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Successful use of BPL Factor X concentrate in a child with severe factor X deficiency

Successful use of BPL Factor X concentrate in a child with severe factor X deficiency AbstractIntroductionSevere factor X deficiency is a rare serious bleeding disorder historically treated with fresh frozen plasma (FFP) and more recently with prothrombin complex concentrate (PCC) which contains activated factors II, VII, IX and X. The infusion volume of PCC is smaller than FFP, but there is a risk of thromboembolic complications given the presence of activated forms of vitamin K-dependent factor concentrates when treating an isolated coagulation factor deficiency.MethodsWe describe the case of a nine-year-old girl of consanguineous origin with co-existent congenital merosin deficient muscular dystrophy and severe factor X deficiency treated with twice-weekly PCC prophylaxis via an indwelling central venous access device (CVAD). Infusion occlusion of her fifth CVAD occurred 24-months post-insertion; thrombus within the right subclavian and brachiocephalic veins was seen on radiological imaging. She started peripheral treatment with BPL Factor X concentrate as infusion volumes were smaller and given her immobility further thrombotic risk was predicted to be reduced. A sixth CVAD was inserted seven months later and BPL Factor X prophylaxis was continued.ResultsBPL Factor X concentrate was effective in maintaining trough levels of 13IU/ml 72-hours post-dose, with no intercurrent bleeding episodes or further problems in terms of occlusion of her portacath. Further radiological screening has not been undertaken.ConclusionBPL Factor X has been shown to be a safe and effective alternative to PCC for treatment of severe factor X deficiency in this case. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png The Journal of Haemophilia Practice de Gruyter

Successful use of BPL Factor X concentrate in a child with severe factor X deficiency

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Publisher
de Gruyter
Copyright
© 2014 Kate Khair, Poornima Kumar, Mary Mathias, Jemma Efford, Ri Liesner, published by Sciendo
eISSN
2055-3390
DOI
10.17225/jhp.00017
Publisher site
See Article on Publisher Site

Abstract

AbstractIntroductionSevere factor X deficiency is a rare serious bleeding disorder historically treated with fresh frozen plasma (FFP) and more recently with prothrombin complex concentrate (PCC) which contains activated factors II, VII, IX and X. The infusion volume of PCC is smaller than FFP, but there is a risk of thromboembolic complications given the presence of activated forms of vitamin K-dependent factor concentrates when treating an isolated coagulation factor deficiency.MethodsWe describe the case of a nine-year-old girl of consanguineous origin with co-existent congenital merosin deficient muscular dystrophy and severe factor X deficiency treated with twice-weekly PCC prophylaxis via an indwelling central venous access device (CVAD). Infusion occlusion of her fifth CVAD occurred 24-months post-insertion; thrombus within the right subclavian and brachiocephalic veins was seen on radiological imaging. She started peripheral treatment with BPL Factor X concentrate as infusion volumes were smaller and given her immobility further thrombotic risk was predicted to be reduced. A sixth CVAD was inserted seven months later and BPL Factor X prophylaxis was continued.ResultsBPL Factor X concentrate was effective in maintaining trough levels of 13IU/ml 72-hours post-dose, with no intercurrent bleeding episodes or further problems in terms of occlusion of her portacath. Further radiological screening has not been undertaken.ConclusionBPL Factor X has been shown to be a safe and effective alternative to PCC for treatment of severe factor X deficiency in this case.

Journal

The Journal of Haemophilia Practice – de Gruyter

Published: May 1, 2013

References

  • Kouides P Diagnosis and treatment of inherited factor X deficiency
  • Factor X deficiency
  • Kouides P Diagnosis and treatment of inherited factor X deficiency
  • Factor X deficiency
  • Prophylaxis in rare coagulation disorders – factor X deficiency
  • Prophylaxis in rare coagulation disorders – factor X deficiency