PMID- 29707881
OWN - NLM
STAT- MEDLINE
DCOM- 20190204
LR  - 20190215
IS  - 1365-2516 (Electronic)
IS  - 1351-8216 (Linking)
VI  - 24
IP  - 6
DP  - 2018 Nov
TI  - Prophylactic treatment of bleeding episodes in children <12 years with moderate 
      to severe hereditary factor X deficiency (FXD): Efficacy and safety of a 
      high-purity plasma-derived factor X (pdFX) concentrate.
PG  - 941-949
LID - 10.1111/hae.13500 [doi]
AB  - BACKGROUND: Hereditary factor X (FX) deficiency (FXD) affects 
      1:500 000-1:1 000 000 people worldwide. A novel, high-purity plasma-derived FX 
      concentrate (pdFX) is available in the United States and European Union as 
      replacement therapy for FXD, but data are scarce on pdFX use in children 
      <12 years. AIM: This prospective, open-label phase 3 study assessed the safety, 
      efficacy and pharmacokinetics of pdFX in children <12 years with moderate/severe 
      FXD. METHODS: Subjects aged <12 years with basal plasma FX activity (FX:C) <5 
      IU/dL received pdFX as prophylactic and on-demand treatment, with doses adjusted 
      to maintain FX:C > 5 IU/dL. After >/=26 weeks and >/=50 exposure days, investigators 
      rated pdFX efficacy for preventing/decreasing bleeds. Secondary endpoints 
      included number and severity of bleeds, trough FX:C and incremental recovery. 
      Safety parameters were adverse events (AEs), inhibitor development and changes in 
      laboratory parameters. RESULTS: The study enrolled 9 subjects (0-5 years, n = 4; 
      6-11 years, n = 5) with severe (n = 8) or moderate (n = 1) FXD. At end of study, 
      investigators rated pdFX efficacy excellent for all subjects. Ten bleeds occurred 
      (n = 3 subjects; 6 major, 3 minor, 1 unassessed for severity). Trough FX:C levels 
      remained >5 IU/dL for all subjects after the last dose adjustment study visit. 
      Mean incremental recovery was significantly lower for younger vs older subjects 
      (1.53 vs 1.91 IU/dL per IU/kg; P = .001). All AEs were unrelated to treatment; no 
      inhibitor development or clinically significant changes in laboratory parameters 
      were observed. CONCLUSIONS: These results demonstrate the efficacy and safety of 
      pdFX for treating children <12 years with moderate/severe hereditary FXD.
CI  - (c) 2018 The Authors Haemophilia Published by John Wiley & Sons Ltd.
FAU - Liesner, R
AU  - Liesner R
AD  - Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital, London, UK.
FAU - Akanezi, C
AU  - Akanezi C
AUID- ORCID: 0000-0001-5454-3893
AD  - Bio Products Laboratory Ltd, Elstree, UK.
FAU - Norton, M
AU  - Norton M
AD  - Bio Products Laboratory Ltd, Elstree, UK.
FAU - Payne, J
AU  - Payne J
AD  - Department of Paediatric Haematology, Sheffield Children's NHS Foundation Trust, 
      Sheffield, UK.
LA  - eng
GR  - Bio Products Laboratory/
PT  - Clinical Trial, Phase III
PT  - Journal Article
DEP - 20180430
PL  - England
TA  - Haemophilia
JT  - Haemophilia : the official journal of the World Federation of Hemophilia
JID - 9442916
RN  - 9001-29-0 (Factor X)
SB  - IM
MH  - Child
MH  - Child, Preschool
MH  - Dose-Response Relationship, Drug
MH  - Factor X/adverse effects/metabolism/pharmacokinetics/*pharmacology
MH  - Factor X Deficiency/*complications
MH  - Female
MH  - Hemorrhage/*complications/*prevention & control
MH  - Humans
MH  - Infant
MH  - Infant, Newborn
MH  - Male
MH  - Plasma/*metabolism
MH  - *Safety
OTO - NOTNLM
OT  - clotting factor concentrate
OT  - efficacy
OT  - factor X deficiency
OT  - paediatric
OT  - safety
EDAT- 2018/05/01 06:00
MHDA- 2019/02/05 06:00
CRDT- 2018/05/01 06:00
PHST- 2018/03/21 00:00 [accepted]
PHST- 2018/05/01 06:00 [pubmed]
PHST- 2019/02/05 06:00 [medline]
PHST- 2018/05/01 06:00 [entrez]
AID - 10.1111/hae.13500 [doi]
PST - ppublish
SO  - Haemophilia. 2018 Nov;24(6):941-949. doi: 10.1111/hae.13500. Epub 2018 Apr 30.