PMID- 27217097
OWN - NLM
STAT- MEDLINE
DCOM- 20170223
LR  - 20170223
IS  - 1365-2516 (Electronic)
IS  - 1351-8216 (Linking)
VI  - 22
IP  - 5
DP  - 2016 Sep
TI  - Experience of a new high-purity factor X concentrate in subjects with hereditary 
      factor X deficiency undergoing surgery.
PG  - 713-20
LID - 10.1111/hae.12954 [doi]
AB  - 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] <20 IU dL(-1) ) undergoing surgery received pdFX 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 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.
CI  - (c) 2016 John Wiley & Sons Ltd.
FAU - Escobar, M A
AU  - Escobar MA
AD  - University of Texas Health Science Center and Gulf States Hemophilia and 
      Thrombophilia Center, Houston, TX, USA.
FAU - Auerswald, G
AU  - Auerswald G
AD  - Klinikum Bremen-Mitte, Prof-Hess-Kinderklinik, Bremen, Germany.
FAU - Austin, S
AU  - Austin S
AD  - St. George's Hospital University NHS Foundation Trust, London, UK.
FAU - Huang, J N
AU  - Huang JN
AD  - University of California San Francisco Benioff Children's Hospital, San 
      Francisco, CA, USA.
FAU - Norton, M
AU  - Norton M
AD  - Bio Products Laboratory, Elstree, UK. miranda.norton@bpl.co.uk.
FAU - Millar, C M
AU  - Millar CM
AD  - Centre for Haematology, Hammersmith Campus, Imperial College Academic Health 
      Sciences Centre, Imperial College, London, UK.
LA  - eng
PT  - Clinical Trial, Phase III
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
DEP - 20160524
PL  - England
TA  - Haemophilia
JT  - Haemophilia : the official journal of the World Federation of Hemophilia
JID - 9442916
RN  - 0 (Coagulants)
RN  - 0 (Hemoglobins)
RN  - 9001-29-0 (Factor X)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Coagulants/analysis/isolation & purification/*therapeutic use
MH  - Factor X/analysis/isolation & purification/*therapeutic use
MH  - Factor X Deficiency/*drug therapy/pathology
MH  - Female
MH  - Hemoglobins/analysis
MH  - Hemorrhage/prevention & control
MH  - Humans
MH  - Male
MH  - Middle Aged
MH  - Preoperative Care
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  - *safety
OT  - *surgery
EDAT- 2016/05/25 06:00
MHDA- 2017/02/24 06:00
CRDT- 2016/05/25 06:00
PHST- 2016/03/14 00:00 [accepted]
PHST- 2016/05/25 06:00 [entrez]
PHST- 2016/05/25 06:00 [pubmed]
PHST- 2017/02/24 06:00 [medline]
AID - 10.1111/hae.12954 [doi]
PST - ppublish
SO  - Haemophilia. 2016 Sep;22(5):713-20. doi: 10.1111/hae.12954. Epub 2016 May 24.