Efficacy, safety and pharmacokinetics of a new high-purity factor X concentrate in subjects with hereditary factor X deficiency

Introduction Hereditary factor X (FX) deficiency is a rare bleeding disorder affecting 1:500 000 to 1:1 000 000 of individuals. Until recently, no specific replacement factor concentrate was available. Aim The aim of this study was to assess safety and efficacy of a new, high‐purity plasma‐derived F...

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Veröffentlicht in:	Haemophilia : the official journal of the World Federation of Hemophilia 2016-05, Vol.22 (3), p.419-425
Hauptverfasser:	Austin, S. K., Kavakli, K., Norton, M., Peyvandi, F., Shapiro, A.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Adverse events Antibodies, Neutralizing - blood Blood Coagulation Tests Child clinical trial clotting factor concentrate Coagulation factors efficacy Factor X - adverse effects Factor X - pharmacokinetics Factor X - therapeutic use factor X deficiency Factor X Deficiency - congenital Factor X Deficiency - drug therapy Factor X Deficiency - pathology Female Half-Life Hemorrhage - prevention & control Humans Hypersensitivity Laboratories Male Menorrhagia - prevention & control Middle Aged orphan drug Pharmacokinetics Prophylaxis Safety Severity of Illness Index Treatment Outcome Young Adult
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Zusammenfassung:	Introduction Hereditary factor X (FX) deficiency is a rare bleeding disorder affecting 1:500 000 to 1:1 000 000 of individuals. Until recently, no specific replacement factor concentrate was available. Aim The aim of this study was to assess safety and efficacy of a new, high‐purity plasma‐derived FX concentrate (pdFX) in subjects with hereditary FX deficiency. Methods Subjects aged ≥12 years with moderate or severe FX deficiency (plasma FX activity
ISSN:	1351-8216 1365-2516
DOI:	10.1111/hae.12893