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References (8)
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Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
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Mark Brooker (2000)
The world federation of hemophiliaHaemophilia : the official journal of the World Federation of Hemophilia, 6 2
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Introduction. Rare bleeding disorders: general aspects of clinical features, diagnosis, and management
Semin Thromb Hemost, 35
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Coagulation factor activity and clinical bleeding severity in rare bleeding disorders: results from the European Network of Rare Bleeding Disorders
J Thromb Haemost, 10
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(2013)
Useful information on rare diseases from an EU perspective -
E. Rodríguez‐Merchán (2000)
World Federation of Hemophilia.The Journal of bone and joint surgery. American volume, 82 8
- Publisher
- de Gruyter
- Copyright
- © 2015 Flora Peyvandi, published by sciendo
- eISSN
- 2055-3390
- DOI
- 10.17225/jhp00051
- Publisher site
- See Article on Publisher Site
Abstract
AbstractThe development of registries through international collaboration has facilitated better understanding of the rare bleeding disorders. Such work has shown that rare bleeding disorders are heterogeneous and need to be studied singularly, and that heterozygous patients may bleed. There is a need to understand the minimum plasma coagulant activity level to prevent spontaneous bleeding. Moreover, due to the low prevalence of rare bleeding disorders, the management of this patient population remains a challenge. Data collection on clinical history, efficacy and side effects of treatment needs to be harmonised.
Journal
The Journal of Haemophilia Practice – de Gruyter
Published: Jul 1, 2015