PMID- 27197801
OWN - NLM
STAT- MEDLINE
DCOM- 20170216
LR  - 20170216
IS  - 1365-2516 (Electronic)
IS  - 1351-8216 (Linking)
VI  - 22
IP  - 3
DP  - 2016 May
TI  - Efficacy, safety and pharmacokinetics of a new high-purity factor X concentrate 
      in subjects with hereditary factor X deficiency.
PG  - 419-25
LID - 10.1111/hae.12893 [doi]
AB  - 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 <5 IU dL(-1) ) 
      received 25 IU kg(-1) pdFX as on-demand treatment or short-term prophylaxis for 6 
      months to 2 years. Subjects assessed pdFX efficacy for each bleed; at 
      end-of-study, investigators assessed overall pdFX efficacy. Blood samples for 
      pharmacokinetic analysis were obtained at baseline and >/=6 months. Safety was 
      assessed by adverse events (AEs), inhibitor development and changes in laboratory 
      parameters. RESULTS: Sixteen enrolled subjects (six aged 12-17 years; 10 aged 
      18-58 years) received a total of 468 pdFX infusions. In the 187 analysed bleeds, 
      pdFX efficacy was categorized as excellent, good, poor or unassessable in 90.9%, 
      7.5%, 1.1% and 0.5% of bleeds respectively; 83% of bleeds were treated with one 
      infusion. For pdFX, mean (median; interquartile range) incremental recovery and 
      half-life were 2.00 (2.12; 1.79-2.37) IU dL(-1) per IU kg(-1) and 29.4 (28.6; 
      25.8-33.1) h respectively. No serious AEs possibly related to pdFX or evidence of 
      FX inhibitors were observed, and no hypersensitivity reactions or clinically 
      significant trends were detected in laboratory parameters. CONCLUSION: These 
      results demonstrate that a dose of 25 IU kg(-1) pdFX is safe and efficacious for 
      on-demand treatment and short-term prophylaxis in subjects with moderate or 
      severe hereditary FX deficiency.
CI  - (c) 2016 John Wiley & Sons Ltd.
FAU - Austin, S K
AU  - Austin SK
AD  - St. George's Haemophilia Centre, St. George's University Hospitals NHS Foundation 
      Trust, London, UK.
FAU - Kavakli, K
AU  - Kavakli K
AD  - Department of Pediatric Hematology, Children's Hospital, Ege University Faculty 
      of Medicine, Izmir, Turkey.
FAU - Norton, M
AU  - Norton M
AD  - Bio Products Laboratory, Elstree, UK.
FAU - Peyvandi, F
AU  - Peyvandi F
AD  - Angelo Bianchi Bonomi Hemophilia & Thrombosis Center, Milan, Italy.
AD  - Department of Pathophysiology and Transplantation, Universita degli Studi di 
      Milano, Milan, Italy.
FAU - Shapiro, A
AU  - Shapiro A
AD  - Indiana Hemophilia & Thrombosis Center, Indianapolis, IN, USA.
CN  - FX Investigators Group
LA  - eng
PT  - Clinical Trial, Phase III
PT  - Journal Article
PT  - Multicenter Study
DEP - 20160308
PL  - England
TA  - Haemophilia
JT  - Haemophilia : the official journal of the World Federation of Hemophilia
JID - 9442916
RN  - 0 (Antibodies, Neutralizing)
RN  - 9001-29-0 (Factor X)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Antibodies, Neutralizing/blood
MH  - Blood Coagulation Tests
MH  - Child
MH  - Factor X/adverse effects/pharmacokinetics/*therapeutic use
MH  - Factor X Deficiency/congenital/*drug therapy/pathology
MH  - Female
MH  - Half-Life
MH  - Hemorrhage/prevention & control
MH  - Humans
MH  - Male
MH  - Menorrhagia/prevention & control
MH  - Middle Aged
MH  - Severity of Illness Index
MH  - Treatment Outcome
MH  - Young Adult
OTO - NOTNLM
OT  - clinical trial
OT  - clotting factor concentrate
OT  - efficacy
OT  - factor X deficiency
OT  - orphan drug
OT  - safety
FIR - Roman, Maria-Teresa Alvarez
IR  - Roman MT
FIR - Auerswald, Gunter
IR  - Auerswald G
FIR - Vega, Nuria Bermejo
IR  - Vega NB
FIR - Celkan, Tiraje
IR  - Celkan T
FIR - Huang, James N
IR  - Huang JN
FIR - Beau Mitchell, W
IR  - Beau Mitchell W
FIR - Oner, Ahmet Faik
IR  - Oner AF
FIR - Pavord, Susannah
IR  - Pavord S
FIR - Timur, Cetin
IR  - Timur C
EDAT- 2016/05/21 06:00
MHDA- 2017/02/17 06:00
CRDT- 2016/05/21 06:00
PHST- 2015/12/04 00:00 [accepted]
PHST- 2016/05/21 06:00 [entrez]
PHST- 2016/05/21 06:00 [pubmed]
PHST- 2017/02/17 06:00 [medline]
AID - 10.1111/hae.12893 [doi]
PST - ppublish
SO  - Haemophilia. 2016 May;22(3):419-25. doi: 10.1111/hae.12893. Epub 2016 Mar 8.