haemophilia Centre Doctors' Organisation , 2004, Haemophilia : the official journal of the World Federation of Hemophilia.

[9]  P. Mannucci,et al.  Recessively inherited coagulation disorders. , 2004, Blood.

[10]  Tim Morris,et al.  Physiological Parameters in Laboratory Animals and Humans , 1993, Pharmaceutical Research.

[11]  T. Sumer,et al.  Severe congenital factor X deficiency with intracranial haemorrhage , 1986, European Journal of Pediatrics.

[12]  P. Mannucci,et al.  Rare coagulation deficiencies , 2002, Haemophilia : the official journal of the World Federation of Hemophilia.

[13]  P. Kouides,et al.  Prophylactic treatment of severe factor X deficiency with prothrombin complex concentrate , 2001, Haemophilia : the official journal of the World Federation of Hemophilia.

[14]  James R. Dixon,et al.  The International Conference on Harmonization Good Clinical Practice guideline. , 1999, Quality assurance.

[15]  P. Mannucci,et al.  Congenital factor X deficiency: spectrum of bleeding symptoms in 32 Iranian patients , 1998, British journal of haematology.

[16]  J. Miletich,et al.  The low-density lipoprotein receptor-related protein (LRP) mediates clearance of coagulation factor Xa in vivo. , 1998, Blood.

[17]  P. Mehta,et al.  Congenital coagulopathies and pregnancy: Report of four pregnancies in a factor X‐deficient woman , 1994, American journal of hematology.

[18]  B. Alving,et al.  Replacement therapy for congenital Factor X deficiency , 1985, Transfusion.

[19]  S. Lewis.  British Committee for Standards in Haematology , 1969 .

[20]  H. Roberts,et al.  Survival of Transfused Factor X in Patients with Stuart Disease , 1965, Thrombosis and Haemostasis.