Treatment of epistaxis in hereditary hemorrhagic telangiectasia with tranexamic acid: a double-blind placebo-controlled cross-over phase IIIB study

Introduction - Epistaxis is the most frequent manifestation in hereditary hemorrhagic telangiectasia, in which no optimal treatment exists. It can lead to severe anemia and reduced quality of life. Positive effects of tranexamic acid, an antifibrinolytic drug, have been reported on epistaxis related...

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Main Authors: Geisthoff, Urban Wilhelm (Author) , Seyfert, Ulrich T. (Author) , Kübler, Marcus (Author) , Bieg, Birgitt (Author) , Plinkert, Peter K. (Author) , König, Jochem (Author)
Format: Article (Journal)
Language:English
Published: 16 June 2014
In: Thrombosis research
Year: 2014, Volume: 134, Issue: 3, Pages: 565-571
ISSN:1879-2472
DOI:10.1016/j.thromres.2014.06.012
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1016/j.thromres.2014.06.012
Verlag, lizenzpflichtig, Volltext: http://www.sciencedirect.com/science/article/pii/S0049384814003387
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Author Notes:Urban W. Geisthoff, Ulrich T. Seyfert, Marcus Kübler, Birgitt Bieg, Peter K. Plinkert, Jochem König

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520 |a Introduction - Epistaxis is the most frequent manifestation in hereditary hemorrhagic telangiectasia, in which no optimal treatment exists. It can lead to severe anemia and reduced quality of life. Positive effects of tranexamic acid, an antifibrinolytic drug, have been reported on epistaxis related to this disorder. We sought to evaluate the efficacy of treating nosebleeds in hereditary hemorrhagic telangiectasia with tranexamic acid. - Materials and Methods - In a randomized, double-blind, placebo controlled, cross-over phase IIIB study, 1 gram of tranexamic acid or placebo was given orally 3 times daily for 3months for a total of 6months. - Results - 22 patients were included in the intention-to-treat analysis. Hemoglobin levels, the primary outcome measure, did not change significantly (p=0.33). The secondary outcome measure was epistaxis score and patients reported a statistically significant reduction in nosebleeds, equaling a clinically relevant 54% diminution (p=0.0031), as compared to the placebo period. No severe side effects were observed. - Conclusion - Tranexamic acid reduces epistaxis in patients with hereditary hemorrhagic telangiectasia. (Clinical trial registration numbers: BfArM 141 CHC 9008-001 and ClinicalTrials.gov NCT01031992) 
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